BIOMARIN PHARMACEUTICAL INC — Call Transcript 2026

At this time, I would like to turn the conference over to Traci McCarty, Head of Investor Relations. Please go ahead. Thank you, operator. To remind you, this non-confidential presentation contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including expectations regarding BioMarin's financial performance, commercial products, and potential future products in different areas of therapeutic research and development. Results may differ materially depending on the progress of BioMarin's product programs, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical market, and developments by competitors, and those factors detailed in BioMarin's filings with the Securities and Exchange Commission such as 10-Q, 10-K, and 8-K reports. In addition, we will use non-GAAP financial measures as defined in Regulation G during the call today. These non-GAAP measures should not be considered in isolation from, as substitutes for, or superior to financial measures prepared in accordance with U.S. GAAP, and you can find the related reconciliations to U.S. GAAP in the earnings release and earnings presentation, both of which are now available in the investor relations section of our website. Please note that our commentary on today's call will focus on non-GAAP financial measures, unless otherwise indicated. Beginning on slide three in introducing BioMarin's management team, joining today's call are Alexander Hardy, Chief Executive Officer, Brian Mueller, Chief Financial Officer, Cristin Hubbard, Chief Commercial Officer, and Greg Freiberg, Chief R&D Officer. I will now turn the call over to BioMarin's President and CEO, Alexander Hardy. Thank you, Traci, and thank you all for joining us today. I am so pleased that we completed the Amicus acquisition last week, starting a new and exciting chapter for BioMarin with the addition of two innovative therapies, Galafold for Fabry disease and Pombiliti and Opfolda for Pompe disease. The acquisition accelerates our anticipated year-over-year 2026 revenue growth to 20% at the midpoint of today's updated guidance. This strengthening trajectory is just the beginning of BioMarin's enhanced longer-term financial outlook, supported by our larger, more diversified commercial portfolio. Since announcing our plans to acquire Amicus late last year, we have been preparing for rapid integration beginning on day one, with the goal of increasing the peak potential of these newly added products. Following the recent close of the transaction, we initiated our targeted integration plan focused on leveraging BioMarin's operating scale and capabilities to drive diagnosis and treatment rates for patients with Fabry disease and late-onset Pompe disease. Next quarter, we plan to share this roadmap, as well as more detail on BioMarin's growth acceleration with the addition of Galafold and Pombiliti and Opfolda to our commercial portfolio and DMX-200 for FSGS in phase III to our late-stage pipeline. Turning briefly to first quarter results and outlook for the remainder of this year and starting with enzyme therapies, I am pleased with the strong interest we are seeing from the PKU community following the recent Palynziq adolescent label expansion in the U.S., which Cristin will discuss in more detail. We expect enzyme therapies will deliver robust growth in 2026, further supported by the addition of Galafold and Pombiliti and Opfolda to the portfolio. Moving to our skeletal conditions business unit, we saw strong patient demand for Voxzogo, with new patient starts increasing across all regions in the 1st quarter. In the U.S., the majority of new patient starts were from the under age two cohort. These results reflect our focused investments in increasing adoption of Voxzogo, particularly among younger patients. Building on our leadership in achondroplasia, we are pleased to have submitted the sNDA for approval of Voxzogo. We expect to hear the timing of our review in the coming months. I want to congratulate our regulatory team for the outstanding work that went into this comprehensive submission package. We also look forward to pivotal results for Voxzogo in hypochondroplasia and BMN 401 for ENPP1 deficiency both later in Q2. In summary, we expect 2026 to be a momentous year for BioMarin. Our immediate focus remains on the seamless and rapid integration of Amicus to accelerate our growth trajectory this year and beyond and pursuing regulatory next steps following the two upcoming pivotal data readouts. The addition of Galafold and Pombiliti and Opfolda to BioMarin's portfolio of innovative medicines provides an opportunity to reach more patients around the world, creating significant value for all of our stakeholders in the near and longer term. As we enter this next chapter, I would like to express my appreciation to the employees of both BioMarin and Amicus, whose dedication forms the foundation of our shared mission to serve patients. Thank you for your attention. I will now turn the call over to Brian to provide additional financial updates. Brian? Thank you, Alexander. Please refer to today's press release for detailed first quarter 2026 results, including reconciliations of GAAP to non-GAAP financial measures. All first quarter results will be available in our upcoming Form 10-Q, which we expect to file in the coming days. Moving to slide seven. Total revenues in the first quarter were $766 million and increased year-over-year, supported by increased patient demand across both enzyme therapies and Voxzogo. As expected, those organic growth drivers were partly offset by order timing dynamics as well as lower revenue from Roctavian, Kuvan, and royalties. Enzyme therapies revenue increased 6% year-over-year, led by growth in Vimizim, Naglazyme, and Brineura. Palynziq's first quarter revenues were impacted by U.S. order timing, which resulted in elevated stocking levels in the fourth quarter of 2025, as discussed last quarter. We expect this stocking dynamic to normalize and anticipate year-over-year revenue growth for Palynziq in full year 2026 as growth in new patient starts in the under 18-year-old population gains momentum and patients continue to titrate to their maintenance dose. Voxzogo revenue was supported by new patient starts across all regions and in line with the expectations that we shared on our prior quarter earnings call. Looking ahead, due to anticipated order timing and consistent with 2025, we expect Voxzogo revenue to be higher in the second half of 2026 compared to the first half. Cristin will provide more color on commercial dynamics in a moment. Turning now to slide 8. Cost of sales increased year-over-year in the first quarter, primarily due to a $31 million charge associated with an unsuccessful process qualification campaign to extend Naglazyme manufacturing capabilities. Importantly, this did not impact commercial supply. While this event decreased margins and earnings per share in the first quarter, we expect this charge to be offset in full year 2026 non-GAAP diluted earnings per share guidance. Q1 non-GAAP R&D expense increased year-over-year, primarily due to spend to support BMN 401, our phase III clinical program acquired in the enzyme transaction in the second half of 2025. Development activities for Voxzogo for hypochondroplasia, BMN 333 for achondroplasia, and BMN 351 for Duchenne muscular dystrophy also contributed to higher year-over-year R&D expense. Non-GAAP SG&A expense also increased, partially driven by investments to support commercial expansion across enzyme therapies and Voxzogo, and partially driven by pre-close costs associated with the Amicus acquisition. First quarter non-GAAP diluted earnings per share was $0.76 and was significantly impacted by the drivers of increased operating expense that I just mentioned, as well as the impact of Q1 revenue, which we believe will be our lowest quarter of the year. The impact of the cost of sales charge and pre-close costs associated with the Amicus acquisition resulted in a $0.20 earnings per share impact to our non-GAAP earnings per share result. Looking past those elements helps to measure BioMarin's underlying business performance, as well as how this Q1 result fits into our full-year earnings per share guidance, which remains unchanged for the historical BioMarin business before layering on the Amicus business. Moving to slide nine and our updated full-year 2026 guidance, which now includes the Amicus financial outlook starting last week. We are raising enzyme therapies revenue guidance to a range of $2.725 billion-$2.775 billion for the full-year 2026, inclusive of meaningful contributions from Galafold and Pombiliti and Opfolda, resulting in approximately 30% growth at the midpoint. Adding these high-growth products to our enzyme therapies portfolio increases our full-year total revenue guidance to a range of $3.825 billion to $3.925 billion, with the midpoint representing approximately 20% year-over-year growth in 2026. For Voxzogo, we are maintaining our revenue guidance of $975 million to $1.025 billion, which continues to reflect high single-digit growth at the midpoint. On non-GAAP diluted earnings per share guidance, we are updating the guidance range to $4.85 to $5.05. As previously communicated, the acquisition of Amicus will be slightly diluted for the full year 2026. We continue to expect the acquisition to be accretive to non-GAAP diluted earnings per share in the first 12 months after close and substantially accretive beginning in 2027. The Amicus P&L will be incorporated into BioMarin's financial results as of last week's closing of the transaction. In 2026, we expect to include the base Amicus operating expenses less initial cost synergies anticipated in 2026. As Alexander touched on, now that the transaction is closed, we have engaged more deeply with the Amicus business and plan to share our outlook on both commercial revenues and cost synergies on our next quarterly earnings call. To give some perspective on timing of the updated guidance. We expect order timing for the historical BioMarin products and 2 full quarters of Galafold and Pombiliti and Opfolda revenues in the second half of the year to drive significantly higher revenues as compared to the first half of 2026. To provide context, we expect more than 55% of total 2026 revenues to be recognized in the second half of the year. Likewise, on the expected timing of our profitability, we expect Q2 non-GAAP diluted earnings per share to be just modestly higher than Q1, partially due to pre-close Amicus costs incurred in April, plus a higher amount of the 2026 Amicus dilution being weighted to the second quarter. Further, the revenue timing weighted to the second half of the year results in most of our expected profitability occurring in Q3 and Q4. These earnings timing dynamics drive approximately two-thirds of our 2026 earnings per share expected in the second half of the year. Briefly, on evolving geopolitical uncertainties, we are watching the situation in the Middle East very closely and note that today's guidance reflects an allowance for a modest amount of disruption in that region in 2026. Looking ahead, we look forward to sharing with you our expanded financial outlook, enhanced by the addition of Galafold and Pombiliti and Opfolda, setting the stage for accelerated near and midterm growth. Thank you for your attention. I will now turn it over to Cristin for a commercial update. Cristin? Thank you, Brian. We were encouraged by the commercial execution across our portfolio so far this year, with strong patient demand across enzyme therapies and Voxzogo. We look forward to providing a more detailed commercial update on our plans to maximize the potential of both Galafold and Pombiliti and Opfolda next quarter. Our initial priorities therein are focused on driving diagnosis in Fabry and switch in Pompe. This will support increased penetration in countries where Galafold and Pombiliti and Opfolda are marketed, while we can currently work on geographic expansion plans for both products. We look forward to updating you on our Q2 call. Now, moving to Slide 11. I'll begin with an update on first quarter 2026 performance, starting with enzyme therapies. As Brian outlined, enzyme therapies delivered 6% year-over-year growth in Q1, led by Vimizim, Naglazyme, and Brineura. Across the enzyme therapies portfolio, we continue to see strong patient demand and adherence. Turning to Palynziq. In the first quarter, we continued to expand the underlying patient base, and physician engagement remained strong. Importantly, following the FDA approval of Palynziq's age label expansion to those 12 years and older in February, we have successfully launched in this age group and are seeing encouraging early momentum. We have observed broad interest and engagement from caregivers and healthcare providers treating adolescents during this critical stage of their development. Since approval, we have observed meaningful enrollments and new patient starts in people under the age of 18. From a prescribing standpoint, adolescent uptake is being driven by both physicians with significant experience prescribing Palynziq, as well as by clinicians who are newer to the therapy. Recall that it can take a patient many months to titrate up to their maintenance dose of Palynziq. We would expect to see the results of positive early prescribing over the coming quarters. Palynziq continues to stand alone in its ability to enable people with PKU to reach physiologic Phe levels while reducing dietary restrictions, regardless of severity. With the U.S. adolescent launch underway and European approval expected later this year, we are excited about the impact Palynziq can have for additional families over time. Turning now to Voxzogo on Slide 12. As discussed, first quarter results reflected expected order timing dynamics following a strong Q-four, particularly in international markets. Importantly, we observed strong growth in patient additions globally, with the number of children being treated with Voxzogo increasing by more than 20% year-over-year. With the competitor having recently entered the U.S. market, our focus has remained on executing our strategy. We continue to see strong momentum. During the first quarter, Voxzogo progress continued, supported by ongoing patient additions across all regions and ages, strong adherence and persistence globally, and continued expansion of the prescriber base. Our teams are focused on driving new patient starts across all ages, with an emphasis on children under two years of age, where international consensus guidelines for achondroplasia recommend early diagnosis and treatment with Voxzogo as soon as possible. In the United States, we are encouraged to see that our efforts educating and engaging with caregivers and HCPs are having an impact in the under two year-old age segment. In Q1, over half of new patient starts were from children under age two, a greater proportion compared to last quarter. Additionally, we have seen an approximate 10% decrease in the average age of children initiating Voxzogo treatment in the under two segment, narrowing the window between diagnosis and treatment starts. Internationally, building on our global expansion into 55 countries to date, our strategy remains focused on reaching more patients in regions that have further opportunity and treating infants in countries that already have high penetration rates. With our sNDA for full approval now submitted, we believe the depth and durability of Voxzogo's clinical evidence can be further reinforced, strengthening its role in treatment decisions for infants and children with achondroplasia. At the same time, we're making progress in preparations for potential expansion of Voxzogo into hypochondroplasia. As we get closer to the phase III data and potential launch, our pre-commercialization activities continue to accelerate. Our initiatives in the U.S. are making a difference. More people are being diagnosed at a younger age, diagnosis rates are rising, and more doctors are requesting diagnostic tests. We look forward to the phase III top-line results in the 2nd quarter of 2026, and submitting to global health authorities in the 2nd half of this year, with potential approval in 2027. In summary, we are pleased with the strong patient demand observed across the portfolio with continued momentum in our core business units and a compelling set of near-term opportunities to accelerate growth. With that, I'll turn it over to Greg for an R&D update. Greg? Thank you, Cristin. As expected, 2026 is shaping up to be an active year for R&D, and we're looking forward to delivering multiple meaningful milestones. Moving to slide 14, we've built a comprehensive Voxzogo evidence package that goes well beyond describing annualized growth velocity, highlighting long-term durability and clinically meaningful health outcomes for children with achondroplasia. Recently, at the Pediatric Endocrine Society annual meeting, we shared data from three ongoing long-term extension studies that illustrate these sustained benefits of Voxzogo over time. Consistent and cumulative improvements in arm span Z-scores were observed across all age groups over the six to eight years of follow-up, as depicted on the left of slide 14. On the right side, you again see consistent and cumulative gains in height and height Z-scores with durable results out to eight years of follow-up. In addition to anatomic measures of benefit, we also presented data demonstrating Voxzogo's favorable impacts on quality-of-life measures. Importantly, these sustained efficacy findings are supported by a robust safety database now comprising more than 10,000 patient years of exposure, reinforcing Voxzogo's well-established long-term safety profile. Taken together, this extensive body of evidence reinforces Voxzogo's differentiated profile with no other achondroplasia therapy supported by this level of long-term data with regard to safety, efficacy, and functional outcomes. Importantly, this data spans the full pediatric age population, and Voxzogo remains the only therapy approved for use immediately from infancy. This allows for early treatment initiation and enables a long window to positively influence endochondral bone formation and all the potential downstream benefits to health outcomes. Together, these data formed a strong foundation for our full approval submission intended to fulfill our post-marketing requirement, which was submitted to the FDA in April and will afford us the opportunity to share direct evidence of Voxzogo's long-term value with the community via peer-reviewed publications and presentations later this year. Now moving to slide 15, the remainder of 2026 includes several anticipated pipeline updates, including two particularly important pivotal data readouts expected in the second quarter. Starting with hypochondroplasia, we believe the healthcare provider community is looking forward to a targeted therapy that addresses this skeletal condition, and we are confident in the scientific rationale for Voxzogo in this indication. That confidence is grounded in the strong proof of concept and durability demonstrated in Dr. Dauber's investigator-sponsored study and in the rapid enrollment we observed in our own phase III pivotal trial. We look forward to sharing these phase III top-line results in the second quarter. Enrollment is progressing very well in our phase II study in children under the age of three, highlighting early interest in Voxzogo as a potential option from infancy in hypochondroplasia. We also expect phase III top-line data for BMN 401 in the second quarter of this year. As a reminder, ENPP1 deficiency is a rare, serious, and progressive genetic condition affecting vascular, skeletal, and soft tissue systems. BMN 401 has the potential to be the first disease-targeted therapy for ENPP1 deficiency. The ENERGY-3 study in children aged 1 to 12 includes two co-primary endpoints. The first is change from baseline and plasma measures of inorganic pyrophosphate through week 52. The second is change in the RGIC, or Radiographic Global Impression of Change, after 52 weeks of treatment, assessing improvement in skeletal health. Clinical experience with BMN 401 in older patients has shown that normalization of pyrophosphate is accompanied by improvements in bone mineral biomarkers, functional performance, and patient and physician-reported outcomes. In addition to these pivotal readouts, I'd also like to highlight progress with BMN 333, our long-acting CNP therapy. We are pleased to share that enrollment is underway in our global registrational enabling phase II/III study. We are rapidly progressing this program with the goal of establishing BMN 333 as a potential next-generation standard of care for achondroplasia and potentially for other skeletal conditions. Before closing, I'd also like to touch on BMN 351 for Duchenne muscular dystrophy. At the Muscular Dystrophy Association Congress in March, we presented initial data demonstrating dose-dependent increases in dystrophin at week 25 in both the 6 and 9 milligram per kilogram dose cohorts. These findings were accompanied by notable decreases in creatine kinase, a biomarker of muscle injury, and the prevention of functional decline as measured by the North Star Ambulatory Assessment and the six-minute walk test when compared to historically matched controls. We are encouraged by both the dystrophin expression levels and the functional improvements observed in our development program. Enrollment in the 12 milligram per kilogram cohort is ongoing, and we look forward to providing an additional update by year-end as this program continues to advance. Finally, we would like to thank the patients, families, and caregivers whose commitment continues to make this progress possible. Thank you for your attention today. We will now open the call to your questions. Operator? Thank you. We will now begin the question and answer session. If you have dialed in and would like to ask a question, please press star one on your telephone keypad to raise your hand and join the queue. If you would like to withdraw your question, simply Press star one again. We'll go first to Sean Layman at Morgan Stanley. Hi, this is Mike on for Sean. Thank you for taking our questions. I guess wanted to touch on two things. First, looking at the recent data at the Pediatric Endocrine Society, can you help to contextualize the benefits you saw on bone mineral content and hypochondroplasia, and how does that maybe inform or influence your expectations heading into the top line? Yep. Thanks for the question, Mike. This is Greg Freiberg. Yeah, in addition to measuring growth velocity across both Achondroplasia historically and Hypochondroplasia, of course, we're measuring a variety of factors of health and well-being, including bone biology and bone health. The DEXA scans that were presented in that cumulative data suggests again that in addition to growing bone length and stimulating endochondral bone formation, that yes, that bone is of healthy and, you know, a strength that we would like to see, and again, reiterates what we've seen in the other FGFR3 related mutation condition of Achondroplasia as well. Going into the card flip for the phase III study, we're certainly excited to see that data. The event is going to occur before the midway point of this year. We mentioned that it's in the second quarter. We're eager to see those results. Again, I think the data at Pediatric Endocrine Society highlights the fact that hypochondroplasia, while a unique and individual indication, as compared to achondroplasia, the related biology suggests again that the hypothesis is a strong one. We've seen in Andrew Dauber's data from Children's National Hospital, again, we've seen the kind of growth that certainly would meet the criteria for a statistically significant improvement that our study is developed to measure. With regard to that safety profile, we're not anticipating any unexpected events there as well. Fingers crossed. We'll be updating you all when that data is available. Thank you so much. That's really helpful. Just maybe circling back onto the intro comments. Regarding the integration of Amicus, you alluded maybe there's like a roadmap for future growth acceleration. We were just wondering if you could help to comment, I mean specifically for Pombiliti or Opfolda, what levers do you see for driving increased switch rates in that market? Thank you. Yeah. Thank you so much. This is Cristin Hubbard here. Just having closed last week, I can say we are really, really excited about the commercial potential of both of these products, both being high-growth products to add to our portfolio. As we've mentioned before, but I'll expand a little bit, for Galafold specifically, we really do think that the biggest growth lever is in and around diagnosis. We know that a large proportion of the amenable patient population remains undiagnosed, and driving diagnosis is exactly where, you know, Amicus has really started to build momentum. We feel that using BioMarin's capability in this regard, we can really continue to drive that forward. On the Pombiliti and Opfolda side, that's more about a switching opportunity, we really do think that there's gonna be a sizable opportunity here to build over the next few years as patients on their existing therapies begin to progress. We're really working on initiatives that again, Amicus has started, and that is around kind of starting new therapy, looking at patients that have identified their own progression and understanding what progression looks like. That's an area that we're really focused on, not to mention the continued generation of evidence to show the benefits of a switch. Again, these are areas that we feel confident in our capability at BioMarin and are very excited about the future trajectory. We'll share more on that on our Q2 call. We'll move to our next question from Salveen Richter at Goldman Sachs. Thanks for taking our question. This is Tommy on for Salveen. Just a quick one on Voxzogo. Wondering if you've seen any early signals of different behavior from competitor entry, whether