BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) Q1 2026 Earnings Call Transcript

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) Q1 2026 Earnings Call Transcript May 4, 2026

BioMarin Pharmaceutical Inc. misses on earnings expectations. Reported EPS is $0.54 EPS, expectations were $0.94.

Operator: Good afternoon and welcome everyone to the BioMarin Pharmaceutical Inc. First Quarter 2026 Conference Call. Today’s conference is being recorded. All lines have been placed on mute to prevent any background noise. After the speakers’ remarks, there will be a question and answer session. If you would like to ask a question during this time, simply press the star key followed by the number one on your telephone keypad. If you would like to withdraw your question, press star 1 again. At this time, I would like to turn the conference over to Traci McCarty, Head of Investor Relations. Please go ahead.

Traci McCarty: 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 Pharmaceutical Inc.’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 Pharmaceutical Inc.’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 Pharmaceutical Inc.’s filings with the Securities and Exchange Commission, such as 10-Q, 10-Ks, and 8-Ks 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. 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 and introducing BioMarin Pharmaceutical Inc.’s management team, joining today’s call are Alexander Hardy, chief executive officer; Brian Mueller, chief financial officer; Cristin Hubbard, chief commercial officer; and Gregory Friberg, chief R&D officer.

I will now turn the call over to BioMarin Pharmaceutical Inc.’s President and CEO, Alexander Hardy.

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 Pharmaceutical Inc. with the addition of two innovative therapies, Galafold for Fabry disease and Pombility 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 strengthening trajectory is just the beginning of BioMarin Pharmaceutical Inc.’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.

Q&A Session

Following the recent close of the transaction, we initiated our targeted integration plan focused on leveraging BioMarin Pharmaceutical Inc.’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 road map as well as more detail on BioMarin Pharmaceutical Inc.’s growth acceleration with the addition of Galafold and Pombility and Opfolda to our commercial portfolio, DMX-200 for FSGS in phase three 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 United States.

With Cristin, who will discuss in more detail, we expect Enzyme Therapies will deliver robust growth in 2026, further supported by the addition of Galafold and Pombility 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 first 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 full 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, those later in Q2. In summary, we expect 2026 to be a momentous year for BioMarin Pharmaceutical Inc. 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 Pombility and Opfolda to BioMarin Pharmaceutical Inc.’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 Pharmaceutical Inc. and Amicus whose dedication forms the foundation of our shared mission to serve patients. Thank you for your attention, and I will now turn the call over to Brian to provide additional financial updates. Brian?

Brian Mueller: 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. Now 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 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 12 to 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 eight. 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 capability. 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 three clinical program acquired in the Enzyme Therapies transaction. The 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 Pharmaceutical Inc.’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 Pharmaceutical Inc.

business before layering on the Amicus business. Now moving to slide nine, 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 to $2.775 billion for the full year 2026 inclusive of meaningful contributions from Galafold and Pombility 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.

For 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 dilutive for the full year 2026. We continue to expect the acquisition to be accretive to non-GAAP diluted earnings per share in the first twelve months after close and substantially accretive beginning in 2027. The Amicus P&L will be incorporated into BioMarin Pharmaceutical Inc.’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 Pharmaceutical Inc. products and two full quarters of Galafold and Pombility 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 Pombility and Opfolda, and setting the stage for accelerated near- and mid-term growth. Thank you for your attention. I will now turn it over to Cristin for a commercial update. Cristin?

Cristin Hubbard: 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 Pombility 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 Pombility and Opfolda are marketed, while we concurrently work on geographic expansion plans for both products. We look forward to updating you on our Q2 call. Now moving to slide 11. I will 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 health care providers treating adolescents during this critical stage of their development. Since approval, we have observed meaningful enrollments and new patient starts in people under 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, so 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 Q4, particularly in international markets. Importantly, we have 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 a competitor having recently entered the U.S. market, our focus has remained on executing our strategy, and we continue to see strong momentum. During the first quarter, solid 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 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 are making progress in preparations for potential expansion of Voxzogo into hypochondroplasia. As we get closer to the phase three 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 three top-line results in the second quarter of 2026 and submitting to global health authorities in the second 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 will turn it over to Greg for an R&D update. Greg?

Gregory Friberg: Thank you, Cristin. As expected, 2026 is shaping up to be an active year for R&D, and we are looking forward to delivering multiple meaningful milestones. Moving to slide 14, we have built a comprehensive Voxzogo evidence pack 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 the 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, these data span 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 requirements, 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 health care 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 three pivotal trial. We look forward to sharing these phase three top-line results in the second quarter. In parallel, enrollment is progressing very well in our phase two 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 three 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 one to 12 includes two co-primary endpoints. The first is change from baseline in plasma measured inorganic pyrophosphate through week 52. The second is change in the RGIC, or Rickets 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 would 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 2/3 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 would 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 mg/kg 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 assessment and the six-minute walk test when compared to historically matched controls. So far, we are encouraged by both the dystrophin expression levels and the functional improvements observed in our development program.