it be switching or in new patient adds. Thank you. Yeah. Thank you for the question. This is Cristin again. As you heard in our prepared comments, our demand in the first quarter really does remain strong. Our enrollments, in fact, in Q1, exceeded that in the average of second half last year, and that trend continued into April. As we see it now, we're really excited. As you know, we're very much focused on the 0 to 2 population based on the consensus guidelines, and we're seeing real momentum there. Not only were our new patient starts weighted and more than half of them being in that 0 to 2 population in the first quarter, but we're also seeing a reduction in the time we have from diagnosis to treatment in that population. That is reduced by 10% already. We know that that real focus therein is working. In addition, we're focused on the patients that are already on treatment and making sure that, you know, those patients for whom they're doing well on treatment, we wanna make sure that they understand the totality of the evidence that exists for Voxzogo and continue to have a positive experience in terms of the benefit. We're very pleased with how Q1 went and look forward to continuing on that trajectory. We'll go next to Paul Matteis at Stifel. Great. Thanks so much for taking my question. Just kind of previewing your update next quarter as it relates to the Amicus integration. I was wondering if you could set the stage for us on just what you might be providing as it relates to the duration of the revenue outlook. Would you talk about peak sales at all, or update those views or anything else? Secondarily, anything you can do on setting the stage on our expectations related to accretion, cost synergies, and again, the duration of profitability outlook as well. Thank you. Hi, Paul. Thanks. Appreciate the question. Yeah, we wanted to highlight today having, you know, just closed the transaction a week ago. You know, brief update on the integration to date, which a weekend is going well and you saw the update of our revenue guidance today, adding our expected range of the 8 months of Amicus revenues, resulting in a $500 million midpoint and the updated guidance, which is 20% year-over-year. We also wanted to set the stage for the Q2 update. From the transaction announcement, we've discussed how the strategic fit between these two businesses was very strong. That because of the both global and commercial and medical capabilities that BioMarin built over time in our existing portfolio, that these two medicines should truly have more potential within BioMarin. That was a key rationale underlying the transaction, and we've been making plans up to this point of closing. Now that we've closed, we're just digging in and engaging more deeply with the Amicus business. That's why we're gonna wait until Q2 to give this additional update. It will include a number of details and metrics about the long-term potential, including our views on peak revenue. Just a reminder, based on our due diligence at the time of announcement of the transaction, we reiterated what Amicus had shared with respect to peak revenue potential of roughly $1 billion each for Galafold and Pombiliti and Opfolda. But as we developed the long-term business plans and strategies to expand both of these medicines, we think that has some potential to be higher. That's what we'll share. It'll be important for us to share some of the key metrics and, you know, tactics in terms of how we expect to achieve that long-term potential. Stay tuned, and we'll look forward to sharing more in Q2. By the way, that will include some of the other financial elements as well, such as synergies, long-term profitability, accretion, et cetera. I will share that for today, we are reaffirming our previous expectation that while, as you saw in the EPS guide today, the Amicus acquisition is slightly dilutive to calendar 26, but accretive for the first 12 months following closing and then substantially accretive for beginning full year 27. We'll look forward to sharing more, but we're off to a strong start. We'll move next to Ellie Merle at Barclays. Hi, this is Jasmine on for Ellie. Thank you so much for taking our question. Just for Voxzogo and hypochondroplasia, what's the latest on what you're thinking will be good data and what you're looking to see on AGV? How are you thinking about the contribution to Voxzogo revenues from hypochondroplasia next year? What do you think the cadence of uptake will be in this population? Can you talk a little bit about your commercial preparations? Thanks. Thanks, Jasmine. This is Greg Freiberg. Maybe I'll take the first part and then hand it off to my colleague. With regard to the CANOPY-HCH-3 study we're turning over, of course, we'll be looking at the data in the second quarter of this year. Absolutely looking forward to that. Of course, the success is a statistically significant improvement in growth as compared to the control arm. You know, in this world, of course, we're measuring a variety of other measures. We're looking of course at other anthropomorphic measures, as well as of course the safety profile and really any statistically positive improvement in growth. We think that that's a win for these patients. I'll just add that, you know, we've seen pretty dramatic, I think accelerations of recruitment, both for the older children as well as, I mentioned in the pre-prepared remarks, the infants, on our hypochondroplasia study, really suggesting that this is a market that's hungry for a disease-targeted therapy. Again, the biology is significantly similar to that of achondroplasia with regard to the mutation that drives this condition. From that standpoint, and of course built upon the data that Andrew Dauber has seen, we're quite excited to see what those end results will be. I think there was a question also about the marketplace, and I'm gonna hand that one off to Cristin. Yeah. Thank you. Overall, our goal of course is to continue the growth of Voxzogo into the midterm, and hypochondroplasia provides an important component of that. Really where we're focused today on our pre-launch activities is in and around diagnosis. We have shared before that the total addressable patient population globally is at 14,000, and that assumes that we can continue to drive diagnosis and awareness of this condition. Our goal at this point in time prior to launch really is around increasing the number of hypochondroplasia patients that have been identified, that we're getting physicians to understand the genetic testing and make sure that they're putting in orders for that. Really, the idea being that patients get diagnosed at a much earlier age. Our goal today is to improve upon that and make sure that we have identified patients at the time of a potential launch. Jasmine, if I could just add one other factor from the medical affairs standpoint. We're obviously actively working in the community even prior to seeing our data to really try to accelerate the number of patients that are diagnosed with this condition. You know, there's probably three prongs to that. Of course, getting patients to be able to be referred sooner to be evaluated. Of course, more testing, as well as we're doing work on the genetic side to reclassify so-called VUSs, variants of uncertain significance. In that world, we're anticipating again that there will be patients available and ready for this therapy should it become available. Just to throw out a few components if I could quickly. On the success we've had already, when looking at the diagnosis program that we're running to date and these kind of non-promotional pre-launch activities, we're already seeing a 90% increase in the number of hypochondroplasia patients that we've identified, as well as a 70% reduction in the age. Those are precisely the types of numbers that we're driving for and wanna continue throughout our launch period. Excuse me. We'll move to our next question from Cory Kasimov at Evercore. Hi, this is Eddie on for Corey. Thanks for taking the question. I had a question on the guidance increase. The $500 million from midpoint increase in guidance, can you frame that? You know, the contribution from Galafold power up, is that conservative relative to the $600 million revenue they generated in FY 2025? What are the key assumptions for, you know, adding these two assets into the guidance? Hi, Eddie. This is Brian. Thanks a lot for the question. I appreciate the opportunity to provide a bit more color on this important element of today's update. Yeah, I'll frame it up for you. First of all, you know, the $500 million represents a midpoint of a range that fits within the range of our existing enzyme therapies guide for 2026, I'll speak to the midpoint. From a timing standpoint, just a reminder that with the transaction closing last week, that $500 million midpoint represents mostly the eight months remaining from May to December for this year. I will share in terms of, you know, your question around conservatism, I would say it's neither conservative nor aggressive. We think it's realistic. I'll share that, you know, we've analyzed the unreported period of Amicus product revenue from January through April. When we looked at that versus consensus, January through April performance, while not reported, was ahead of consensus. You know, both Galafold and Pombiliti together are off to a strong start in 2026. With that being said, again, it's, this is the transaction closed just a week ago. We'll come back next quarter with additional color, including the long-term potential. I'll also share that, since you commented on the $600 million reported for 2025, the range that's underlying today's update, from an Amicus, you know, organic year-over-year growth rate standpoint ranges from the high teens to the low 20s. We think it's a really healthy growth, and we're excited that these assets are now part of our portfolio. Again, you see this 30% increase year-over-year as a result of lay-layering this onto the BioMarin enzyme therapies and total revenue 20% at the midpoint. We're feeling good about it, and we'll share more going forward. Thank you. We'll go next to Jessica Fye at JPMorgan. Hi, this is Jose on for Jess. Thanks for taking our questions. How should we think about the adoption of Voxzogo in hypochondroplasia relative to achondroplasia? What do you see as the similarities and differences between these markets? Second, can you help us bridge the disconnect between the 20% year-over-year patient growth in 1Q versus a 3% revenue growth? Is it entirely explained by larger orders in the fourth quarter last year? Thank you. Yeah. I'll address the first part of the question in terms of how we see it, in terms of uptake going in. I think we can consider it being similar to that of achondroplasia. I think, as I'd mentioned kinda earlier, what is most important to us is ensuring that the amount of disease awareness, the urgency to treat, and the diagnosis are really what we're focused on because we believe this is what accelerates that adoption curve. Our intention has been to drive as much of that so that we have patients identified at the time of our potential launch in the beginning of 2027. Importantly, that we continue on with that momentum because that ultimately is going to be what drives the shape of the adoption curve. Cristin, from a medical standpoint, if I could just comment. Hypochondroplasia, children are not born with the same growth deficit that the achondroplasia patients may be born with. As a result, they often are diagnosed a bit later. They're referred and diagnosed. That data point that we referred to earlier, which was one of our goals to try to shrink that number in terms of age of diagnosis, is something that we see as a real positive sign that the work that we're doing to get these children in the hands of the right physicians, who again, can know what to do for them, that seems like a real early success, and we're looking forward to continuing to help shape that community and make these therapies should they become available actually in the hands of the physicians who treat the patients. Thanks, Greg Freiberg and Cristin Hubbard. I'll take that revenue timing or fluctuation question. I appreciate you pointing that out because it is important to emphasize that the disconnect between that underlying patient demand growth and reported revenues is entirely order timing. There's a couple of different layers to that. one, both of which we discussed last quarter, by the way. one was, you know, large international orders that were processed in Q4 that just didn't recur in Q1. The two was something that was somewhat unique to our business when looking back over the years, which was a modest amount of U.S. stocking impact from Q4 to Q1. This affected both Palynziq and Voxzogo. It wasn't significant, just, you know, about an extra week of inventory, but it was enough to show up as a variance in the quarter-over-quarter revenue. This is why we wanted to emphasize the underlying patient demand growth. There's no significant price drivers in there, by the way. It is entirely order timing dynamics. We'll move next to Phil Nadeau at TD Cowen. Good afternoon. Thanks for taking our questions.two from us. First, could you give an update on the ITC hearing? In particular, we're curious to get your thoughts on the ITC pretrial brief that was recently posted online. That's first. Second, in the press release, there's a note that there's gonna be some data from BMN 333's phase II, III trial in 2027. Could you provide a little bit more details around what data will be released at that time? Thanks. Thanks very much, Phil. It's Alexander. I'll take the first part of your question, then hand it over to Greg for the second part. With regard to the ITC, I mean, you know, overall, you know, we believe that pursuing an exclusion order at the ITC, enforcing our IP is the most expeditious way to protect our IP in the U.S. As you mentioned, we recently completed the ITC evidentiary hearing, and post-hearing briefs are now being submitted to the presiding chief administrative law judge. What happens next is we expect to receive a decision on whether Ascendis's product infringes our patent on or about August 21st. At that point, if the full commission decides to review the decision, the final decision is expected on or about December 21st. This is a little bit of, you know, where we are and what can we expect going forward. I will also add that, you know, on completion of the ITC process, we would expect to also enforce our patent in the federal district court, where, of course, monetary damages are available. I'll now hand it over to Greg. Thanks for the question, Phil Nadeau, about BMN 333. As a reminder, for those on the call, this is our long-acting CNP analog, again, designed to release continuous, potentially higher AUC exposures of CNP, when administered on a weekly basis. Again, as a reminder, in our phase I, we saw over 10x increases in the AUC levels that we were able to achieve safely in the healthy volunteers. We've recently initiated our phase II/III study in children with achondroplasia. This is a multi-regional clinical trial. It's currently open in a variety of countries around the world. We're enrolling as we speak. Our goal is to run the phase II portion where one of three doses of BMN 333 will be administered to the children. There'll be one arm that has Voxzogo as well. There's no placebo on this study. What we will ultimately report out in 2027 is that we will be able to measure the annualized growth velocity at the six month time point, and we'll be using that data along with other measures, anthropomorphic measures, other measures of safety and well-being, to make a decision based on a Bayesian analysis of which dose we should bring forward into a phase III to run head-to-head against Voxzogo. Again, looking for a superiority profile when it comes to AGV, with the presumption that more AGV will drive more in the measurements of health and wellness that we're all familiar with. We'll go next to Chris Raymond at Raymond James. Thanks. Just a question on the pivotal trial for BMN 333. I know this has come up before, but I guess I wanna just ask maybe in a more pointed way on the decision to go with a superiority trial versus non-inferiority. I think I've heard what you guys have said around BMN 333 providing 2 to 3x free CNP and that should translate into higher efficacy. Maybe just strategically, if a non-inferiority trial could show that data, why take the risk and run a superiority study? Thanks. Thanks for the question. This is Greg Freiberg. You know, our goal with BMN 333 is to evolve this space, not just make a more convenient version of Voxzogo. I'll just highlight that that 3x target was an at least 3x. We're actually testing a 3x, a 5x, and greater than 7x AUC exposure. From that standpoint, we believe strongly that BMN 333 is the right reagent to test this hypothesis. I'll also add that, you know, from a non-inferiority standpoint, it's a natural question to ask, wouldn't this be easier? From a pure mathematical standpoint, it's actually much harder, and the study would be upwards of 10x the size in order to show non-inferiority versus, you know, a drug like Voxzogo. From that standpoint, there's a practicality of designing the study, but I do want to reassure you that we will have an opportunity to look at the data after our phase II portion. If there need to be adjustments, if the Bayesian model gives us an update that tells us we should be reevaluating, those would be potential opportunities. That being said, we are very clear with what we want out of this molecule. We want a superior CNP product that can be the cornerstone for future therapies for achondroplasia. I would just add, absolutely, as Greg Freiberg said, this is Alexander Hardy. You know, we think the opportunity and the need here is really around superiority, in terms of efficacy, AGV, and all the benefits beyond linear growth. But also, you know, by this design of a study, we actually have an active control with Voxzogo. The size of the study is smaller than it would be if it was a non-inferiority design, as Greg Freiberg has already covered. We also think the proposition to both caregivers and physicians means that, you know, this should be a study that's very attractive to potential patients to sign up to be included, and therefore, you know, speed of recruitment, which is extremely important. We think that superiority profile we're aiming for from an efficacy standpoint, together with rapid trial recruitment and milestones being achieved is really a compelling proposition. Yeah, placebo-controlled studies, when there are active, safe and effective therapies, are no longer really, you know, possible to run. It's not the right thing to do either. We'll take our next question from Mohit Bansal at Wells Fargo. Great. This is Chen Xu, sound for Mohit Bansal. Thank you for taking our question. I just wanna double-click on the BMN 333 a little bit. I think you guys discussed that BMN 333 targeting, you know, improvement in achondroplasia. Just wondering with a competitor that is also advancing their weekly CNP analog together with the growth hormone and potentially reporting a higher AGV. Just how should we think about BioMarin's view of this, you know, evolving treatment landscape and the role that 333 could potentially play in over time? Thank you. Thanks for the question. This is Greg Freiberg again. It is true, I think the data in combination with growth hormone, of which we've seen about 12 months of data, does show in, you know, in the early studies that there is potentially additional growth to be had by adding in a second agent. I want to caution you that it is early days for the combination. Growth hormone has been around for quite a while, in achondroplasia, it's only approved in one market that I'm aware of, that's Japan. The reason for that is that, growth that can be stimulated with growth hormone, ultimately has not historically resulted in increases in final adult height. It's growth that gets uncorked, but at the expense of potentially closing the growth plates earlier. That remains an open question. I think we do need to see data two, three years or more, not only to see the safety, of course. Growth hormone, while it has a manageable safety profile, does have its own set of challenges that need to be monitored by a physician. We need to see whether or not those gains are long-standing. I want to reassure you that this is something that we're watching very closely. If there are opportunities and levers that can help CNP do its job better, so to speak, we will evaluate those at the right time and place. We don't feel that that is the correct time right now, and we're certainly interested in seeing additional data before the paradigm shifts. This is consistent with what we've heard from many of our stakeholders as well. We'll move to our next question from Joseph Schwartz at Leerink Partners. Great. Thanks very much. I was wondering if we could get some more perspective on your guidance raise for the enzyme therapies business. We see it increased by $500 million, and we estimate that Amicus has generated around $450 million in revenue for Galafold and Pombiliti in the comparable 8 months last year. That implies low double-digit growth, around 11%, I think. I heard you reference a high double-digit percent growth, Brian. I'm just wondering if you can help us reconcile that difference. Thanks Thanks, Joe. Appreciate the question and your math there. I'd probably attribute the reconciliation difference that you're trying to get at with two elements. One, by doing a pro rata eight months of 2025, you know, there's, you know, some missing variables there and a lack of precision that we couldn't do a strict apples to apples comparison. Secondly, you know, on a full year basis, again, I pointed to that strong performance for the first four months of the year for both Galafold and Pombiliti and Opfolda. You know, again, not reported. This is internal management data. We're not disclosing it because it didn't go through the usual, you know, external reporting process like a regular quarter does. We thought it was important to add color to that full year. I'd say it's more important, and this goes to our own business as well when we think about some of these quarterly timing dynamics. I think it's most important to compare the full year annual cycle year-over-year, and that's where when we piece together the range that's implied today over that, you know, full year $634 million that Amicus reported for 2025, we have a range of high teens to low 20s. I'd encourage you to anchor to that rather than trying to calculate some intra-quarter math. We'll move next to Jason Gerberry at Bank of America. Hey, guys. Thanks for taking my questions. Alex, just to follow up on the ITC question, I'm trying to just get my head around, do you have any knowledge of the Ascendis ability to do manufacturing workaround? Just help us think through scenarios if you get a positive ruling, like could a decision be stayed pending any sort of appeals? Then just as a follow-up, just on Palm Op, I believe, you've inherited the asset basically launched into 15 country markets and I think the goal is to get it into 80 country markets. How should we think about the phasing of that now that you've got the asset either 2026 or 2027? Thanks. Thanks very much for the questions. I'm, unfortunately, I'm not going to give any further details of the ITC, the potential, you know, scenarios around it. I hope you can understand that we don't want to get into too many specifics, especially whilst the case is very much pending. I'll hand it over now to Cristin for the second part. Yeah. Thank you very much for the question around Pombiliti and Opfolda. You're absolutely correct in that there are currently we are reimbursed in 15 countries. What we've been doing, you know, before the transaction was closed, we've been doing significant and deep dive, you know, discovery sessions, really looking at the business in each market for both Galafold and Pombiliti and Opfolda, really understanding what the dynamics are therein. Importantly looking at, as you've mentioned, our 80-country footprint and saying, "Where would the potential and the opportunity be?" We're identifying opportunities for both Galafold as well as Pombiliti and Opfolda. Given that Pombiliti and Opfolda is much earlier in its launch trajectory, you can imagine that there'll be a larger number of countries to look at therein. It's important to note that we're not necessarily going to put it into our entire 80-country footprint, but importantly look at where we believe the potential opportunities are and then have a cadence to that that we'll share more of in the Q2 call. That concludes the Q&A session. I will now turn the conference back over to BioMarin CEO, Alexander Hardy, for closing remarks. Thank you, operator, and thank you all for joining us today. This quarter, as you can tell, marks a really important inflection point for BioMarin. With the recent close of the Amicus acquisition expanding our commercial re-reach, strengthening our 2026 revenue growth outlook to 20% and enhancing our ability to serve more patients globally. We're encouraged by the robust patient demand observed across our portfolio. In enzyme therapies, we anticipate that the momentum from the Palynziq launch in adolescence will continue to build. With Voxzogo, a consistent rise in new patient initiations, more than 20% year-over-year in Q1, especially among younger children, demonstrates confidence in its long-term safety and efficacy and highlights the importance of starting treatment as early as possible. With the integration of Amicus now well underway, several near-term catalysts ahead, pipeline readouts, we are focused on translating this momentum into accelerated growth, broader patient impact, meaningful value creation. We appreciate your continued support and look forward to updating you next quarter. Thank you. This concludes today's Conference Call. Thank you for your participation. You may now disconnect.

Speaker 9: At this time, I would like to turn the conference over to Traci McCarty, Head of Investor Relations. Please go ahead. At this time, I would like to turn the conference over to Traci McCarty, Head of Investor Relations. at this time i would like to turn the conference over to traci mccarty head of investor relations Please go ahead. please go ahead

Speaker 12: Thank you, operator. To remind you, this non-confidential presentation contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including expectations regarding BioMarin's financial performance, commercial products, and potential future products in different areas of therapeutic research and development. Results may differ materially depending on the progress of BioMarin's product programs, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical market, and developments by competitors, and those factors detailed in BioMarin's filings with the Securities and Exchange Commission such as 10-Q, 10-K, and 8-K reports. In addition, we will use non-GAAP financial measures as defined in Regulation G during the call today. Thank you, operator. thank you operator To remind you, this non-confidential presentation contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including expectations regarding BioMarin's financial performance, commercial products, and potential future products in different areas of therapeutic research and development. to remind you this non-confidential presentation contains forward-looking statements about the business prospects of biomarin pharmaceutical inc including expectations regarding biomarin's financial performance commercial products and potential future products in different areas of therapeutic research and development Results may differ materially depending on the progress of BioMarin's product programs, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical market, and developments by competitors, and those factors detailed in BioMarin's filings with the Securities and Exchange Commission such as 10-Q, 10-K, and 8-K reports. results may differ materially depending on the progress of biomarin's product programs actions of regulatory authorities availability of capital future actions in the pharmaceutical market and developments by competitors and those factors detailed in biomarin's filings with the securities and exchange commission such as 10-q 10-k and 8-k reports In addition, we will use non-GAAP financial measures as defined in Regulation G during the call today. in addition we will use non-gaap financial measures as defined in regulation g during the call today These non-GAAP measures should not be considered in isolation from, as substitutes for, or superior to financial measures prepared in accordance with U.S. GAAP, and you can find the related reconciliations to U.S. GAAP in the earnings release and earnings presentation, both of which are now available in the investor relations section of our website. Please note that our commentary on today's call will focus on non-GAAP financial measures, unless otherwise indicated. Beginning on slide three in introducing BioMarin's management team, joining today's call are Alexander Hardy, Chief Executive Officer, Brian Mueller, Chief Financial Officer, Cristin Hubbard, Chief Commercial Officer, and Greg Freiberg, Chief R&D Officer. I will now turn the call over to BioMarin's President and CEO, Alexander Hardy. These non-GAAP measures should not be considered in isolation from, as substitutes for, or superior to financial measures prepared in accordance with U.S. these non-gaap measures should not be considered in isolation from as substitutes for or superior to financial measures prepared in accordance with u.s GAAP, and you can find the related reconciliations to U.S. gaap and you can find the related reconciliations to u.s GAAP in the earnings release and earnings presentation, both of which are now available in the investor relations section of our website. gaap in the earnings release and earnings presentation both of which are now available in the investor relations section of our website Please note that our commentary on today's call will focus on non-GAAP financial measures, unless otherwise indicated. please note that our commentary on today's call will focus on non-gaap financial measures unless otherwise indicated Beginning on slide three in introducing BioMarin's management team, joining today's call are Alexander Hardy, Chief Executive Officer, Brian Mueller, Chief Financial Officer, Cristin Hubbard, Chief Commercial Officer, and Greg Freiberg, Chief R&D Officer. beginning on slide three in introducing biomarin's management team joining today's call are alexander hardy chief executive officer brian mueller chief financial officer cristin hubbard chief commercial officer and greg freiberg chief r&d officer I will now turn the call over to BioMarin's President and CEO, Alexander Hardy. i will now turn the call over to biomarin's president and ceo alexander hardy

Speaker 1: Thank you, Traci, and thank you all for joining us today. I am so pleased that we completed the Amicus acquisition last week, starting a new and exciting chapter for BioMarin with the addition of two innovative therapies, Galafold for Fabry disease and Pombiliti and Opfolda for Pompe disease. The acquisition accelerates our anticipated year-over-year 2026 revenue growth to 20% at the midpoint of today's updated guidance. This strengthening trajectory is just the beginning of BioMarin's enhanced longer-term financial outlook, supported by our larger, more diversified commercial portfolio. Since announcing our plans to acquire Amicus late last year, we have been preparing for rapid integration beginning on day one, with the goal of increasing the peak potential of these newly added products. Thank you, Traci, and thank you all for joining us today. thank you traci and thank you all for joining us today I am so pleased that we completed the Amicus acquisition last week, starting a new and exciting chapter for BioMarin with the addition of two innovative therapies, Galafold for Fabry disease and Pombiliti and Opfolda for Pompe disease. i am so pleased that we completed the amicus acquisition last week starting a new and exciting chapter for biomarin with the addition of two innovative therapies galafold for fabry disease and pombiliti and opfolda for pompe disease The acquisition accelerates our anticipated year-over-year 2026 revenue growth to 20% at the midpoint of today's updated guidance. the acquisition accelerates our anticipated year-over-year 2026 revenue growth to 20% at the midpoint of today's updated guidance This strengthening trajectory is just the beginning of BioMarin's enhanced longer-term financial outlook, supported by our larger, more diversified commercial portfolio. this strengthening trajectory is just the beginning of biomarin's enhanced longer-term financial outlook supported by our larger more diversified commercial portfolio Since announcing our plans to acquire Amicus late last year, we have been preparing for rapid integration beginning on day one, with the goal of increasing the peak potential of these newly added products. since announcing our plans to acquire amicus late last year we have been preparing for rapid integration beginning on day one with the goal of increasing the peak potential of these newly added products Following the recent close of the transaction, we initiated our targeted integration plan focused on leveraging BioMarin's operating scale and capabilities to drive diagnosis and treatment rates for patients with Fabry disease and late-onset Pompe disease. Next quarter, we plan to share this roadmap, as well as more detail on BioMarin's growth acceleration with the addition of Galafold and Pombiliti and Opfolda to our commercial portfolio and DMX-200 for FSGS in phase III to our late-stage pipeline. Turning briefly to first quarter results and outlook for the remainder of this year and starting with enzyme therapies, I am pleased with the strong interest we are seeing from the PKU community following the recent Palynziq adolescent label expansion in the U.S., which Cristin will discuss in more detail. Following the recent close of the transaction, we initiated our targeted integration plan focused on leveraging BioMarin's operating scale and capabilities to drive diagnosis and treatment rates for patients with Fabry disease and late-onset Pompe disease. following the recent close of the transaction we initiated our targeted integration plan focused on leveraging biomarin's operating scale and capabilities to drive diagnosis and treatment rates for patients with fabry disease and late-onset pompe disease Next quarter, we plan to share this roadmap, as well as more detail on BioMarin's growth acceleration with the addition of Galafold and Pombiliti and Opfolda to our commercial portfolio and DMX-200 for FSGS in phase III to our late-stage pipeline. next quarter we plan to share this roadmap as well as more detail on biomarin's growth acceleration with the addition of galafold and pombiliti and opfolda to our commercial portfolio and dmx-200 for fsgs in phase iii to our late-stage pipeline Turning briefly to first quarter results and outlook for the remainder of this year and starting with enzyme therapies, I am pleased with the strong interest we are seeing from the PKU community following the recent Palynziq adolescent label expansion in the U.S., which Cristin will discuss in more detail. turning briefly to first quarter results and outlook for the remainder of this year and starting with enzyme therapies i am pleased with the strong interest we are seeing from the pku community following the recent palynziq adolescent label expansion in the u.s which cristin will discuss in more detail We expect enzyme therapies will deliver robust growth in 2026, further supported by the addition of Galafold and Pombiliti and Opfolda to the portfolio. Moving to our skeletal conditions business unit, we saw strong patient demand for Voxzogo, with new patient starts increasing across all regions in the 1st quarter. In the U.S., the majority of new patient starts were from the under age two cohort. These results reflect our focused investments in increasing adoption of Voxzogo, particularly among younger patients. Building on our leadership in achondroplasia, we are pleased to have submitted the sNDA for approval of Voxzogo. We expect to hear the timing of our review in the coming months. I want to congratulate our regulatory team for the outstanding work that went into this comprehensive submission package. We expect enzyme therapies will deliver robust growth in 2026, further supported by the addition of Galafold and Pombiliti and Opfolda to the portfolio. we expect enzyme therapies will deliver robust growth in 2026 further supported by the addition of galafold and pombiliti and opfolda to the portfolio Moving to our skeletal conditions business unit, we saw strong patient demand for Voxzogo, with new patient starts increasing across all regions in the 1st quarter. moving to our skeletal conditions business unit we saw strong patient demand for voxzogo with new patient starts increasing across all regions in the 1st quarter In the U.S., the majority of new patient starts were from the under age two cohort. in the u.s the majority of new patient starts were from the under age two cohort These results reflect our focused investments in increasing adoption of Voxzogo, particularly among younger patients. these results reflect our focused investments in increasing adoption of voxzogo particularly among younger patients Building on our leadership in achondroplasia, we are pleased to have submitted the sNDA for approval of Voxzogo. building on our leadership in achondroplasia we are pleased to have submitted the snda for approval of voxzogo We expect to hear the timing of our review in the coming months. we expect to hear the timing of our review in the coming months I want to congratulate our regulatory team for the outstanding work that went into this comprehensive submission package. i want to congratulate our regulatory team for the outstanding work that went into this comprehensive submission package We also look forward to pivotal results for Voxzogo in hypochondroplasia and BMN 401 for ENPP1 deficiency both later in Q2. In summary, we expect 2026 to be a momentous year for BioMarin. Our immediate focus remains on the seamless and rapid integration of Amicus to accelerate our growth trajectory this year and beyond and pursuing regulatory next steps following the two upcoming pivotal data readouts. The addition of Galafold and Pombiliti and Opfolda to BioMarin's portfolio of innovative medicines provides an opportunity to reach more patients around the world, creating significant value for all of our stakeholders in the near and longer term. As we enter this next chapter, I would like to express my appreciation to the employees of both BioMarin and Amicus, whose dedication forms the foundation of our shared mission to serve patients. We also look forward to pivotal results for Voxzogo in hypochondroplasia and BMN 401 for ENPP1 deficiency both later in Q2. we also look forward to pivotal results for voxzogo in hypochondroplasia and bmn 401 for enpp1 deficiency both later in q2 In summary, we expect 2026 to be a momentous year for BioMarin. in summary we expect 2026 to be a momentous year for biomarin Our immediate focus remains on the seamless and rapid integration of Amicus to accelerate our growth trajectory this year and beyond and pursuing regulatory next steps following the two upcoming pivotal data readouts. our immediate focus remains on the seamless and rapid integration of amicus to accelerate our growth trajectory this year and beyond and pursuing regulatory next steps following the two upcoming pivotal data readouts The addition of Galafold and Pombiliti and Opfolda to BioMarin's portfolio of innovative medicines provides an opportunity to reach more patients around the world, creating significant value for all of our stakeholders in the near and longer term. the addition of galafold and pombiliti and opfolda to biomarin's portfolio of innovative medicines provides an opportunity to reach more patients around the world creating significant value for all of our stakeholders in the near and longer term As we enter this next chapter, I would like to express my appreciation to the employees of both BioMarin and Amicus, whose dedication forms the foundation of our shared mission to serve patients. as we enter this next chapter i would like to express my appreciation to the employees of both biomarin and amicus whose dedication forms the foundation of our shared mission to serve patients Thank you for your attention. I will now turn the call over to Brian to provide additional financial updates. Brian? Thank you for your attention. thank you for your attention I will now turn the call over to Brian to provide additional financial updates. i will now turn the call over to brian to provide additional financial updates Brian? brian