Enrollment in the 12 mg/kg cohort is ongoing, and we look forward to providing an additional update by year end as the 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?

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 1 on your telephone keypad to raise your hand and join the queue. We will go first to Sean Laaman at Morgan Stanley.

Analyst: Hi. This is Mike on for Sean. Thank you for taking our questions. I guess I 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 in hypochondroplasia? And how does that maybe inform or influence your expectations heading into the top line? And then just maybe circling back onto the intro comments regarding the integration of Amicus, you alluded to a road map for future growth acceleration. We were just wondering if you could help to comment, specifically for Pombility and Opfolda, what levers you see for driving increased switch rates in that market?

Gregory Friberg: Yep. Thanks for the question, Mike. In addition to measuring growth velocity across both achondroplasia historically and hypochondroplasia, we are measuring a variety of factors of health and well-being, bone biology, and bone health. The DEXA scans that were presented in that cumulative data suggest that, in addition to growing bone length and stimulating endochondral bone formation, that bone shows the strength and health we would like to see and reiterates what we have seen in the other FGFR3-related mutation conditions of achondroplasia as well. Going into the card flip for the phase three study, we are certainly excited to see that data. The event is going to occur before the midway point of this year. As we mentioned, it is in the second quarter.

We are eager to see those results. The data at PES highlights the fact that hypochondroplasia, while a unique and individual indication as compared to achondroplasia, has related biology that suggests the hypothesis is a strong one. We have seen in Dr. Dauber’s data from Children’s National that the kind of growth observed would meet the criteria for a statistically significant improvement that our study is developed to measure. With regard to the safety profile, we are not anticipating any unexpected events there as well. So fingers crossed, and we will be updating you all when that data is available.

Cristin Hubbard: Thank you so much. This is Cristin Hubbard here. Just having closed last week, I can say we are really excited about the commercial potential of both of these products, both being high-growth products to add to our portfolio. As we have mentioned before, for Galafold specifically, we really 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 Amicus has really started to build momentum. We feel that, using BioMarin Pharmaceutical Inc.’s capability in this regard, we can really continue to drive that forward. On the Pombility and Opfolda side, that is more about a switching opportunity, and we really think that there is going to be a sizable opportunity here to build over the next few years as patients on their existing therapies begin to progress.

We are working on initiatives that Amicus has started, including starting new therapy, looking at patients who have identified their own progression and understanding what progression looks like. That is an area that we are really focused on, not to mention the continued generation of evidence to show the benefits of a switch. These are areas that we feel confident in our capability at BioMarin Pharmaceutical Inc. and are very excited about the future trajectory. We will share more on that on our Q2 call.

Tommie Reerink: This is Tommie on for Salveen. Just a quick one on Voxzogo, wondering if you have seen any early signals of different behavior from competitor entry, whether it be switching or in new patients. Thank you.

Cristin Hubbard: Thank you for the question. As you heard in our prepared comments, our demand in the first quarter remains strong. Our enrollments in Q1 exceeded the average of the second half last year, and that trend continued into April. We are very much focused on the zero to two population based on the consensus guidelines. We are seeing real momentum there. Not only were more than half of new patient starts in that zero to two population in the first quarter, but we are also seeing a reduction in the time from diagnosis to treatment in that population, reduced by 10% already. We know that focus is working. In addition, we are focused on the patients who are already on treatment and making sure that those patients who are doing well on treatment understand the totality of the evidence that exists for Voxzogo and continue to have a positive experience in terms of benefit. We are very pleased with how Q1 went and look forward to continuing on that trajectory.

Paul Andrew Matteis: Great. Thanks so much for taking my question. Just kind of previewing your update next quarter as it relates to the Amicus integration. Was wondering if you could set the stage for us on 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? And then secondarily, anything you can do on setting the stage on our expectations related to accretion, synergies, and again, the duration of profitability outlook as well? Thank you.

Brian Mueller: Hi, Paul. Thanks. We wanted to highlight today, having just closed the transaction a week ago, a brief update on the integration to date, which is going well, and you saw the update of our revenue guidance today adding our expected range of the eight 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. At transaction announcement, we discussed how the strategic fit between these two businesses was very strong and that, because of the global commercial and medical capabilities BioMarin Pharmaceutical Inc. built over time in our existing portfolio, these two medicines should truly have more potential within BioMarin Pharmaceutical Inc.

That was a key rationale underlying the transaction, and we have been making plans up to this point of closing. Now that we have closed, we are digging in and engaging more deeply with the Amicus business. That is why we are going to wait until Q2 to give this additional update, but 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 Pombility and Opfolda. As we develop the long-term business plans and strategies to expand both of these medicines, we think that has some potential to be higher.