Speaker 2: Thank you, Alexander. Please refer to today's press release for detailed first quarter 2026 results, including reconciliations of GAAP to non-GAAP financial measures. All first quarter results will be available in our upcoming Form 10-Q, which we expect to file in the coming days. Thank you, Alexander. thank you alexander Please refer to today's press release for detailed first quarter 2026 results, including reconciliations of GAAP to non-GAAP financial measures. please refer to today's press release for detailed first quarter 2026 results including reconciliations of gaap to non-gaap financial measures All first quarter results will be available in our upcoming Form 10-Q, which we expect to file in the coming days. all first quarter results will be available in our upcoming form 10-q which we expect to file in the coming days Moving to slide seven. Total revenues in the first quarter were $766 million and increased year-over-year, supported by increased patient demand across both enzyme therapies and Voxzogo. As expected, those organic growth drivers were partly offset by order timing dynamics as well as lower revenue from Roctavian, Kuvan, and royalties. Enzyme therapies revenue increased 6% year-over-year, led by growth in Vimizim, Naglazyme, and Brineura. Palynziq's first quarter revenues were impacted by U.S. order timing, which resulted in elevated stocking levels in the fourth quarter of 2025, as discussed last quarter. We expect this stocking dynamic to normalize and anticipate year-over-year revenue growth for Palynziq in full year 2026 as growth in new patient starts in the under 18-year-old population gains momentum and patients continue to titrate to their maintenance dose. Moving to slide seven. moving to slide seven Total revenues in the first quarter were $766 million and increased year-over-year, supported by increased patient demand across both enzyme therapies and Voxzogo. total revenues in the first quarter were $766 million and increased year-over-year supported by increased patient demand across both enzyme therapies and voxzogo As expected, those organic growth drivers were partly offset by order timing dynamics as well as lower revenue from Roctavian, Kuvan, and royalties. as expected those organic growth drivers were partly offset by order timing dynamics as well as lower revenue from roctavian kuvan and royalties Enzyme therapies revenue increased 6% year-over-year, led by growth in Vimizim, Naglazyme, and Brineura. enzyme therapies revenue increased 6% year-over-year led by growth in vimizim naglazyme and brineura Palynziq's first quarter revenues were impacted by U.S. order timing, which resulted in elevated stocking levels in the fourth quarter of 2025, as discussed last quarter. palynziq's first quarter revenues were impacted by u.s order timing which resulted in elevated stocking levels in the fourth quarter of 2025 as discussed last quarter We expect this stocking dynamic to normalize and anticipate year-over-year revenue growth for Palynziq in full year 2026 as growth in new patient starts in the under 18-year-old population gains momentum and patients continue to titrate to their maintenance dose. we expect this stocking dynamic to normalize and anticipate year-over-year revenue growth for palynziq in full year 2026 as growth in new patient starts in the under 18-year-old population gains momentum and patients continue to titrate to their maintenance dose Voxzogo revenue was supported by new patient starts across all regions and in line with the expectations that we shared on our prior quarter earnings call. Looking ahead, due to anticipated order timing and consistent with 2025, we expect Voxzogo revenue to be higher in the second half of 2026 compared to the first half. Cristin will provide more color on commercial dynamics in a moment. Turning now to slide 8. Cost of sales increased year-over-year in the first quarter, primarily due to a $31 million charge associated with an unsuccessful process qualification campaign to extend Naglazyme manufacturing capabilities. Importantly, this did not impact commercial supply. While this event decreased margins and earnings per share in the first quarter, we expect this charge to be offset in full year 2026 non-GAAP diluted earnings per share guidance. Voxzogo revenue was supported by new patient starts across all regions and in line with the expectations that we shared on our prior quarter earnings call. voxzogo revenue was supported by new patient starts across all regions and in line with the expectations that we shared on our prior quarter earnings call Looking ahead, due to anticipated order timing and consistent with 2025, we expect Voxzogo revenue to be higher in the second half of 2026 compared to the first half. looking ahead due to anticipated order timing and consistent with 2025 we expect voxzogo revenue to be higher in the second half of 2026 compared to the first half Cristin will provide more color on commercial dynamics in a moment. cristin will provide more color on commercial dynamics in a moment Turning now to slide 8. turning now to slide 8 Cost of sales increased year-over-year in the first quarter, primarily due to a $31 million charge associated with an unsuccessful process qualification campaign to extend Naglazyme manufacturing capabilities. cost of sales increased year-over-year in the first quarter primarily due to a $31 million charge associated with an unsuccessful process qualification campaign to extend naglazyme manufacturing capabilities Importantly, this did not impact commercial supply. importantly this did not impact commercial supply While this event decreased margins and earnings per share in the first quarter, we expect this charge to be offset in full year 2026 non-GAAP diluted earnings per share guidance. while this event decreased margins and earnings per share in the first quarter we expect this charge to be offset in full year 2026 non-gaap diluted earnings per share guidance Q1 non-GAAP R&D expense increased year-over-year, primarily due to spend to support BMN 401, our phase III clinical program acquired in the enzyme transaction in the second half of 2025. Development activities for Voxzogo for hypochondroplasia, BMN 333 for achondroplasia, and BMN 351 for Duchenne muscular dystrophy also contributed to higher year-over-year R&D expense. Non-GAAP SG&A expense also increased, partially driven by investments to support commercial expansion across enzyme therapies and Voxzogo, and partially driven by pre-close costs associated with the Amicus acquisition. First quarter non-GAAP diluted earnings per share was $0.76 and was significantly impacted by the drivers of increased operating expense that I just mentioned, as well as the impact of Q1 revenue, which we believe will be our lowest quarter of the year. Q1 non-GAAP R&D expense increased year-over-year, primarily due to spend to support BMN 401, our phase III clinical program acquired in the enzyme transaction in the second half of 2025. q1 non-gaap r&d expense increased year-over-year primarily due to spend to support bmn 401 our phase iii clinical program acquired in the enzyme transaction in the second half of 2025 Development activities for Voxzogo for hypochondroplasia, BMN 333 for achondroplasia, and BMN 351 for Duchenne muscular dystrophy also contributed to higher year-over-year R&D expense. development activities for voxzogo for hypochondroplasia bmn 333 for achondroplasia and bmn 351 for duchenne muscular dystrophy also contributed to higher year-over-year r&d expense Non-GAAP SG&A expense also increased, partially driven by investments to support commercial expansion across enzyme therapies and Voxzogo, and partially driven by pre-close costs associated with the Amicus acquisition. non-gaap sg&a expense also increased partially driven by investments to support commercial expansion across enzyme therapies and voxzogo and partially driven by pre-close costs associated with the amicus acquisition First quarter non-GAAP diluted earnings per share was $0.76 and was significantly impacted by the drivers of increased operating expense that I just mentioned, as well as the impact of Q1 revenue, which we believe will be our lowest quarter of the year. first quarter non-gaap diluted earnings per share was $0.76 and was significantly impacted by the drivers of increased operating expense that i just mentioned as well as the impact of q1 revenue which we believe will be our lowest quarter of the year The impact of the cost of sales charge and pre-close costs associated with the Amicus acquisition resulted in a $0.20 earnings per share impact to our non-GAAP earnings per share result. Looking past those elements helps to measure BioMarin's underlying business performance, as well as how this Q1 result fits into our full-year earnings per share guidance, which remains unchanged for the historical BioMarin business before layering on the Amicus business. Moving to slide nine and our updated full-year 2026 guidance, which now includes the Amicus financial outlook starting last week. We are raising enzyme therapies revenue guidance to a range of $2.725 billion-$2.775 billion for the full-year 2026, inclusive of meaningful contributions from Galafold and Pombiliti and Opfolda, resulting in approximately 30% growth at the midpoint. The impact of the cost of sales charge and pre-close costs associated with the Amicus acquisition resulted in a $0.20 earnings per share impact to our non-GAAP earnings per share result. the impact of the cost of sales charge and pre-close costs associated with the amicus acquisition resulted in a $0.20 earnings per share impact to our non-gaap earnings per share result Looking past those elements helps to measure BioMarin's underlying business performance, as well as how this Q1 result fits into our full-year earnings per share guidance, which remains unchanged for the historical BioMarin business before layering on the Amicus business. looking past those elements helps to measure biomarin's underlying business performance as well as how this q1 result fits into our full-year earnings per share guidance which remains unchanged for the historical biomarin business before layering on the amicus business Moving to slide nine and our updated full-year 2026 guidance, which now includes the Amicus financial outlook starting last week. moving to slide nine and our updated full-year 2026 guidance which now includes the amicus financial outlook starting last week We are raising enzyme therapies revenue guidance to a range of $2.725 billion-$2.775 billion for the full-year 2026, inclusive of meaningful contributions from Galafold and Pombiliti and Opfolda, resulting in approximately 30% growth at the midpoint. we are raising enzyme therapies revenue guidance to a range of $2.725 billion-$2.775 billion for the full-year 2026 inclusive of meaningful contributions from galafold and pombiliti and opfolda resulting in approximately 30% growth at the midpoint Adding these high-growth products to our enzyme therapies portfolio increases our full-year total revenue guidance to a range of $3.825 billion to $3.925 billion, with the midpoint representing approximately 20% year-over-year growth in 2026. For Voxzogo, we are maintaining our revenue guidance of $975 million to $1.025 billion, which continues to reflect high single-digit growth at the midpoint. On non-GAAP diluted earnings per share guidance, we are updating the guidance range to $4.85 to $5.05. As previously communicated, the acquisition of Amicus will be slightly diluted for the full year 2026. We continue to expect the acquisition to be accretive to non-GAAP diluted earnings per share in the first 12 months after close and substantially accretive beginning in 2027. Adding these high-growth products to our enzyme therapies portfolio increases our full-year total revenue guidance to a range of $3.825 billion to $3.925 billion, with the midpoint representing approximately 20% year-over-year growth in 2026. adding these high-growth products to our enzyme therapies portfolio increases our full-year total revenue guidance to a range of $3.825 billion to $3.925 billion with the midpoint representing approximately 20% year-over-year growth in 2026 For Voxzogo, we are maintaining our revenue guidance of $975 million to $1.025 billion, which continues to reflect high single-digit growth at the midpoint. for voxzogo we are maintaining our revenue guidance of $975 million to $1.025 billion which continues to reflect high single-digit growth at the midpoint On non-GAAP diluted earnings per share guidance, we are updating the guidance range to $4.85 to $5.05. on non-gaap diluted earnings per share guidance we are updating the guidance range to $4.85 to $5.05 As previously communicated, the acquisition of Amicus will be slightly diluted for the full year 2026. as previously communicated the acquisition of amicus will be slightly diluted for the full year 2026 We continue to expect the acquisition to be accretive to non-GAAP diluted earnings per share in the first 12 months after close and substantially accretive beginning in 2027. we continue to expect the acquisition to be accretive to non-gaap diluted earnings per share in the first 12 months after close and substantially accretive beginning in 2027 The Amicus P&L will be incorporated into BioMarin's financial results as of last week's closing of the transaction. In 2026, we expect to include the base Amicus operating expenses less initial cost synergies anticipated in 2026. As Alexander touched on, now that the transaction is closed, we have engaged more deeply with the Amicus business and plan to share our outlook on both commercial revenues and cost synergies on our next quarterly earnings call. To give some perspective on timing of the updated guidance. We expect order timing for the historical BioMarin products and 2 full quarters of Galafold and Pombiliti and Opfolda revenues in the second half of the year to drive significantly higher revenues as compared to the first half of 2026. To provide context, we expect more than 55% of total 2026 revenues to be recognized in the second half of the year. The Amicus P&L will be incorporated into BioMarin's financial results as of last week's closing of the transaction. the amicus p&l will be incorporated into biomarin's financial results as of last week's closing of the transaction In 2026, we expect to include the base Amicus operating expenses less initial cost synergies anticipated in 2026. in 2026 we expect to include the base amicus operating expenses less initial cost synergies anticipated in 2026 As Alexander touched on, now that the transaction is closed, we have engaged more deeply with the Amicus business and plan to share our outlook on both commercial revenues and cost synergies on our next quarterly earnings call. as alexander touched on now that the transaction is closed we have engaged more deeply with the amicus business and plan to share our outlook on both commercial revenues and cost synergies on our next quarterly earnings call To give some perspective on timing of the updated guidance. We expect order timing for the historical BioMarin products and 2 full quarters of Galafold and Pombiliti and Opfolda revenues in the second half of the year to drive significantly higher revenues as compared to the first half of 2026. to give some perspective on timing of the updated guidance. we expect order timing for the historical biomarin products and 2 full quarters of galafold and pombiliti and opfolda revenues in the second half of the year to drive significantly higher revenues as compared to the first half of 2026 To provide context, we expect more than 55% of total 2026 revenues to be recognized in the second half of the year. to provide context we expect more than 55% of total 2026 revenues to be recognized in the second half of the year Likewise, on the expected timing of our profitability, we expect Q2 non-GAAP diluted earnings per share to be just modestly higher than Q1, partially due to pre-close Amicus costs incurred in April, plus a higher amount of the 2026 Amicus dilution being weighted to the second quarter. Further, the revenue timing weighted to the second half of the year results in most of our expected profitability occurring in Q3 and Q4. These earnings timing dynamics drive approximately two-thirds of our 2026 earnings per share expected in the second half of the year. Briefly, on evolving geopolitical uncertainties, we are watching the situation in the Middle East very closely and note that today's guidance reflects an allowance for a modest amount of disruption in that region in 2026. Likewise, on the expected timing of our profitability, we expect Q2 non-GAAP diluted earnings per share to be just modestly higher than Q1, partially due to pre-close Amicus costs incurred in April, plus a higher amount of the 2026 Amicus dilution being weighted to the second quarter. likewise on the expected timing of our profitability we expect q2 non-gaap diluted earnings per share to be just modestly higher than q1 partially due to pre-close amicus costs incurred in april plus a higher amount of the 2026 amicus dilution being weighted to the second quarter Further, the revenue timing weighted to the second half of the year results in most of our expected profitability occurring in Q3 and Q4. further the revenue timing weighted to the second half of the year results in most of our expected profitability occurring in q3 and q4 These earnings timing dynamics drive approximately two-thirds of our 2026 earnings per share expected in the second half of the year. these earnings timing dynamics drive approximately two-thirds of our 2026 earnings per share expected in the second half of the year Briefly, on evolving geopolitical uncertainties, we are watching the situation in the Middle East very closely and note that today's guidance reflects an allowance for a modest amount of disruption in that region in 2026. briefly on evolving geopolitical uncertainties we are watching the situation in the middle east very closely and note that today's guidance reflects an allowance for a modest amount of disruption in that region in 2026 Looking ahead, we look forward to sharing with you our expanded financial outlook, enhanced by the addition of Galafold and Pombiliti and Opfolda, setting the stage for accelerated near and midterm growth. Thank you for your attention. I will now turn it over to Cristin for a commercial update. Cristin? Looking ahead, we look forward to sharing with you our expanded financial outlook, enhanced by the addition of Galafold and Pombiliti and Opfolda, setting the stage for accelerated near and midterm growth. looking ahead we look forward to sharing with you our expanded financial outlook enhanced by the addition of galafold and pombiliti and opfolda setting the stage for accelerated near and midterm growth Thank you for your attention. thank you for your attention I will now turn it over to Cristin for a commercial update. i will now turn it over to cristin for a commercial update Cristin? cristin

Speaker 5: Thank you, Brian. We were encouraged by the commercial execution across our portfolio so far this year, with strong patient demand across enzyme therapies and Voxzogo. We look forward to providing a more detailed commercial update on our plans to maximize the potential of both Galafold and Pombiliti and Opfolda next quarter. Our initial priorities therein are focused on driving diagnosis in Fabry and switch in Pompe. This will support increased penetration in countries where Galafold and Pombiliti and Opfolda are marketed, while we can currently work on geographic expansion plans for both products. We look forward to updating you on our Q2 call. Now, moving to Slide 11. I'll begin with an update on first quarter 2026 performance, starting with enzyme therapies. As Brian outlined, enzyme therapies delivered 6% year-over-year growth in Q1, led by Vimizim, Naglazyme, and Brineura. Thank you, Brian. thank you brian We were encouraged by the commercial execution across our portfolio so far this year, with strong patient demand across enzyme therapies and Voxzogo. we were encouraged by the commercial execution across our portfolio so far this year with strong patient demand across enzyme therapies and voxzogo We look forward to providing a more detailed commercial update on our plans to maximize the potential of both Galafold and Pombiliti and Opfolda next quarter. we look forward to providing a more detailed commercial update on our plans to maximize the potential of both galafold and pombiliti and opfolda next quarter Our initial priorities therein are focused on driving diagnosis in Fabry and switch in Pompe. our initial priorities therein are focused on driving diagnosis in fabry and switch in pompe This will support increased penetration in countries where Galafold and Pombiliti and Opfolda are marketed, while we can currently work on geographic expansion plans for both products. this will support increased penetration in countries where galafold and pombiliti and opfolda are marketed while we can currently work on geographic expansion plans for both products We look forward to updating you on our Q2 call. we look forward to updating you on our q2 call Now, moving to Slide 11. now moving to slide 11 I'll begin with an update on first quarter 2026 performance, starting with enzyme therapies. i'll begin with an update on first quarter 2026 performance starting with enzyme therapies As Brian outlined, enzyme therapies delivered 6% year-over-year growth in Q1, led by Vimizim, Naglazyme, and Brineura. as brian outlined enzyme therapies delivered 6% year-over-year growth in q1 led by vimizim naglazyme and brineura Across the enzyme therapies portfolio, we continue to see strong patient demand and adherence. Turning to Palynziq. In the first quarter, we continued to expand the underlying patient base, and physician engagement remained strong. Importantly, following the FDA approval of Palynziq's age label expansion to those 12 years and older in February, we have successfully launched in this age group and are seeing encouraging early momentum. We have observed broad interest and engagement from caregivers and healthcare providers treating adolescents during this critical stage of their development. Since approval, we have observed meaningful enrollments and new patient starts in people under the age of 18. From a prescribing standpoint, adolescent uptake is being driven by both physicians with significant experience prescribing Palynziq, as well as by clinicians who are newer to the therapy. Across the enzyme therapies portfolio, we continue to see strong patient demand and adherence. across the enzyme therapies portfolio we continue to see strong patient demand and adherence Turning to Palynziq. turning to palynziq In the first quarter, we continued to expand the underlying patient base, and physician engagement remained strong. in the first quarter we continued to expand the underlying patient base and physician engagement remained strong Importantly, following the FDA approval of Palynziq's age label expansion to those 12 years and older in February, we have successfully launched in this age group and are seeing encouraging early momentum. importantly following the fda approval of palynziq's age label expansion to those 12 years and older in february we have successfully launched in this age group and are seeing encouraging early momentum We have observed broad interest and engagement from caregivers and healthcare providers treating adolescents during this critical stage of their development. we have observed broad interest and engagement from caregivers and healthcare providers treating adolescents during this critical stage of their development Since approval, we have observed meaningful enrollments and new patient starts in people under the age of 18. since approval we have observed meaningful enrollments and new patient starts in people under the age of 18 From a prescribing standpoint, adolescent uptake is being driven by both physicians with significant experience prescribing Palynziq, as well as by clinicians who are newer to the therapy. from a prescribing standpoint adolescent uptake is being driven by both physicians with significant experience prescribing palynziq as well as by clinicians who are newer to the therapy Recall that it can take a patient many months to titrate up to their maintenance dose of Palynziq. We would expect to see the results of positive early prescribing over the coming quarters. Palynziq continues to stand alone in its ability to enable people with PKU to reach physiologic Phe levels while reducing dietary restrictions, regardless of severity. With the U.S. adolescent launch underway and European approval expected later this year, we are excited about the impact Palynziq can have for additional families over time. Turning now to Voxzogo on Slide 12. As discussed, first quarter results reflected expected order timing dynamics following a strong Q-four, particularly in international markets. Importantly, we observed strong growth in patient additions globally, with the number of children being treated with Voxzogo increasing by more than 20% year-over-year. Recall that it can take a patient many months to titrate up to their maintenance dose of Palynziq. recall that it can take a patient many months to titrate up to their maintenance dose of palynziq We would expect to see the results of positive early prescribing over the coming quarters. we would expect to see the results of positive early prescribing over the coming quarters Palynziq continues to stand alone in its ability to enable people with PKU to reach physiologic Phe levels while reducing dietary restrictions, regardless of severity. palynziq continues to stand alone in its ability to enable people with pku to reach physiologic phe levels while reducing dietary restrictions regardless of severity With the U.S. adolescent launch underway and European approval expected later this year, we are excited about the impact Palynziq can have for additional families over time. with the u.s adolescent launch underway and european approval expected later this year we are excited about the impact palynziq can have for additional families over time Turning now to Voxzogo on Slide 12. turning now to voxzogo on slide 12 As discussed, first quarter results reflected expected order timing dynamics following a strong Q-four, particularly in international markets. as discussed first quarter results reflected expected order timing dynamics following a strong q-four particularly in international markets Importantly, we observed strong growth in patient additions globally, with the number of children being treated with Voxzogo increasing by more than 20% year-over-year. importantly we observed strong growth in patient additions globally with the number of children being treated with voxzogo increasing by more than 20% year-over-year With the competitor having recently entered the U.S. market, our focus has remained on executing our strategy. We continue to see strong momentum. During the first quarter, Voxzogo progress continued, supported by ongoing patient additions across all regions and ages, strong adherence and persistence globally, and continued expansion of the prescriber base. Our teams are focused on driving new patient starts across all ages, with an emphasis on children under two years of age, where international consensus guidelines for achondroplasia recommend early diagnosis and treatment with Voxzogo as soon as possible. In the United States, we are encouraged to see that our efforts educating and engaging with caregivers and HCPs are having an impact in the under two year-old age segment. In Q1, over half of new patient starts were from children under age two, a greater proportion compared to last quarter. With the competitor having recently entered the U.S. market, our focus has remained on executing our strategy. with the competitor having recently entered the u.s market our focus has remained on executing our strategy We continue to see strong momentum. we continue to see strong momentum During the first quarter, Voxzogo progress continued, supported by ongoing patient additions across all regions and ages, strong adherence and persistence globally, and continued expansion of the prescriber base. during the first quarter voxzogo progress continued supported by ongoing patient additions across all regions and ages strong adherence and persistence globally and continued expansion of the prescriber base Our teams are focused on driving new patient starts across all ages, with an emphasis on children under two years of age, where international consensus guidelines for achondroplasia recommend early diagnosis and treatment with Voxzogo as soon as possible. our teams are focused on driving new patient starts across all ages with an emphasis on children under two years of age where international consensus guidelines for achondroplasia recommend early diagnosis and treatment with voxzogo as soon as possible In the United States, we are encouraged to see that our efforts educating and engaging with caregivers and HCPs are having an impact in the under two year-old age segment. in the united states we are encouraged to see that our efforts educating and engaging with caregivers and hcps are having an impact in the under two year-old age segment In Q1, over half of new patient starts were from children under age two, a greater proportion compared to last quarter. in q1 over half of new patient starts were from children under age two a greater proportion compared to last quarter Additionally, we have seen an approximate 10% decrease in the average age of children initiating Voxzogo treatment in the under two segment, narrowing the window between diagnosis and treatment starts. Internationally, building on our global expansion into 55 countries to date, our strategy remains focused on reaching more patients in regions that have further opportunity and treating infants in countries that already have high penetration rates. With our sNDA for full approval now submitted, we believe the depth and durability of Voxzogo's clinical evidence can be further reinforced, strengthening its role in treatment decisions for infants and children with achondroplasia. At the same time, we're making progress in preparations for potential expansion of Voxzogo into hypochondroplasia. As we get closer to the phase III data and potential launch, our pre-commercialization activities continue to accelerate. Our initiatives in the U.S. are making a difference. Additionally, we have seen an approximate 10% decrease in the average age of children initiating Voxzogo treatment in the under two segment, narrowing the window between diagnosis and treatment starts. additionally we have seen an approximate 10% decrease in the average age of children initiating voxzogo treatment in the under two segment narrowing the window between diagnosis and treatment starts Internationally, building on our global expansion into 55 countries to date, our strategy remains focused on reaching more patients in regions that have further opportunity and treating infants in countries that already have high penetration rates. internationally building on our global expansion into 55 countries to date our strategy remains focused on reaching more patients in regions that have further opportunity and treating infants in countries that already have high penetration rates With our sNDA for full approval now submitted, we believe the depth and durability of Voxzogo's clinical evidence can be further reinforced, strengthening its role in treatment decisions for infants and children with achondroplasia. with our snda for full approval now submitted we believe the depth and durability of voxzogo's clinical evidence can be further reinforced strengthening its role in treatment decisions for infants and children with achondroplasia At the same time, we're making progress in preparations for potential expansion of Voxzogo into hypochondroplasia. As we get closer to the phase III data and potential launch, our pre-commercialization activities continue to accelerate. at the same time we're making progress in preparations for potential expansion of voxzogo into hypochondroplasia. as we get closer to the phase iii data and potential launch our pre-commercialization activities continue to accelerate Our initiatives in the U.S. are making a difference. our initiatives in the u.s are making a difference More people are being diagnosed at a younger age, diagnosis rates are rising, and more doctors are requesting diagnostic tests. We look forward to the phase III top-line results in the 2nd quarter of 2026, and submitting to global health authorities in the 2nd half of this year, with potential approval in 2027. In summary, we are pleased with the strong patient demand observed across the portfolio with continued momentum in our core business units and a compelling set of near-term opportunities to accelerate growth. With that, I'll turn it over to Greg for an R&D update. Greg? More people are being diagnosed at a younger age, diagnosis rates are rising, and more doctors are requesting diagnostic tests. more people are being diagnosed at a younger age diagnosis rates are rising and more doctors are requesting diagnostic tests We look forward to the phase III top-line results in the 2nd quarter of 2026, and submitting to global health authorities in the 2nd half of this year, with potential approval in 2027. we look forward to the phase iii top-line results in the 2nd quarter of 2026 and submitting to global health authorities in the 2nd half of this year with potential approval in 2027 In summary, we are pleased with the strong patient demand observed across the portfolio with continued momentum in our core business units and a compelling set of near-term opportunities to accelerate growth. in summary we are pleased with the strong patient demand observed across the portfolio with continued momentum in our core business units and a compelling set of near-term opportunities to accelerate growth With that, I'll turn it over to Greg for an R&D update. with that i'll turn it over to greg for an r&d update Greg? greg

Speaker 6: Thank you, Cristin. As expected, 2026 is shaping up to be an active year for R&D, and we're looking forward to delivering multiple meaningful milestones. Moving to slide 14, we've built a comprehensive Voxzogo evidence package that goes well beyond describing annualized growth velocity, highlighting long-term durability and clinically meaningful health outcomes for children with achondroplasia. Recently, at the Pediatric Endocrine Society annual meeting, we shared data from three ongoing long-term extension studies that illustrate these sustained benefits of Voxzogo over time. Consistent and cumulative improvements in arm span Z-scores were observed across all age groups over the six to eight years of follow-up, as depicted on the left of slide 14. On the right side, you again see consistent and cumulative gains in height and height Z-scores with durable results out to eight years of follow-up. Thank you, Cristin. thank you cristin As expected, 2026 is shaping up to be an active year for R&D, and we're looking forward to delivering multiple meaningful milestones. as expected 2026 is shaping up to be an active year for r&d and we're looking forward to delivering multiple meaningful milestones Moving to slide 14, we've built a comprehensive Voxzogo evidence package that goes well beyond describing annualized growth velocity, highlighting long-term durability and clinically meaningful health outcomes for children with achondroplasia. moving to slide 14 we've built a comprehensive voxzogo evidence package that goes well beyond describing annualized growth velocity highlighting long-term durability and clinically meaningful health outcomes for children with achondroplasia Recently, at the Pediatric Endocrine Society annual meeting, we shared data from three ongoing long-term extension studies that illustrate these sustained benefits of Voxzogo over time. recently at the pediatric endocrine society annual meeting we shared data from three ongoing long-term extension studies that illustrate these sustained benefits of voxzogo over time Consistent and cumulative improvements in arm span Z-scores were observed across all age groups over the six to eight years of follow-up, as depicted on the left of slide 14. consistent and cumulative improvements in arm span z-scores were observed across all age groups over the six to eight years of follow-up as depicted on the left of slide 14 On the right side, you again see consistent and cumulative gains in height and height Z-scores with durable results out to eight years of follow-up. on the right side you again see consistent and cumulative gains in height and height z-scores with durable results out to eight years of follow-up In addition to anatomic measures of benefit, we also presented data demonstrating Voxzogo's favorable impacts on quality-of-life measures. Importantly, these sustained efficacy findings are supported by a robust safety database now comprising more than 10,000 patient years of exposure, reinforcing Voxzogo's well-established long-term safety profile. Taken together, this extensive body of evidence reinforces Voxzogo's differentiated profile with no other achondroplasia therapy supported by this level of long-term data with regard to safety, efficacy, and functional outcomes. Importantly, this data spans the full pediatric age population, and Voxzogo remains the only therapy approved for use immediately from infancy. This allows for early treatment initiation and enables a long window to positively influence endochondral bone formation and all the potential downstream benefits to health outcomes. In addition to anatomic measures of benefit, we also presented data demonstrating Voxzogo's favorable impacts on quality-of-life measures. in addition to anatomic measures of benefit we also presented data demonstrating voxzogo's favorable impacts on quality-of-life measures Importantly, these sustained efficacy findings are supported by a robust safety database now comprising more than 10,000 patient years of exposure, reinforcing Voxzogo's well-established long-term safety profile. importantly these sustained efficacy findings are supported by a robust safety database now comprising more than 10,000 patient years of exposure reinforcing voxzogo's well-established long-term safety profile Taken together, this extensive body of evidence reinforces Voxzogo's differentiated profile with no other achondroplasia therapy supported by this level of long-term data with regard to safety, efficacy, and functional outcomes. taken together this extensive body of evidence reinforces voxzogo's differentiated profile with no other achondroplasia therapy supported by this level of long-term data with regard to safety efficacy and functional outcomes Importantly, this data spans the full pediatric age population, and Voxzogo remains the only therapy approved for use immediately from infancy. importantly this data spans the full pediatric age population and voxzogo remains the only therapy approved for use immediately from infancy This allows for early treatment initiation and enables a long window to positively influence endochondral bone formation and all the potential downstream benefits to health outcomes. this allows for early treatment initiation and enables a long window to positively influence endochondral bone formation and all the potential downstream benefits to health outcomes Together, these data formed a strong foundation for our full approval submission intended to fulfill our post-marketing requirement, which was submitted to the FDA in April and will afford us the opportunity to share direct evidence of Voxzogo's long-term value with the community via peer-reviewed publications and presentations later this year. Now moving to slide 15, the remainder of 2026 includes several anticipated pipeline updates, including two particularly important pivotal data readouts expected in the second quarter. Starting with hypochondroplasia, we believe the healthcare provider community is looking forward to a targeted therapy that addresses this skeletal condition, and we are confident in the scientific rationale for Voxzogo in this indication. That confidence is grounded in the strong proof of concept and durability demonstrated in Dr. Dauber's investigator-sponsored study and in the rapid enrollment we observed in our own phase III pivotal trial. Together, these data formed a strong foundation for our full approval submission intended to fulfill our post-marketing requirement, which was submitted to the FDA in April and will afford us the opportunity to share direct evidence of Voxzogo's long-term value with the community via peer-reviewed publications and presentations later this year. together these data formed a strong foundation for our full approval submission intended to fulfill our post-marketing requirement which was submitted to the fda in april and will afford us the opportunity to share direct evidence of voxzogo's long-term value with the community via peer-reviewed publications and presentations later this year Now moving to slide 15, the remainder of 2026 includes several anticipated pipeline updates, including two particularly important pivotal data readouts expected in the second quarter. now moving to slide 15 the remainder of 2026 includes several anticipated pipeline updates including two particularly important pivotal data readouts expected in the second quarter Starting with hypochondroplasia, we believe the healthcare provider community is looking forward to a targeted therapy that addresses this skeletal condition, and we are confident in the scientific rationale for Voxzogo in this indication. starting with hypochondroplasia we believe the healthcare provider community is looking forward to a targeted therapy that addresses this skeletal condition and we are confident in the scientific rationale for voxzogo in this indication That confidence is grounded in the strong proof of concept and durability demonstrated in Dr. Dauber's investigator-sponsored study and in the rapid enrollment we observed in our own phase III pivotal trial. that confidence is grounded in the strong proof of concept and durability demonstrated in dr dauber's investigator-sponsored study and in the rapid enrollment we observed in our own phase iii pivotal trial We look forward to sharing these phase III top-line results in the second quarter. Enrollment is progressing very well in our phase II study in children under the age of three, highlighting early interest in Voxzogo as a potential option from infancy in hypochondroplasia. We also expect phase III top-line data for BMN 401 in the second quarter of this year. As a reminder, ENPP1 deficiency is a rare, serious, and progressive genetic condition affecting vascular, skeletal, and soft tissue systems. BMN 401 has the potential to be the first disease-targeted therapy for ENPP1 deficiency. The ENERGY-3 study in children aged 1 to 12 includes two co-primary endpoints. The first is change from baseline and plasma measures of inorganic pyrophosphate through week 52. The second is change in the RGIC, or Radiographic Global Impression of Change, after 52 weeks of treatment, assessing improvement in skeletal health. We look forward to sharing these phase III top-line results in the second quarter. we look forward to sharing these phase iii top-line results in the second quarter Enrollment is progressing very well in our phase II study in children under the age of three, highlighting early interest in Voxzogo as a potential option from infancy in hypochondroplasia. enrollment is progressing very well in our phase ii study in children under the age of three highlighting early interest in voxzogo as a potential option from infancy in hypochondroplasia We also expect phase III top-line data for BMN 401 in the second quarter of this year. we also expect phase iii top-line data for bmn 401 in the second quarter of this year As a reminder, ENPP1 deficiency is a rare, serious, and progressive genetic condition affecting vascular, skeletal, and soft tissue systems. as a reminder enpp1 deficiency is a rare serious and progressive genetic condition affecting vascular skeletal and soft tissue systems BMN 401 has the potential to be the first disease-targeted therapy for ENPP1 deficiency. bmn 401 has the potential to be the first disease-targeted therapy for enpp1 deficiency The ENERGY-3 study in children aged 1 to 12 includes two co-primary endpoints. the energy-3 study in children aged 1 to 12 includes two co-primary endpoints The first is change from baseline and plasma measures of inorganic pyrophosphate through week 52. the first is change from baseline and plasma measures of inorganic pyrophosphate through week 52 The second is change in the RGIC, or Radiographic Global Impression of Change, after 52 weeks of treatment, assessing improvement in skeletal health. the second is change in the rgic or radiographic global impression of change after 52 weeks of treatment assessing improvement in skeletal health Clinical experience with BMN 401 in older patients has shown that normalization of pyrophosphate is accompanied by improvements in bone mineral biomarkers, functional performance, and patient and physician-reported outcomes. In addition to these pivotal readouts, I'd also like to highlight progress with BMN 333, our long-acting CNP therapy. We are pleased to share that enrollment is underway in our global registrational enabling phase II/III study. We are rapidly progressing this program with the goal of establishing BMN 333 as a potential next-generation standard of care for achondroplasia and potentially for other skeletal conditions. Before closing, I'd also like to touch on BMN 351 for Duchenne muscular dystrophy. At the Muscular Dystrophy Association Congress in March, we presented initial data demonstrating dose-dependent increases in dystrophin at week 25 in both the 6 and 9 milligram per kilogram dose cohorts. Clinical experience with BMN 401 in older patients has shown that normalization of pyrophosphate is accompanied by improvements in bone mineral biomarkers, functional performance, and patient and physician-reported outcomes. clinical experience with bmn 401 in older patients has shown that normalization of pyrophosphate is accompanied by improvements in bone mineral biomarkers functional performance and patient and physician-reported outcomes In addition to these pivotal readouts, I'd also like to highlight progress with BMN 333, our long-acting CNP therapy. in addition to these pivotal readouts i'd also like to highlight progress with bmn 333 our long-acting cnp therapy We are pleased to share that enrollment is underway in our global registrational enabling phase II/III study. we are pleased to share that enrollment is underway in our global registrational enabling phase ii/iii study We are rapidly progressing this program with the goal of establishing BMN 333 as a potential next-generation standard of care for achondroplasia and potentially for other skeletal conditions. we are rapidly progressing this program with the goal of establishing bmn 333 as a potential next-generation standard of care for achondroplasia and potentially for other skeletal conditions Before closing, I'd also like to touch on BMN 351 for Duchenne muscular dystrophy. before closing i'd also like to touch on bmn 351 for duchenne muscular dystrophy At the Muscular Dystrophy Association Congress in March, we presented initial data demonstrating dose-dependent increases in dystrophin at week 25 in both the 6 and 9 milligram per kilogram dose cohorts. at the muscular dystrophy association congress in march we presented initial data demonstrating dose-dependent increases in dystrophin at week 25 in both the 6 and 9 milligram per kilogram dose cohorts These findings were accompanied by notable decreases in creatine kinase, a biomarker of muscle injury, and the prevention of functional decline as measured by the North Star Ambulatory Assessment and the six-minute walk test when compared to historically matched controls. We are encouraged by both the dystrophin expression levels and the functional improvements observed in our development program. Enrollment in the 12 milligram per kilogram cohort is ongoing, and we look forward to providing an additional update by year-end as this program continues to advance. Finally, we would like to thank the patients, families, and caregivers whose commitment continues to make this progress possible. Thank you for your attention today. We will now open the call to your questions. Operator? These findings were accompanied by notable decreases in creatine kinase, a biomarker of muscle injury, and the prevention of functional decline as measured by the North Star Ambulatory Assessment and the six-minute walk test when compared to historically matched controls. these findings were accompanied by notable decreases in creatine kinase a biomarker of muscle injury and the prevention of functional decline as measured by the north star ambulatory assessment and the six-minute walk test when compared to historically matched controls We are encouraged by both the dystrophin expression levels and the functional improvements observed in our development program. Enrollment in the 12 milligram per kilogram cohort is ongoing, and we look forward to providing an additional update by year-end as this program continues to advance. we are encouraged by both the dystrophin expression levels and the functional improvements observed in our development program. enrollment in the 12 milligram per kilogram cohort is ongoing and we look forward to providing an additional update by year-end as this program continues to advance Finally, we would like to thank the patients, families, and caregivers whose commitment continues to make this progress possible. finally we would like to thank the patients families and caregivers whose commitment continues to make this progress possible Thank you for your attention today. thank you for your attention today We will now open the call to your questions. we will now open the call to your questions Operator? operator

Speaker 9: Thank you. We will now begin the question and answer session. If you have dialed in and would like to ask a question, please press star one on your telephone keypad to raise your hand and join the queue. If you would like to withdraw your question, simply Press star one again. We'll go first to Sean Layman at Morgan Stanley. Thank you. thank you We will now begin the question and answer session. we will now begin the question and answer session If you have dialed in and would like to ask a question, please press star one on your telephone keypad to raise your hand and join the queue. if you have dialed in and would like to ask a question please press star one on your telephone keypad to raise your hand and join the queue If you would like to withdraw your question, simply Press star one again. if you would like to withdraw your question simply press star one again We'll go first to Sean Layman at Morgan Stanley. we'll go first to sean layman at morgan stanley

Speaker 17: Hi, this is Mike on for Sean. Thank you for taking our questions. I guess wanted to touch on two things. First, looking at the recent data at the Pediatric Endocrine Society, can you help to contextualize the benefits you saw on bone mineral content and hypochondroplasia, and how does that maybe inform or influence your expectations heading into the top line? Hi, this is Mike on for Sean. hi this is mike on for sean Thank you for taking our questions. thank you for taking our questions I guess wanted to touch on two things. i guess wanted to touch on two things First, looking at the recent data at the Pediatric Endocrine Society, can you help to contextualize the benefits you saw on bone mineral content and hypochondroplasia, and how does that maybe inform or influence your expectations heading into the top line? first looking at the recent data at the pediatric endocrine society can you help to contextualize the benefits you saw on bone mineral content and hypochondroplasia and how does that maybe inform or influence your expectations heading into the top line

Speaker 6: Yep. Thanks for the question, Mike. This is Greg Freiberg. Yeah, in addition to measuring growth velocity across both Achondroplasia historically and Hypochondroplasia, of course, we're measuring a variety of factors of health and well-being, including bone biology and bone health. The DEXA scans that were presented in that cumulative data suggests again that in addition to growing bone length and stimulating endochondral bone formation, that yes, that bone is of healthy and, you know, a strength that we would like to see, and again, reiterates what we've seen in the other FGFR3 related mutation condition of Achondroplasia as well. Going into the card flip for the phase III study, we're certainly excited to see that data. Yep. yep Thanks for the question, Mike. thanks for the question mike This is Greg Freiberg. this is greg freiberg Yeah, in addition to measuring growth velocity across both Achondroplasia historically and Hypochondroplasia, of course, we're measuring a variety of factors of health and well-being, including bone biology and bone health. yeah in addition to measuring growth velocity across both achondroplasia historically and hypochondroplasia of course we're measuring a variety of factors of health and well-being including bone biology and bone health The DEXA scans that were presented in that cumulative data suggests again that in addition to growing bone length and stimulating endochondral bone formation, that yes, that bone is of healthy and, you know, a strength that we would like to see, and again, reiterates what we've seen in the other FGFR3 related mutation condition of Achondroplasia as well. the dexa scans that were presented in that cumulative data suggests again that in addition to growing bone length and stimulating endochondral bone formation that yes that bone is of healthy and you know a strength that we would like to see and again reiterates what we've seen in the other fgfr3 related mutation condition of achondroplasia as well Going into the card flip for the phase III study, we're certainly excited to see that data. going into the card flip for the phase iii study we're certainly excited to see that data The event is going to occur before the midway point of this year. We mentioned that it's in the second quarter. We're eager to see those results. Again, I think the data at Pediatric Endocrine Society highlights the fact that hypochondroplasia, while a unique and individual indication, as compared to achondroplasia, the related biology suggests again that the hypothesis is a strong one. We've seen in Andrew Dauber's data from Children's National Hospital, again, we've seen the kind of growth that certainly would meet the criteria for a statistically significant improvement that our study is developed to measure. With regard to that safety profile, we're not anticipating any unexpected events there as well. Fingers crossed. We'll be updating you all when that data is available. The event is going to occur before the midway point of this year. the event is going to occur before the midway point of this year We mentioned that it's in the second quarter. we mentioned that it's in the second quarter We're eager to see those results. we're eager to see those results Again, I think the data at Pediatric Endocrine Society highlights the fact that hypochondroplasia, while a unique and individual indication, as compared to achondroplasia, the related biology suggests again that the hypothesis is a strong one. again i think the data at pediatric endocrine society highlights the fact that hypochondroplasia while a unique and individual indication as compared to achondroplasia the related biology suggests again that the hypothesis is a strong one We've seen in Andrew Dauber's data from Children's National Hospital, again, we've seen the kind of growth that certainly would meet the criteria for a statistically significant improvement that our study is developed to measure. we've seen in andrew dauber's data from children's national hospital again we've seen the kind of growth that certainly would meet the criteria for a statistically significant improvement that our study is developed to measure With regard to that safety profile, we're not anticipating any unexpected events there as well. with regard to that safety profile we're not anticipating any unexpected events there as well Fingers crossed. fingers crossed We'll be updating you all when that data is available. we'll be updating you all when that data is available

Speaker 17: Thank you so much. That's really helpful. Just maybe circling back onto the intro comments. Regarding the integration of Amicus, you alluded maybe there's like a roadmap for future growth acceleration. We were just wondering if you could help to comment, I mean specifically for Pombiliti or Opfolda, what levers do you see for driving increased switch rates in that market? Thank you. Thank you so much. thank you so much That's really helpful. that's really helpful Just maybe circling back onto the intro comments. just maybe circling back onto the intro comments Regarding the integration of Amicus, you alluded maybe there's like a roadmap for future growth acceleration. regarding the integration of amicus you alluded maybe there's like a roadmap for future growth acceleration We were just wondering if you could help to comment, I mean specifically for Pombiliti or Opfolda, what levers do you see for driving increased switch rates in that market? we were just wondering if you could help to comment i mean specifically for pombiliti or opfolda what levers do you see for driving increased switch rates in that market Thank you. thank you

Speaker 5: Yeah. Thank you so much. This is Cristin Hubbard here. Just having closed last week, I can say we are really, really excited about the commercial potential of both of these products, both being high-growth products to add to our portfolio. As we've mentioned before, but I'll expand a little bit, for Galafold specifically, we really do think that the biggest growth lever is in and around diagnosis. We know that a large proportion of the amenable patient population remains undiagnosed, and driving diagnosis is exactly where, you know, Amicus has really started to build momentum. We feel that using BioMarin's capability in this regard, we can really continue to drive that forward. Yeah. yeah Thank you so much. thank you so much This is Cristin Hubbard here. this is cristin hubbard here Just having closed last week, I can say we are really, really excited about the commercial potential of both of these products, both being high-growth products to add to our portfolio. just having closed last week i can say we are really really excited about the commercial potential of both of these products both being high-growth products to add to our portfolio As we've mentioned before, but I'll expand a little bit, for Galafold specifically, we really do think that the biggest growth lever is in and around diagnosis. as we've mentioned before but i'll expand a little bit for galafold specifically we really do think that the biggest growth lever is in and around diagnosis We know that a large proportion of the amenable patient population remains undiagnosed, and driving diagnosis is exactly where, you know, Amicus has really started to build momentum. we know that a large proportion of the amenable patient population remains undiagnosed and driving diagnosis is exactly where you know amicus has really started to build momentum We feel that using BioMarin's capability in this regard, we can really continue to drive that forward. we feel that using biomarin's capability in this regard we can really continue to drive that forward On the Pombiliti and Opfolda side, that's more about a switching opportunity, we really do think that there's gonna be a sizable opportunity here to build over the next few years as patients on their existing therapies begin to progress. We're really working on initiatives that again, Amicus has started, and that is around kind of starting new therapy, looking at patients that have identified their own progression and understanding what progression looks like. That's an area that we're really focused on, not to mention the continued generation of evidence to show the benefits of a switch. Again, these are areas that we feel confident in our capability at BioMarin and are very excited about the future trajectory. We'll share more on that on our Q2 call. On the Pombiliti and Opfolda side, that's more about a switching opportunity, we really do think that there's gonna be a sizable opportunity here to build over the next few years as patients on their existing therapies begin to progress. on the pombiliti and opfolda side that's more about a switching opportunity we really do think that there's gonna be a sizable opportunity here to build over the next few years as patients on their existing therapies begin to progress We're really working on initiatives that again, Amicus has started, and that is around kind of starting new therapy, looking at patients that have identified their own progression and understanding what progression looks like. we're really working on initiatives that again amicus has started and that is around kind of starting new therapy looking at patients that have identified their own progression and understanding what progression looks like That's an area that we're really focused on, not to mention the continued generation of evidence to show the benefits of a switch. that's an area that we're really focused on not to mention the continued generation of evidence to show the benefits of a switch Again, these are areas that we feel confident in our capability at BioMarin and are very excited about the future trajectory. again these are areas that we feel confident in our capability at biomarin and are very excited about the future trajectory We'll share more on that on our Q2 call. we'll share more on that on our q2 call

Speaker 9: We'll move to our next question from Salveen Richter at Goldman Sachs. We'll move to our next question from Salveen Richter at Goldman Sachs. we'll move to our next question from salveen richter at goldman sachs

Speaker 15: Thanks for taking our question. This is Tommy on for Salveen. Just a quick one on Voxzogo. Wondering if you've seen any early signals of different behavior from competitor entry, whether it be switching or in new patient adds. Thank you. Thanks for taking our question. thanks for taking our question This is Tommy on for Salveen. this is tommy on for salveen Just a quick one on Voxzogo. just a quick one on voxzogo Wondering if you've seen any early signals of different behavior from competitor entry, whether it be switching or in new patient adds. wondering if you've seen any early signals of different behavior from competitor entry whether it be switching or in new patient adds Thank you. thank you

Speaker 5: Yeah. Thank you for the question. This is Cristin again. As you heard in our prepared comments, our demand in the first quarter really does remain strong. Our enrollments, in fact, in Q1, exceeded that in the average of second half last year, and that trend continued into April. As we see it now, we're really excited. As you know, we're very much focused on the 0 to 2 population based on the consensus guidelines, and we're seeing real momentum there. Not only were our new patient starts weighted and more than half of them being in that 0 to 2 population in the first quarter, but we're also seeing a reduction in the time we have from diagnosis to treatment in that population. That is reduced by 10% already. Yeah. yeah Thank you for the question. thank you for the question This is Cristin again. this is cristin again As you heard in our prepared comments, our demand in the first quarter really does remain strong. as you heard in our prepared comments our demand in the first quarter really does remain strong Our enrollments, in fact, in Q1, exceeded that in the average of second half last year, and that trend continued into April. our enrollments in fact in q1 exceeded that in the average of second half last year and that trend continued into april As we see it now, we're really excited. as we see it now we're really excited As you know, we're very much focused on the 0 to 2 population based on the consensus guidelines, and we're seeing real momentum there. as you know we're very much focused on the 0 to 2 population based on the consensus guidelines and we're seeing real momentum there Not only were our new patient starts weighted and more than half of them being in that 0 to 2 population in the first quarter, but we're also seeing a reduction in the time we have from diagnosis to treatment in that population. not only were our new patient starts weighted and more than half of them being in that 0 to 2 population in the first quarter but we're also seeing a reduction in the time we have from diagnosis to treatment in that population That is reduced by 10% already. that is reduced by 10% already We know that that real focus therein is working. In addition, we're focused on the patients that are already on treatment and making sure that, you know, those patients for whom they're doing well on treatment, we wanna make sure that they understand the totality of the evidence that exists for Voxzogo and continue to have a positive experience in terms of the benefit. We're very pleased with how Q1 went and look forward to continuing on that trajectory. We know that that real focus therein is working. we know that that real focus therein is working In addition, we're focused on the patients that are already on treatment and making sure that, you know, those patients for whom they're doing well on treatment, we wanna make sure that they understand the totality of the evidence that exists for Voxzogo and continue to have a positive experience in terms of the benefit. in addition we're focused on the patients that are already on treatment and making sure that you know those patients for whom they're doing well on treatment we wanna make sure that they understand the totality of the evidence that exists for voxzogo and continue to have a positive experience in terms of the benefit We're very pleased with how Q1 went and look forward to continuing on that trajectory. we're very pleased with how q1 went and look forward to continuing on that trajectory

Speaker 9: We'll go next to Paul Matteis at Stifel. We'll go next to Paul Matteis at Stifel. we'll go next to paul matteis at stifel

Speaker 10: Great. Thanks so much for taking my question. Just kind of previewing your update next quarter as it relates to the Amicus integration. I was wondering if you could set the stage for us on just what you might be providing as it relates to the duration of the revenue outlook. Would you talk about peak sales at all, or update those views or anything else? Secondarily, anything you can do on setting the stage on our expectations related to accretion, cost synergies, and again, the duration of profitability outlook as well. Thank you. Great. great Thanks so much for taking my question. thanks so much for taking my question Just kind of previewing your update next quarter as it relates to the Amicus integration. just kind of previewing your update next quarter as it relates to the amicus integration I was wondering if you could set the stage for us on just what you might be providing as it relates to the duration of the revenue outlook. i was wondering if you could set the stage for us on just what you might be providing as it relates to the duration of the revenue outlook Would you talk about peak sales at all, or update those views or anything else? would you talk about peak sales at all or update those views or anything else Secondarily, anything you can do on setting the stage on our expectations related to accretion, cost synergies, and again, the duration of profitability outlook as well. secondarily anything you can do on setting the stage on our expectations related to accretion cost synergies and again the duration of profitability outlook as well Thank you. thank you

Speaker 2: Hi, Paul. Thanks. Appreciate the question. Yeah, we wanted to highlight today having, you know, just closed the transaction a week ago. You know, brief update on the integration to date, which a weekend is going well and you saw the update of our revenue guidance today, adding our expected range of the 8 months of Amicus revenues, resulting in a $500 million midpoint and the updated guidance, which is 20% year-over-year. We also wanted to set the stage for the Q2 update. From the transaction announcement, we've discussed how the strategic fit between these two businesses was very strong. Hi, Paul. hi paul Thanks. thanks Appreciate the question. appreciate the question Yeah, we wanted to highlight today having, you know, just closed the transaction a week ago. yeah we wanted to highlight today having you know just closed the transaction a week ago You know, brief update on the integration to date, which a weekend is going well and you saw the update of our revenue guidance today, adding our expected range of the 8 months of Amicus revenues, resulting in a $500 million midpoint and the updated guidance, which is 20% year-over-year. you know brief update on the integration to date which a weekend is going well and you saw the update of our revenue guidance today adding our expected range of the 8 months of amicus revenues resulting in a $500 million midpoint and the updated guidance which is 20% year-over-year We also wanted to set the stage for the Q2 update. we also wanted to set the stage for the q2 update From the transaction announcement, we've discussed how the strategic fit between these two businesses was very strong. from the transaction announcement we've discussed how the strategic fit between these two businesses was very strong That because of the both global and commercial and medical capabilities that BioMarin built over time in our existing portfolio, that these two medicines should truly have more potential within BioMarin. That was a key rationale underlying the transaction, and we've been making plans up to this point of closing. Now that we've closed, we're just digging in and engaging more deeply with the Amicus business. That's why we're gonna wait until Q2 to give this additional update. It will include a number of details and metrics about the long-term potential, including our views on peak revenue. That because of the both global and commercial and medical capabilities that BioMarin built over time in our existing portfolio, that these two medicines should truly have more potential within BioMarin. that because of the both global and commercial and medical capabilities that biomarin built over time in our existing portfolio that these two medicines should truly have more potential within biomarin That was a key rationale underlying the transaction, and we've been making plans up to this point of closing. that was a key rationale underlying the transaction and we've been making plans up to this point of closing Now that we've closed, we're just digging in and engaging more deeply with the Amicus business. now that we've closed we're just digging in and engaging more deeply with the amicus business That's why we're gonna wait until Q2 to give this additional update. that's why we're gonna wait until q2 to give this additional update It will include a number of details and metrics about the long-term potential, including our views on peak revenue. it will include a number of details and metrics about the long-term potential including our views on peak revenue Just a reminder, based on our due diligence at the time of announcement of the transaction, we reiterated what Amicus had shared with respect to peak revenue potential of roughly $1 billion each for Galafold and Pombiliti and Opfolda. But as we developed the long-term business plans and strategies to expand both of these medicines, we think that has some potential to be higher. That's what we'll share. It'll be important for us to share some of the key metrics and, you know, tactics in terms of how we expect to achieve that long-term potential. Stay tuned, and we'll look forward to sharing more in Q2. By the way, that will include some of the other financial elements as well, such as synergies, long-term profitability, accretion, et cetera. Just a reminder, based on our due diligence at the time of announcement of the transaction, we reiterated what Amicus had shared with respect to peak revenue potential of roughly $1 billion each for Galafold and Pombiliti and Opfolda. just a reminder based on our due diligence at the time of announcement of the transaction we reiterated what amicus had shared with respect to peak revenue potential of roughly $1 billion each for galafold and pombiliti and opfolda But as we developed the long-term business plans and strategies to expand both of these medicines, we think that has some potential to be higher. but as we developed the long-term business plans and strategies to expand both of these medicines we think that has some potential to be higher That's what we'll share. that's what we'll share It'll be important for us to share some of the key metrics and, you know, tactics in terms of how we expect to achieve that long-term potential. it'll be important for us to share some of the key metrics and you know tactics in terms of how we expect to achieve that long-term potential Stay tuned, and we'll look forward to sharing more in Q2. stay tuned and we'll look forward to sharing more in q2 By the way, that will include some of the other financial elements as well, such as synergies, long-term profitability, accretion, et cetera. by the way that will include some of the other financial elements as well such as synergies long-term profitability accretion et cetera I will share that for today, we are reaffirming our previous expectation that while, as you saw in the EPS guide today, the Amicus acquisition is slightly dilutive to calendar 26, but accretive for the first 12 months following closing and then substantially accretive for beginning full year 27. We'll look forward to sharing more, but we're off to a strong start. I will share that for today, we are reaffirming our previous expectation that while, as you saw in the EPS guide today, the Amicus acquisition is slightly dilutive to calendar 26, but accretive for the first 12 months following closing and then substantially accretive for beginning full year 27. i will share that for today we are reaffirming our previous expectation that while as you saw in the eps guide today the amicus acquisition is slightly dilutive to calendar 26 but accretive for the first 12 months following closing and then substantially accretive for beginning full year 27 We'll look forward to sharing more, but we're off to a strong start. we'll look forward to sharing more but we're off to a strong start

Speaker 9: We'll move next to Ellie Merle at Barclays. We'll move next to Ellie Merle at Barclays. we'll move next to ellie merle at barclays

Speaker 13: Hi, this is Jasmine on for Ellie. Thank you so much for taking our question. Just for Voxzogo and hypochondroplasia, what's the latest on what you're thinking will be good data and what you're looking to see on AGV? How are you thinking about the contribution to Voxzogo revenues from hypochondroplasia next year? What do you think the cadence of uptake will be in this population? Can you talk a little bit about your commercial preparations? Thanks. Hi, this is Jasmine on for Ellie. hi this is jasmine on for ellie Thank you so much for taking our question. thank you so much for taking our question Just for Voxzogo and hypochondroplasia, what's the latest on what you're thinking will be good data and what you're looking to see on AGV? just for voxzogo and hypochondroplasia what's the latest on what you're thinking will be good data and what you're looking to see on agv How are you thinking about the contribution to Voxzogo revenues from hypochondroplasia next year? how are you thinking about the contribution to voxzogo revenues from hypochondroplasia next year What do you think the cadence of uptake will be in this population? what do you think the cadence of uptake will be in this population Can you talk a little bit about your commercial preparations? can you talk a little bit about your commercial preparations Thanks. thanks

Speaker 6: Thanks, Jasmine. This is Greg Freiberg. Maybe I'll take the first part and then hand it off to my colleague. With regard to the CANOPY-HCH-3 study we're turning over, of course, we'll be looking at the data in the second quarter of this year. Absolutely looking forward to that. Of course, the success is a statistically significant improvement in growth as compared to the control arm. You know, in this world, of course, we're measuring a variety of other measures. We're looking of course at other anthropomorphic measures, as well as of course the safety profile and really any statistically positive improvement in growth. We think that that's a win for these patients. Thanks, Jasmine. thanks jasmine This is Greg Freiberg. this is greg freiberg Maybe I'll take the first part and then hand it off to my colleague. maybe i'll take the first part and then hand it off to my colleague With regard to the CANOPY-HCH-3 study we're turning over, of course, we'll be looking at the data in the second quarter of this year. with regard to the canopy-hch-3 study we're turning over of course we'll be looking at the data in the second quarter of this year Absolutely looking forward to that. absolutely looking forward to that Of course, the success is a statistically significant improvement in growth as compared to the control arm. of course the success is a statistically significant improvement in growth as compared to the control arm You know, in this world, of course, we're measuring a variety of other measures. you know in this world of course we're measuring a variety of other measures We're looking of course at other anthropomorphic measures, as well as of course the safety profile and really any statistically positive improvement in growth. we're looking of course at other anthropomorphic measures as well as of course the safety profile and really any statistically positive improvement in growth We think that that's a win for these patients. we think that that's a win for these patients I'll just add that, you know, we've seen pretty dramatic, I think accelerations of recruitment, both for the older children as well as, I mentioned in the pre-prepared remarks, the infants, on our hypochondroplasia study, really suggesting that this is a market that's hungry for a disease-targeted therapy. Again, the biology is significantly similar to that of achondroplasia with regard to the mutation that drives this condition. From that standpoint, and of course built upon the data that Andrew Dauber has seen, we're quite excited to see what those end results will be. I think there was a question also about the marketplace, and I'm gonna hand that one off to Cristin. I'll just add that, you know, we've seen pretty dramatic, I think accelerations of recruitment, both for the older children as well as, I mentioned in the pre-prepared remarks, the infants, on our hypochondroplasia study, really suggesting that this is a market that's hungry for a disease-targeted therapy. i'll just add that you know we've seen pretty dramatic i think accelerations of recruitment both for the older children as well as i mentioned in the pre-prepared remarks the infants on our hypochondroplasia study really suggesting that this is a market that's hungry for a disease-targeted therapy Again, the biology is significantly similar to that of achondroplasia with regard to the mutation that drives this condition. again the biology is significantly similar to that of achondroplasia with regard to the mutation that drives this condition From that standpoint, and of course built upon the data that Andrew Dauber has seen, we're quite excited to see what those end results will be. from that standpoint and of course built upon the data that andrew dauber has seen we're quite excited to see what those end results will be I think there was a question also about the marketplace, and I'm gonna hand that one off to Cristin. i think there was a question also about the marketplace and i'm gonna hand that one off to cristin

Speaker 5: Yeah. Thank you. Overall, our goal of course is to continue the growth of Voxzogo into the midterm, and hypochondroplasia provides an important component of that. Really where we're focused today on our pre-launch activities is in and around diagnosis. We have shared before that the total addressable patient population globally is at 14,000, and that assumes that we can continue to drive diagnosis and awareness of this condition. Our goal at this point in time prior to launch really is around increasing the number of hypochondroplasia patients that have been identified, that we're getting physicians to understand the genetic testing and make sure that they're putting in orders for that. Really, the idea being that patients get diagnosed at a much earlier age. Yeah. yeah Thank you. thank you Overall, our goal of course is to continue the growth of Voxzogo into the midterm, and hypochondroplasia provides an important component of that. overall our goal of course is to continue the growth of voxzogo into the midterm and hypochondroplasia provides an important component of that Really where we're focused today on our pre-launch activities is in and around diagnosis. We have shared before that the total addressable patient population globally is at 14,000, and that assumes that we can continue to drive diagnosis and awareness of this condition. really where we're focused today on our pre-launch activities is in and around diagnosis. we have shared before that the total addressable patient population globally is at 14,000 and that assumes that we can continue to drive diagnosis and awareness of this condition Our goal at this point in time prior to launch really is around increasing the number of hypochondroplasia patients that have been identified, that we're getting physicians to understand the genetic testing and make sure that they're putting in orders for that. our goal at this point in time prior to launch really is around increasing the number of hypochondroplasia patients that have been identified that we're getting physicians to understand the genetic testing and make sure that they're putting in orders for that Really, the idea being that patients get diagnosed at a much earlier age. really the idea being that patients get diagnosed at a much earlier age Our goal today is to improve upon that and make sure that we have identified patients at the time of a potential launch. Our goal today is to improve upon that and make sure that we have identified patients at the time of a potential launch. our goal today is to improve upon that and make sure that we have identified patients at the time of a potential launch

Speaker 6: Jasmine, if I could just add one other factor from the medical affairs standpoint. We're obviously actively working in the community even prior to seeing our data to really try to accelerate the number of patients that are diagnosed with this condition. You know, there's probably three prongs to that. Of course, getting patients to be able to be referred sooner to be evaluated. Of course, more testing, as well as we're doing work on the genetic side to reclassify so-called VUSs, variants of uncertain significance. In that world, we're anticipating again that there will be patients available and ready for this therapy should it become available. Jasmine, if I could just add one other factor from the medical affairs standpoint. jasmine if i could just add one other factor from the medical affairs standpoint We're obviously actively working in the community even prior to seeing our data to really try to accelerate the number of patients that are diagnosed with this condition. we're obviously actively working in the community even prior to seeing our data to really try to accelerate the number of patients that are diagnosed with this condition You know, there's probably three prongs to that. you know there's probably three prongs to that Of course, getting patients to be able to be referred sooner to be evaluated. of course getting patients to be able to be referred sooner to be evaluated Of course, more testing, as well as we're doing work on the genetic side to reclassify so-called VUSs, variants of uncertain significance. of course more testing as well as we're doing work on the genetic side to reclassify so-called vuss variants of uncertain significance In that world, we're anticipating again that there will be patients available and ready for this therapy should it become available. in that world we're anticipating again that there will be patients available and ready for this therapy should it become available

Speaker 5: Just to throw out a few components if I could quickly. On the success we've had already, when looking at the diagnosis program that we're running to date and these kind of non-promotional pre-launch activities, we're already seeing a 90% increase in the number of hypochondroplasia patients that we've identified, as well as a 70% reduction in the age. Those are precisely the types of numbers that we're driving for and wanna continue throughout our launch period. Just to throw out a few components if I could quickly. just to throw out a few components if i could quickly On the success we've had already, when looking at the diagnosis program that we're running to date and these kind of non-promotional pre-launch activities, we're already seeing a 90% increase in the number of hypochondroplasia patients that we've identified, as well as a 70% reduction in the age. on the success we've had already when looking at the diagnosis program that we're running to date and these kind of non-promotional pre-launch activities we're already seeing a 90% increase in the number of hypochondroplasia patients that we've identified as well as a 70% reduction in the age Those are precisely the types of numbers that we're driving for and wanna continue throughout our launch period. those are precisely the types of numbers that we're driving for and wanna continue throughout our launch period

Speaker 9: Excuse me. We'll move to our next question from Cory Kasimov at Evercore. Excuse me. excuse me We'll move to our next question from Cory Kasimov at Evercore. we'll move to our next question from cory kasimov at evercore

Speaker 14: Hi, this is Eddie on for Corey. Thanks for taking the question. I had a question on the guidance increase. The $500 million from midpoint increase in guidance, can you frame that? You know, the contribution from Galafold power up, is that conservative relative to the $600 million revenue they generated in FY 2025? What are the key assumptions for, you know, adding these two assets into the guidance? Hi, this is Eddie on for Corey. hi this is eddie on for corey Thanks for taking the question. thanks for taking the question I had a question on the guidance increase. i had a question on the guidance increase The $500 million from midpoint increase in guidance, can you frame that? the $500 million from midpoint increase in guidance can you frame that You know, the contribution from Galafold power up, is that conservative relative to the $600 million revenue they generated in FY 2025? you know the contribution from galafold power up is that conservative relative to the $600 million revenue they generated in fy 2025 What are the key assumptions for, you know, adding these two assets into the guidance? what are the key assumptions for you know adding these two assets into the guidance

Speaker 2: Hi, Eddie. This is Brian. Thanks a lot for the question. I appreciate the opportunity to provide a bit more color on this important element of today's update. Yeah, I'll frame it up for you. First of all, you know, the $500 million represents a midpoint of a range that fits within the range of our existing enzyme therapies guide for 2026, I'll speak to the midpoint. From a timing standpoint, just a reminder that with the transaction closing last week, that $500 million midpoint represents mostly the eight months remaining from May to December for this year. I will share in terms of, you know, your question around conservatism, I would say it's neither conservative nor aggressive. We think it's realistic. Hi, Eddie. hi eddie This is Brian. this is brian Thanks a lot for the question. thanks a lot for the question I appreciate the opportunity to provide a bit more color on this important element of today's update. i appreciate the opportunity to provide a bit more color on this important element of today's update Yeah, I'll frame it up for you. yeah i'll frame it up for you First of all, you know, the $500 million represents a midpoint of a range that fits within the range of our existing enzyme therapies guide for 2026, I'll speak to the midpoint. first of all you know the $500 million represents a midpoint of a range that fits within the range of our existing enzyme therapies guide for 2026 i'll speak to the midpoint From a timing standpoint, just a reminder that with the transaction closing last week, that $500 million midpoint represents mostly the eight months remaining from May to December for this year. from a timing standpoint just a reminder that with the transaction closing last week that $500 million midpoint represents mostly the eight months remaining from may to december for this year I will share in terms of, you know, your question around conservatism, I would say it's neither conservative nor aggressive. i will share in terms of you know your question around conservatism i would say it's neither conservative nor aggressive We think it's realistic. we think it's realistic I'll share that, you know, we've analyzed the unreported period of Amicus product revenue from January through April. When we looked at that versus consensus, January through April performance, while not reported, was ahead of consensus. You know, both Galafold and Pombiliti together are off to a strong start in 2026. With that being said, again, it's, this is the transaction closed just a week ago. We'll come back next quarter with additional color, including the long-term potential. I'll also share that, since you commented on the $600 million reported for 2025, the range that's underlying today's update, from an Amicus, you know, organic year-over-year growth rate standpoint ranges from the high teens to the low 20s. I'll share that, you know, we've analyzed the unreported period of Amicus product revenue from January through April. i'll share that you know we've analyzed the unreported period of amicus product revenue from january through april When we looked at that versus consensus, January through April performance, while not reported, was ahead of consensus. when we looked at that versus consensus january through april performance while not reported was ahead of consensus You know, both Galafold and Pombiliti together are off to a strong start in 2026. you know both galafold and pombiliti together are off to a strong start in 2026 With that being said, again, it's, this is the transaction closed just a week ago. with that being said again it's this is the transaction closed just a week ago We'll come back next quarter with additional color, including the long-term potential. we'll come back next quarter with additional color including the long-term potential I'll also share that, since you commented on the $600 million reported for 2025, the range that's underlying today's update, from an Amicus, you know, organic year-over-year growth rate standpoint ranges from the high teens to the low 20s. i'll also share that since you commented on the $600 million reported for 2025 the range that's underlying today's update from an amicus you know organic year-over-year growth rate standpoint ranges from the high teens to the low 20s We think it's a really healthy growth, and we're excited that these assets are now part of our portfolio. Again, you see this 30% increase year-over-year as a result of lay-layering this onto the BioMarin enzyme therapies and total revenue 20% at the midpoint. We're feeling good about it, and we'll share more going forward. We think it's a really healthy growth, and we're excited that these assets are now part of our portfolio. we think it's a really healthy growth and we're excited that these assets are now part of our portfolio Again, you see this 30% increase year-over-year as a result of lay-layering this onto the BioMarin enzyme therapies and total revenue 20% at the midpoint. again you see this 30% increase year-over-year as a result of lay-layering this onto the biomarin enzyme therapies and total revenue 20% at the midpoint We're feeling good about it, and we'll share more going forward. we're feeling good about it and we'll share more going forward

Speaker 14: Thank you. Thank you. thank you

Speaker 9: We'll go next to Jessica Fye at JPMorgan. We'll go next to Jessica Fye at JPMorgan. we'll go next to jessica fye at jpmorgan

Speaker 16: Hi, this is Jose on for Jess. Thanks for taking our questions. How should we think about the adoption of Voxzogo in hypochondroplasia relative to achondroplasia? What do you see as the similarities and differences between these markets? Second, can you help us bridge the disconnect between the 20% year-over-year patient growth in 1Q versus a 3% revenue growth? Is it entirely explained by larger orders in the fourth quarter last year? Thank you. Hi, this is Jose on for Jess. hi this is jose on for jess Thanks for taking our questions. thanks for taking our questions How should we think about the adoption of Voxzogo in hypochondroplasia relative to achondroplasia? how should we think about the adoption of voxzogo in hypochondroplasia relative to achondroplasia What do you see as the similarities and differences between these markets? what do you see as the similarities and differences between these markets Second, can you help us bridge the disconnect between the 20% year-over-year patient growth in 1Q versus a 3% revenue growth? second can you help us bridge the disconnect between the 20% year-over-year patient growth in 1q versus a 3% revenue growth Is it entirely explained by larger orders in the fourth quarter last year? is it entirely explained by larger orders in the fourth quarter last year Thank you. thank you

Speaker 5: Yeah. I'll address the first part of the question in terms of how we see it, in terms of uptake going in. I think we can consider it being similar to that of achondroplasia. I think, as I'd mentioned kinda earlier, what is most important to us is ensuring that the amount of disease awareness, the urgency to treat, and the diagnosis are really what we're focused on because we believe this is what accelerates that adoption curve. Our intention has been to drive as much of that so that we have patients identified at the time of our potential launch in the beginning of 2027. Importantly, that we continue on with that momentum because that ultimately is going to be what drives the shape of the adoption curve. Yeah. yeah I'll address the first part of the question in terms of how we see it, in terms of uptake going in. i'll address the first part of the question in terms of how we see it in terms of uptake going in I think we can consider it being similar to that of achondroplasia. i think we can consider it being similar to that of achondroplasia I think, as I'd mentioned kinda earlier, what is most important to us is ensuring that the amount of disease awareness, the urgency to treat, and the diagnosis are really what we're focused on because we believe this is what accelerates that adoption curve. i think as i'd mentioned kinda earlier what is most important to us is ensuring that the amount of disease awareness the urgency to treat and the diagnosis are really what we're focused on because we believe this is what accelerates that adoption curve Our intention has been to drive as much of that so that we have patients identified at the time of our potential launch in the beginning of 2027. our intention has been to drive as much of that so that we have patients identified at the time of our potential launch in the beginning of 2027 Importantly, that we continue on with that momentum because that ultimately is going to be what drives the shape of the adoption curve. importantly that we continue on with that momentum because that ultimately is going to be what drives the shape of the adoption curve

Speaker 6: Cristin, from a medical standpoint, if I could just comment. Hypochondroplasia, children are not born with the same growth deficit that the achondroplasia patients may be born with. As a result, they often are diagnosed a bit later. They're referred and diagnosed. Cristin, from a medical standpoint, if I could just comment. cristin from a medical standpoint if i could just comment Hypochondroplasia, children are not born with the same growth deficit that the achondroplasia patients may be born with. hypochondroplasia children are not born with the same growth deficit that the achondroplasia patients may be born with As a result, they often are diagnosed a bit later. as a result they often are diagnosed a bit later They're referred and diagnosed. they're referred and diagnosed That data point that we referred to earlier, which was one of our goals to try to shrink that number in terms of age of diagnosis, is something that we see as a real positive sign that the work that we're doing to get these children in the hands of the right physicians, who again, can know what to do for them, that seems like a real early success, and we're looking forward to continuing to help shape that community and make these therapies should they become available actually in the hands of the physicians who treat the patients. That data point that we referred to earlier, which was one of our goals to try to shrink that number in terms of age of diagnosis, is something that we see as a real positive sign that the work that we're doing to get these children in the hands of the right physicians, who again, can know what to do for them, that seems like a real early success, and we're looking forward to continuing to help shape that community and make these therapies should they become available actually in the hands of the physicians who treat the patients. that data point that we referred to earlier which was one of our goals to try to shrink that number in terms of age of diagnosis is something that we see as a real positive sign that the work that we're doing to get these children in the hands of the right physicians who again can know what to do for them that seems like a real early success and we're looking forward to continuing to help shape that community and make these therapies should they become available actually in the hands of the physicians who treat the patients

Speaker 2: Thanks, Greg Freiberg and Cristin Hubbard. I'll take that revenue timing or fluctuation question. I appreciate you pointing that out because it is important to emphasize that the disconnect between that underlying patient demand growth and reported revenues is entirely order timing. There's a couple of different layers to that. one, both of which we discussed last quarter, by the way. one was, you know, large international orders that were processed in Q4 that just didn't recur in Q1. The two was something that was somewhat unique to our business when looking back over the years, which was a modest amount of U.S. stocking impact from Q4 to Q1. This affected both Palynziq and Voxzogo. Thanks, Greg Freiberg and Cristin Hubbard. thanks greg freiberg and cristin hubbard I'll take that revenue timing or fluctuation question. i'll take that revenue timing or fluctuation question I appreciate you pointing that out because it is important to emphasize that the disconnect between that underlying patient demand growth and reported revenues is entirely order timing. i appreciate you pointing that out because it is important to emphasize that the disconnect between that underlying patient demand growth and reported revenues is entirely order timing There's a couple of different layers to that. one, both of which we discussed last quarter, by the way. one was, you know, large international orders that were processed in Q4 that just didn't recur in Q1. there's a couple of different layers to that one both of which we discussed last quarter by the way one was you know large international orders that were processed in q4 that just didn't recur in q1 The two was something that was somewhat unique to our business when looking back over the years, which was a modest amount of U.S. stocking impact from Q4 to Q1. the two was something that was somewhat unique to our business when looking back over the years which was a modest amount of u.s stocking impact from q4 to q1 This affected both Palynziq and Voxzogo. this affected both palynziq and voxzogo It wasn't significant, just, you know, about an extra week of inventory, but it was enough to show up as a variance in the quarter-over-quarter revenue. This is why we wanted to emphasize the underlying patient demand growth. There's no significant price drivers in there, by the way. It is entirely order timing dynamics. It wasn't significant, just, you know, about an extra week of inventory, but it was enough to show up as a variance in the quarter-over-quarter revenue. it wasn't significant just you know about an extra week of inventory but it was enough to show up as a variance in the quarter-over-quarter revenue This is why we wanted to emphasize the underlying patient demand growth. this is why we wanted to emphasize the underlying patient demand growth There's no significant price drivers in there, by the way. there's no significant price drivers in there by the way It is entirely order timing dynamics. it is entirely order timing dynamics

Speaker 9: We'll move next to Phil Nadeau at TD Cowen. We'll move next to Phil Nadeau at TD Cowen. we'll move next to phil nadeau at td cowen

Speaker 11: Good afternoon. Thanks for taking our questions.two from us. First, could you give an update on the ITC hearing? In particular, we're curious to get your thoughts on the ITC pretrial brief that was recently posted online. That's first. Second, in the press release, there's a note that there's gonna be some data from BMN 333's phase II, III trial in 2027. Could you provide a little bit more details around what data will be released at that time? Thanks. Good afternoon. good afternoon Thanks for taking our questions.two from us. thanks for taking our questions.two from us First, could you give an update on the ITC hearing? first could you give an update on the itc hearing In particular, we're curious to get your thoughts on the ITC pretrial brief that was recently posted online. in particular we're curious to get your thoughts on the itc pretrial brief that was recently posted online That's first. that's first Second, in the press release, there's a note that there's gonna be some data from BMN 333's phase II, III trial in 2027. second in the press release there's a note that there's gonna be some data from bmn 333's phase ii iii trial in 2027 Could you provide a little bit more details around what data will be released at that time? could you provide a little bit more details around what data will be released at that time Thanks. thanks

Speaker 1: Thanks very much, Phil. It's Alexander. I'll take the first part of your question, then hand it over to Greg for the second part. With regard to the ITC, I mean, you know, overall, you know, we believe that pursuing an exclusion order at the ITC, enforcing our IP is the most expeditious way to protect our IP in the U.S. As you mentioned, we recently completed the ITC evidentiary hearing, and post-hearing briefs are now being submitted to the presiding chief administrative law judge. What happens next is we expect to receive a decision on whether Ascendis's product infringes our patent on or about August 21st. At that point, if the full commission decides to review the decision, the final decision is expected on or about December 21st. Thanks very much, Phil. thanks very much phil It's Alexander. it's alexander I'll take the first part of your question, then hand it over to Greg for the second part. i'll take the first part of your question then hand it over to greg for the second part With regard to the ITC, I mean, you know, overall, you know, we believe that pursuing an exclusion order at the ITC, enforcing our IP is the most expeditious way to protect our IP in the U.S. with regard to the itc i mean you know overall you know we believe that pursuing an exclusion order at the itc enforcing our ip is the most expeditious way to protect our ip in the u.s As you mentioned, we recently completed the ITC evidentiary hearing, and post-hearing briefs are now being submitted to the presiding chief administrative law judge. as you mentioned we recently completed the itc evidentiary hearing and post-hearing briefs are now being submitted to the presiding chief administrative law judge What happens next is we expect to receive a decision on whether Ascendis's product infringes our patent on or about August 21st. what happens next is we expect to receive a decision on whether ascendis's product infringes our patent on or about august 21st At that point, if the full commission decides to review the decision, the final decision is expected on or about December 21st. at that point if the full commission decides to review the decision the final decision is expected on or about december 21st This is a little bit of, you know, where we are and what can we expect going forward. I will also add that, you know, on completion of the ITC process, we would expect to also enforce our patent in the federal district court, where, of course, monetary damages are available. I'll now hand it over to Greg. This is a little bit of, you know, where we are and what can we expect going forward. this is a little bit of you know where we are and what can we expect going forward I will also add that, you know, on completion of the ITC process, we would expect to also enforce our patent in the federal district court, where, of course, monetary damages are available. i will also add that you know on completion of the itc process we would expect to also enforce our patent in the federal district court where of course monetary damages are available I'll now hand it over to Greg. i'll now hand it over to greg

Speaker 6: Thanks for the question, Phil Nadeau, about BMN 333. As a reminder, for those on the call, this is our long-acting CNP analog, again, designed to release continuous, potentially higher AUC exposures of CNP, when administered on a weekly basis. Again, as a reminder, in our phase I, we saw over 10x increases in the AUC levels that we were able to achieve safely in the healthy volunteers. We've recently initiated our phase II/III study in children with achondroplasia. This is a multi-regional clinical trial. It's currently open in a variety of countries around the world. We're enrolling as we speak. Our goal is to run the phase II portion where one of three doses of BMN 333 will be administered to the children. Thanks for the question, Phil Nadeau, about BMN 333. thanks for the question phil nadeau about bmn 333 As a reminder, for those on the call, this is our long-acting CNP analog, again, designed to release continuous, potentially higher AUC exposures of CNP, when administered on a weekly basis. as a reminder for those on the call this is our long-acting cnp analog again designed to release continuous potentially higher auc exposures of cnp when administered on a weekly basis Again, as a reminder, in our phase I, we saw over 10x increases in the AUC levels that we were able to achieve safely in the healthy volunteers. again as a reminder in our phase i we saw over 10x increases in the auc levels that we were able to achieve safely in the healthy volunteers We've recently initiated our phase II/III study in children with achondroplasia. we've recently initiated our phase ii/iii study in children with achondroplasia This is a multi-regional clinical trial. this is a multi-regional clinical trial It's currently open in a variety of countries around the world. it's currently open in a variety of countries around the world We're enrolling as we speak. we're enrolling as we speak Our goal is to run the phase II portion where one of three doses of BMN 333 will be administered to the children. our goal is to run the phase ii portion where one of three doses of bmn 333 will be administered to the children There'll be one arm that has Voxzogo as well. There's no placebo on this study. What we will ultimately report out in 2027 is that we will be able to measure the annualized growth velocity at the six month time point, and we'll be using that data along with other measures, anthropomorphic measures, other measures of safety and well-being, to make a decision based on a Bayesian analysis of which dose we should bring forward into a phase III to run head-to-head against Voxzogo. Again, looking for a superiority profile when it comes to AGV, with the presumption that more AGV will drive more in the measurements of health and wellness that we're all familiar with. There'll be one arm that has Voxzogo as well. there'll be one arm that has voxzogo as well There's no placebo on this study. there's no placebo on this study What we will ultimately report out in 2027 is that we will be able to measure the annualized growth velocity at the six month time point, and we'll be using that data along with other measures, anthropomorphic measures, other measures of safety and well-being, to make a decision based on a Bayesian analysis of which dose we should bring forward into a phase III to run head-to-head against Voxzogo. what we will ultimately report out in 2027 is that we will be able to measure the annualized growth velocity at the six month time point and we'll be using that data along with other measures anthropomorphic measures other measures of safety and well-being to make a decision based on a bayesian analysis of which dose we should bring forward into a phase iii to run head-to-head against voxzogo Again, looking for a superiority profile when it comes to AGV, with the presumption that more AGV will drive more in the measurements of health and wellness that we're all familiar with. again looking for a superiority profile when it comes to agv with the presumption that more agv will drive more in the measurements of health and wellness that we're all familiar with

Speaker 9: We'll go next to Chris Raymond at Raymond James. We'll go next to Chris Raymond at Raymond James. we'll go next to chris raymond at raymond james

Speaker 4: Thanks. Just a question on the pivotal trial for BMN 333. I know this has come up before, but I guess I wanna just ask maybe in a more pointed way on the decision to go with a superiority trial versus non-inferiority. Thanks. thanks Just a question on the pivotal trial for BMN 333. just a question on the pivotal trial for bmn 333 I know this has come up before, but I guess I wanna just ask maybe in a more pointed way on the decision to go with a superiority trial versus non-inferiority. i know this has come up before but i guess i wanna just ask maybe in a more pointed way on the decision to go with a superiority trial versus non-inferiority I think I've heard what you guys have said around BMN 333 providing 2 to 3x free CNP and that should translate into higher efficacy. Maybe just strategically, if a non-inferiority trial could show that data, why take the risk and run a superiority study? Thanks. I think I've heard what you guys have said around BMN 333 providing 2 to 3x free CNP and that should translate into higher efficacy. i think i've heard what you guys have said around bmn 333 providing 2 to 3x free cnp and that should translate into higher efficacy Maybe just strategically, if a non-inferiority trial could show that data, why take the risk and run a superiority study? maybe just strategically if a non-inferiority trial could show that data why take the risk and run a superiority study Thanks. thanks

Speaker 6: Thanks for the question. This is Greg Freiberg. You know, our goal with BMN 333 is to evolve this space, not just make a more convenient version of Voxzogo. I'll just highlight that that 3x target was an at least 3x. We're actually testing a 3x, a 5x, and greater than 7x AUC exposure. From that standpoint, we believe strongly that BMN 333 is the right reagent to test this hypothesis. I'll also add that, you know, from a non-inferiority standpoint, it's a natural question to ask, wouldn't this be easier? From a pure mathematical standpoint, it's actually much harder, and the study would be upwards of 10x the size in order to show non-inferiority versus, you know, a drug like Voxzogo. Thanks for the question. thanks for the question This is Greg Freiberg. this is greg freiberg You know, our goal with BMN 333 is to evolve this space, not just make a more convenient version of Voxzogo. you know our goal with bmn 333 is to evolve this space not just make a more convenient version of voxzogo I'll just highlight that that 3x target was an at least 3x. i'll just highlight that that 3x target was an at least 3x We're actually testing a 3x, a 5x, and greater than 7x AUC exposure. we're actually testing a 3x a 5x and greater than 7x auc exposure From that standpoint, we believe strongly that BMN 333 is the right reagent to test this hypothesis. from that standpoint we believe strongly that bmn 333 is the right reagent to test this hypothesis I'll also add that, you know, from a non-inferiority standpoint, it's a natural question to ask, wouldn't this be easier? i'll also add that you know from a non-inferiority standpoint it's a natural question to ask wouldn't this be easier From a pure mathematical standpoint, it's actually much harder, and the study would be upwards of 10x the size in order to show non-inferiority versus, you know, a drug like Voxzogo. from a pure mathematical standpoint it's actually much harder and the study would be upwards of 10x the size in order to show non-inferiority versus you know a drug like voxzogo From that standpoint, there's a practicality of designing the study, but I do want to reassure you that we will have an opportunity to look at the data after our phase II portion. If there need to be adjustments, if the Bayesian model gives us an update that tells us we should be reevaluating, those would be potential opportunities. That being said, we are very clear with what we want out of this molecule. We want a superior CNP product that can be the cornerstone for future therapies for achondroplasia. From that standpoint, there's a practicality of designing the study, but I do want to reassure you that we will have an opportunity to look at the data after our phase II portion. from that standpoint there's a practicality of designing the study but i do want to reassure you that we will have an opportunity to look at the data after our phase ii portion If there need to be adjustments, if the Bayesian model gives us an update that tells us we should be reevaluating, those would be potential opportunities. if there need to be adjustments if the bayesian model gives us an update that tells us we should be reevaluating those would be potential opportunities That being said, we are very clear with what we want out of this molecule. that being said we are very clear with what we want out of this molecule We want a superior CNP product that can be the cornerstone for future therapies for achondroplasia. we want a superior cnp product that can be the cornerstone for future therapies for achondroplasia

Speaker 1: I would just add, absolutely, as Greg Freiberg said, this is Alexander Hardy. You know, we think the opportunity and the need here is really around superiority, in terms of efficacy, AGV, and all the benefits beyond linear growth. But also, you know, by this design of a study, we actually have an active control with Voxzogo. The size of the study is smaller than it would be if it was a non-inferiority design, as Greg Freiberg has already covered. We also think the proposition to both caregivers and physicians means that, you know, this should be a study that's very attractive to potential patients to sign up to be included, and therefore, you know, speed of recruitment, which is extremely important. I would just add, absolutely, as Greg Freiberg said, this is Alexander Hardy. i would just add absolutely as greg freiberg said this is alexander hardy You know, we think the opportunity and the need here is really around superiority, in terms of efficacy, AGV, and all the benefits beyond linear growth. you know we think the opportunity and the need here is really around superiority in terms of efficacy agv and all the benefits beyond linear growth But also, you know, by this design of a study, we actually have an active control with Voxzogo. but also you know by this design of a study we actually have an active control with voxzogo The size of the study is smaller than it would be if it was a non-inferiority design, as Greg Freiberg has already covered. the size of the study is smaller than it would be if it was a non-inferiority design as greg freiberg has already covered We also think the proposition to both caregivers and physicians means that, you know, this should be a study that's very attractive to potential patients to sign up to be included, and therefore, you know, speed of recruitment, which is extremely important. we also think the proposition to both caregivers and physicians means that you know this should be a study that's very attractive to potential patients to sign up to be included and therefore you know speed of recruitment which is extremely important We think that superiority profile we're aiming for from an efficacy standpoint, together with rapid trial recruitment and milestones being achieved is really a compelling proposition. We think that superiority profile we're aiming for from an efficacy standpoint, together with rapid trial recruitment and milestones being achieved is really a compelling proposition. we think that superiority profile we're aiming for from an efficacy standpoint together with rapid trial recruitment and milestones being achieved is really a compelling proposition

Speaker 6: Yeah, placebo-controlled studies, when there are active, safe and effective therapies, are no longer really, you know, possible to run. It's not the right thing to do either. Yeah, placebo-controlled studies, when there are active, safe and effective therapies, are no longer really, you know, possible to run. yeah placebo-controlled studies when there are active safe and effective therapies are no longer really you know possible to run It's not the right thing to do either. it's not the right thing to do either

Speaker 9: We'll take our next question from Mohit Bansal at Wells Fargo. We'll take our next question from Mohit Bansal at Wells Fargo. we'll take our next question from mohit bansal at wells fargo

Speaker 3: Great. This is Chen Xu, sound for Mohit Bansal. Thank you for taking our question. I just wanna double-click on the BMN 333 a little bit. I think you guys discussed that BMN 333 targeting, you know, improvement in achondroplasia. Just wondering with a competitor that is also advancing their weekly CNP analog together with the growth hormone and potentially reporting a higher AGV. Just how should we think about BioMarin's view of this, you know, evolving treatment landscape and the role that 333 could potentially play in over time? Thank you. Great. great This is Chen Xu, sound for Mohit Bansal. this is chen xu sound for mohit bansal Thank you for taking our question. thank you for taking our question I just wanna double-click on the BMN 333 a little bit. i just wanna double-click on the bmn 333 a little bit I think you guys discussed that BMN 333 targeting, you know, improvement in achondroplasia. i think you guys discussed that bmn 333 targeting you know improvement in achondroplasia Just wondering with a competitor that is also advancing their weekly CNP analog together with the growth hormone and potentially reporting a higher AGV. just wondering with a competitor that is also advancing their weekly cnp analog together with the growth hormone and potentially reporting a higher agv Just how should we think about BioMarin's view of this, you know, evolving treatment landscape and the role that 333 could potentially play in over time? just how should we think about biomarin's view of this you know evolving treatment landscape and the role that 333 could potentially play in over time Thank you. thank you

Speaker 6: Thanks for the question. This is Greg Freiberg again. It is true, I think the data in combination with growth hormone, of which we've seen about 12 months of data, does show in, you know, in the early studies that there is potentially additional growth to be had by adding in a second agent. I want to caution you that it is early days for the combination. Growth hormone has been around for quite a while, in achondroplasia, it's only approved in one market that I'm aware of, that's Japan. The reason for that is that, growth that can be stimulated with growth hormone, ultimately has not historically resulted in increases in final adult height. It's growth that gets uncorked, but at the expense of potentially closing the growth plates earlier. That remains an open question. Thanks for the question. thanks for the question This is Greg Freiberg again. this is greg freiberg again It is true, I think the data in combination with growth hormone, of which we've seen about 12 months of data, does show in, you know, in the early studies that there is potentially additional growth to be had by adding in a second agent. it is true i think the data in combination with growth hormone of which we've seen about 12 months of data does show in you know in the early studies that there is potentially additional growth to be had by adding in a second agent I want to caution you that it is early days for the combination. i want to caution you that it is early days for the combination Growth hormone has been around for quite a while, in achondroplasia, it's only approved in one market that I'm aware of, that's Japan. growth hormone has been around for quite a while in achondroplasia it's only approved in one market that i'm aware of that's japan The reason for that is that, growth that can be stimulated with growth hormone, ultimately has not historically resulted in increases in final adult height. the reason for that is that growth that can be stimulated with growth hormone ultimately has not historically resulted in increases in final adult height It's growth that gets uncorked, but at the expense of potentially closing the growth plates earlier. it's growth that gets uncorked but at the expense of potentially closing the growth plates earlier That remains an open question. that remains an open question I think we do need to see data two, three years or more, not only to see the safety, of course. Growth hormone, while it has a manageable safety profile, does have its own set of challenges that need to be monitored by a physician. We need to see whether or not those gains are long-standing. I want to reassure you that this is something that we're watching very closely. If there are opportunities and levers that can help CNP do its job better, so to speak, we will evaluate those at the right time and place. We don't feel that that is the correct time right now, and we're certainly interested in seeing additional data before the paradigm shifts. This is consistent with what we've heard from many of our stakeholders as well. I think we do need to see data two, three years or more, not only to see the safety, of course. i think we do need to see data two three years or more not only to see the safety of course Growth hormone, while it has a manageable safety profile, does have its own set of challenges that need to be monitored by a physician. growth hormone while it has a manageable safety profile does have its own set of challenges that need to be monitored by a physician We need to see whether or not those gains are long-standing. we need to see whether or not those gains are long-standing I want to reassure you that this is something that we're watching very closely. i want to reassure you that this is something that we're watching very closely If there are opportunities and levers that can help CNP do its job better, so to speak, we will evaluate those at the right time and place. if there are opportunities and levers that can help cnp do its job better so to speak we will evaluate those at the right time and place We don't feel that that is the correct time right now, and we're certainly interested in seeing additional data before the paradigm shifts. we don't feel that that is the correct time right now and we're certainly interested in seeing additional data before the paradigm shifts This is consistent with what we've heard from many of our stakeholders as well. this is consistent with what we've heard from many of our stakeholders as well

Speaker 9: We'll move to our next question from Joseph Schwartz at Leerink Partners. We'll move to our next question from Joseph Schwartz at Leerink Partners. we'll move to our next question from joseph schwartz at leerink partners

Speaker 8: Great. Thanks very much. I was wondering if we could get some more perspective on your guidance raise for the enzyme therapies business. We see it increased by $500 million, and we estimate that Amicus has generated around $450 million in revenue for Galafold and Pombiliti in the comparable 8 months last year. That implies low double-digit growth, around 11%, I think. I heard you reference a high double-digit percent growth, Brian. I'm just wondering if you can help us reconcile that difference. Thanks Great. great Thanks very much. thanks very much I was wondering if we could get some more perspective on your guidance raise for the enzyme therapies business. i was wondering if we could get some more perspective on your guidance raise for the enzyme therapies business We see it increased by $500 million, and we estimate that Amicus has generated around $450 million in revenue for Galafold and Pombiliti in the comparable 8 months last year. we see it increased by $500 million and we estimate that amicus has generated around $450 million in revenue for galafold and pombiliti in the comparable 8 months last year That implies low double-digit growth, around 11%, I think. that implies low double-digit growth around 11% i think I heard you reference a high double-digit percent growth, Brian. i heard you reference a high double-digit percent growth brian I'm just wondering if you can help us reconcile that difference. i'm just wondering if you can help us reconcile that difference Thanks thanks

Speaker 2: Thanks, Joe. Appreciate the question and your math there. I'd probably attribute the reconciliation difference that you're trying to get at with two elements. One, by doing a pro rata eight months of 2025, you know, there's, you know, some missing variables there and a lack of precision that we couldn't do a strict apples to apples comparison. Secondly, you know, on a full year basis, again, I pointed to that strong performance for the first four months of the year for both Galafold and Pombiliti and Opfolda. You know, again, not reported. This is internal management data. Thanks, Joe. thanks joe Appreciate the question and your math there. appreciate the question and your math there I'd probably attribute the reconciliation difference that you're trying to get at with two elements. i'd probably attribute the reconciliation difference that you're trying to get at with two elements One, by doing a pro rata eight months of 2025, you know, there's, you know, some missing variables there and a lack of precision that we couldn't do a strict apples to apples comparison. one by doing a pro rata eight months of 2025 you know there's you know some missing variables there and a lack of precision that we couldn't do a strict apples to apples comparison Secondly, you know, on a full year basis, again, I pointed to that strong performance for the first four months of the year for both Galafold and Pombiliti and Opfolda. secondly you know on a full year basis again i pointed to that strong performance for the first four months of the year for both galafold and pombiliti and opfolda You know, again, not reported. you know again not reported This is internal management data. this is internal management data We're not disclosing it because it didn't go through the usual, you know, external reporting process like a regular quarter does. We thought it was important to add color to that full year. I'd say it's more important, and this goes to our own business as well when we think about some of these quarterly timing dynamics. I think it's most important to compare the full year annual cycle year-over-year, and that's where when we piece together the range that's implied today over that, you know, full year $634 million that Amicus reported for 2025, we have a range of high teens to low 20s. I'd encourage you to anchor to that rather than trying to calculate some intra-quarter math. We're not disclosing it because it didn't go through the usual, you know, external reporting process like a regular quarter does. we're not disclosing it because it didn't go through the usual you know external reporting process like a regular quarter does We thought it was important to add color to that full year. we thought it was important to add color to that full year I'd say it's more important, and this goes to our own business as well when we think about some of these quarterly timing dynamics. i'd say it's more important and this goes to our own business as well when we think about some of these quarterly timing dynamics I think it's most important to compare the full year annual cycle year-over-year, and that's where when we piece together the range that's implied today over that, you know, full year $634 million that Amicus reported for 2025, we have a range of high teens to low 20s. i think it's most important to compare the full year annual cycle year-over-year and that's where when we piece together the range that's implied today over that you know full year $634 million that amicus reported for 2025 we have a range of high teens to low 20s I'd encourage you to anchor to that rather than trying to calculate some intra-quarter math. i'd encourage you to anchor to that rather than trying to calculate some intra-quarter math

Speaker 9: We'll move next to Jason Gerberry at Bank of America. We'll move next to Jason Gerberry at Bank of America. we'll move next to jason gerberry at bank of america

Speaker 7: Hey, guys. Thanks for taking my questions. Alex, just to follow up on the ITC question, I'm trying to just get my head around, do you have any knowledge of the Ascendis ability to do manufacturing workaround? Just help us think through scenarios if you get a positive ruling, like could a decision be stayed pending any sort of appeals? Then just as a follow-up, just on Palm Op, I believe, you've inherited the asset basically launched into 15 country markets and I think the goal is to get it into 80 country markets. How should we think about the phasing of that now that you've got the asset either 2026 or 2027? Thanks. Hey, guys. hey guys Thanks for taking my questions. thanks for taking my questions Alex, just to follow up on the ITC question, I'm trying to just get my head around, do you have any knowledge of the Ascendis ability to do manufacturing workaround? alex just to follow up on the itc question i'm trying to just get my head around do you have any knowledge of the ascendis ability to do manufacturing workaround Just help us think through scenarios if you get a positive ruling, like could a decision be stayed pending any sort of appeals? just help us think through scenarios if you get a positive ruling like could a decision be stayed pending any sort of appeals Then just as a follow-up, just on Palm Op, I believe, you've inherited the asset basically launched into 15 country markets and I think the goal is to get it into 80 country markets. then just as a follow-up just on palm op i believe you've inherited the asset basically launched into 15 country markets and i think the goal is to get it into 80 country markets How should we think about the phasing of that now that you've got the asset either 2026 or 2027? how should we think about the phasing of that now that you've got the asset either 2026 or 2027 Thanks. thanks

Speaker 1: Thanks very much for the questions. I'm, unfortunately, I'm not going to give any further details of the ITC, the potential, you know, scenarios around it. I hope you can understand that we don't want to get into too many specifics, especially whilst the case is very much pending. I'll hand it over now to Cristin for the second part. Thanks very much for the questions. thanks very much for the questions I'm, unfortunately, I'm not going to give any further details of the ITC, the potential, you know, scenarios around it. i'm unfortunately i'm not going to give any further details of the itc the potential you know scenarios around it I hope you can understand that we don't want to get into too many specifics, especially whilst the case is very much pending. i hope you can understand that we don't want to get into too many specifics especially whilst the case is very much pending I'll hand it over now to Cristin for the second part. i'll hand it over now to cristin for the second part

Speaker 5: Yeah. Thank you very much for the question around Pombiliti and Opfolda. You're absolutely correct in that there are currently we are reimbursed in 15 countries. What we've been doing, you know, before the transaction was closed, we've been doing significant and deep dive, you know, discovery sessions, really looking at the business in each market for both Galafold and Pombiliti and Opfolda, really understanding what the dynamics are therein. Importantly looking at, as you've mentioned, our 80-country footprint and saying, "Where would the potential and the opportunity be?" We're identifying opportunities for both Galafold as well as Pombiliti and Opfolda. Given that Pombiliti and Opfolda is much earlier in its launch trajectory, you can imagine that there'll be a larger number of countries to look at therein. Yeah. yeah Thank you very much for the question around Pombiliti and Opfolda. thank you very much for the question around pombiliti and opfolda You're absolutely correct in that there are currently we are reimbursed in 15 countries. you're absolutely correct in that there are currently we are reimbursed in 15 countries What we've been doing, you know, before the transaction was closed, we've been doing significant and deep dive, you know, discovery sessions, really looking at the business in each market for both Galafold and Pombiliti and Opfolda, really understanding what the dynamics are therein. what we've been doing you know before the transaction was closed we've been doing significant and deep dive you know discovery sessions really looking at the business in each market for both galafold and pombiliti and opfolda really understanding what the dynamics are therein Importantly looking at, as you've mentioned, our 80-country footprint and saying, "Where would the potential and the opportunity be?" We're identifying opportunities for both Galafold as well as Pombiliti and Opfolda. importantly looking at as you've mentioned our 80-country footprint and saying "where would the potential and the opportunity be?" we're identifying opportunities for both galafold as well as pombiliti and opfolda Given that Pombiliti and Opfolda is much earlier in its launch trajectory, you can imagine that there'll be a larger number of countries to look at therein. given that pombiliti and opfolda is much earlier in its launch trajectory you can imagine that there'll be a larger number of countries to look at therein It's important to note that we're not necessarily going to put it into our entire 80-country footprint, but importantly look at where we believe the potential opportunities are and then have a cadence to that that we'll share more of in the Q2 call. It's important to note that we're not necessarily going to put it into our entire 80-country footprint, but importantly look at where we believe the potential opportunities are and then have a cadence to that that we'll share more of in the Q2 call. it's important to note that we're not necessarily going to put it into our entire 80-country footprint but importantly look at where we believe the potential opportunities are and then have a cadence to that that we'll share more of in the q2 call

Speaker 9: That concludes the Q&A session. I will now turn the conference back over to BioMarin CEO, Alexander Hardy, for closing remarks. That concludes the Q&A session. that concludes the q&a session I will now turn the conference back over to BioMarin CEO, Alexander Hardy, for closing remarks. i will now turn the conference back over to biomarin ceo alexander hardy for closing remarks

Speaker 1: Thank you, operator, and thank you all for joining us today. This quarter, as you can tell, marks a really important inflection point for BioMarin. With the recent close of the Amicus acquisition expanding our commercial re-reach, strengthening our 2026 revenue growth outlook to 20% and enhancing our ability to serve more patients globally. We're encouraged by the robust patient demand observed across our portfolio. In enzyme therapies, we anticipate that the momentum from the Palynziq launch in adolescence will continue to build. With Voxzogo, a consistent rise in new patient initiations, more than 20% year-over-year in Q1, especially among younger children, demonstrates confidence in its long-term safety and efficacy and highlights the importance of starting treatment as early as possible. Thank you, operator, and thank you all for joining us today. thank you operator and thank you all for joining us today This quarter, as you can tell, marks a really important inflection point for BioMarin. this quarter as you can tell marks a really important inflection point for biomarin With the recent close of the Amicus acquisition expanding our commercial re-reach, strengthening our 2026 revenue growth outlook to 20% and enhancing our ability to serve more patients globally. with the recent close of the amicus acquisition expanding our commercial re-reach strengthening our 2026 revenue growth outlook to 20% and enhancing our ability to serve more patients globally We're encouraged by the robust patient demand observed across our portfolio. we're encouraged by the robust patient demand observed across our portfolio In enzyme therapies, we anticipate that the momentum from the Palynziq launch in adolescence will continue to build. in enzyme therapies we anticipate that the momentum from the palynziq launch in adolescence will continue to build With Voxzogo, a consistent rise in new patient initiations, more than 20% year-over-year in Q1, especially among younger children, demonstrates confidence in its long-term safety and efficacy and highlights the importance of starting treatment as early as possible. with voxzogo a consistent rise in new patient initiations more than 20% year-over-year in q1 especially among younger children demonstrates confidence in its long-term safety and efficacy and highlights the importance of starting treatment as early as possible With the integration of Amicus now well underway, several near-term catalysts ahead, pipeline readouts, we are focused on translating this momentum into accelerated growth, broader patient impact, meaningful value creation. We appreciate your continued support and look forward to updating you next quarter. Thank you. With the integration of Amicus now well underway, several near-term catalysts ahead, pipeline readouts, we are focused on translating this momentum into accelerated growth, broader patient impact, meaningful value creation. with the integration of amicus now well underway several near-term catalysts ahead pipeline readouts we are focused on translating this momentum into accelerated growth broader patient impact meaningful value creation We appreciate your continued support and look forward to updating you next quarter. we appreciate your continued support and look forward to updating you next quarter Thank you. thank you

Speaker 9: This concludes today's Conference Call. Thank you for your participation. You may now disconnect. This concludes today's Conference Call. this concludes today's conference call Thank you for your participation. thank you for your participation You may now disconnect. you may now disconnect