We will share key metrics and tactics in terms of how we expect to achieve that potential. Stay tuned, and we will look forward to sharing more in Q2. That will include other financial elements as well, such as synergies, long-term profitability, and accretion. 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 2026, it will be accretive for the first twelve months following closing and then substantially accretive beginning full year 2027. We are off to a strong start.

Jasmine Fels: Hi. This is Jasmine on for Ellie. Thank you so much for taking our question. For Voxzogo in hypochondroplasia, what is the latest on what you are thinking will be good data and what you are looking to see on AGV? Also, 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? And can you talk a little bit about your commercial preparations?

Gregory Friberg: Thanks, Jasmine. With regard to the VOX-HCH study, we will be looking at the data in the second quarter of this year and absolutely look forward to that. Success is a statistically significant improvement in growth as compared to the control arm. We are measuring a variety of other measures, including other anthropometric measures, as well as the safety profile. Any statistically positive improvement in growth is a win for these patients. We have seen pretty dramatic accelerations of recruitment both for the older children and, as mentioned in the prepared remarks, the infants on our hypochondroplasia study, suggesting that this is a market that is hungry for a disease-targeted therapy. The biology is significantly similar to that of achondroplasia with regard to the mutation that drives this condition, and built upon the data that Dr. Dauber has seen, we are excited to see the end results.

I think there was a question also about the marketplace, and I will hand that one off to Cristin.

Cristin Hubbard: Thank you. Overall, our goal is to continue the growth of Voxzogo, and hypochondroplasia provides an important component of that. Our pre-launch activities are focused on 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 prior to launch is increasing the number of hypochondroplasia patients that have been identified, getting physicians to understand the genetic testing, and making sure they are ordering it, with the aim of diagnosing patients at a much earlier age so they are identified at the time of a potential launch.

Gregory Friberg: From the medical affairs standpoint, we are actively working in the community, even prior to seeing our data, to accelerate the number of patients diagnosed with this condition. There are three prongs: getting patients referred sooner to be evaluated; enabling testing; and doing work on the genetic side to reclassify so-called VUSs, variants of uncertain significance. We anticipate there will be patients available and ready for this therapy should it become available.

Cristin Hubbard: On the success we have had already with the diagnosis program and non–pre-launch activities, we are seeing a 90% increase in the number of hypochondroplasia patients identified, as well as a 70% reduction in the age at diagnosis. Those are precisely the types of numbers that we are driving for and want to continue throughout our launch period.

Cory William Kasimov: This is Adi on for Cory. I had a question on the guidance increase. The $500 million midpoint increase in guidance—can you frame the contribution from Galafold and Opfolda? Is that conservative relative to the $600 million revenue they generated in fiscal year 2025? What are the key assumptions for adding these two assets into the guidance?

Brian Mueller: Hi, Adi. Thanks for the question. The $500 million represents a midpoint of a range that fits within the range of our existing Enzyme Therapies guide for 2026, and, from a timing standpoint, represents mostly the eight months remaining from May to December for this year. On conservatism, I would say it is neither conservative nor aggressive—realistic. We analyzed the unreported period of Amicus product revenue from January through April, and when we looked at that versus consensus, January through April performance, while not reported, was ahead of consensus. Both Galafold and Pombility together are off to a strong start in 2026. We will come back next quarter with additional color, including the long-term potential.

Since you commented on the $600 million reported for 2025, the range underlying today’s update from an Amicus organic year-over-year growth rate standpoint ranges from the high teens to the low 20s. It is really healthy growth, and we are excited these assets are now part of our portfolio. You see the 30% increase year over year in Enzyme Therapies as a result of layering this onto BioMarin Pharmaceutical Inc., and total revenue growth of 20% at the midpoint. We will share more going forward.

Analyst: 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? And second, can you help us bridge the disconnect between the 20% year-over-year patient growth in Q1 versus a 3% revenue growth? Is it entirely explained by larger orders in the fourth quarter last year? Thank you.

Cristin Hubbard: I think we can consider adoption to be similar to that of achondroplasia, but, as mentioned earlier, what is most important is ensuring disease awareness, urgency to treat, and diagnosis. We believe this is what accelerates the adoption curve. Our intention has been to drive as much of that so that we have patients identified at the time of a potential launch in the beginning of 2027 and importantly to continue that momentum, because that ultimately will drive the shape of the adoption curve.

Gregory Friberg: From a medical standpoint, hypochondroplasia children are not born with the same growth deficit that achondroplasia patients may be born with. As a result, they often are referred and diagnosed a bit later. Shrinking the age at diagnosis is a real positive sign that the work we are doing to get these children in the hands of the right physicians is effective. We are 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.

Brian Mueller: Thanks for the revenue timing question. It is important to emphasize that the disconnect between the underlying patient demand growth and reported revenues is entirely order timing. There are a couple of layers to that, both of which we discussed last quarter. One was large international orders that were processed in Q4 that did not recur in Q1. The second was a modest amount of U.S. stocking impact from Q4 to Q1. This affected both Palynziq and Voxzogo. It was enough to show up as variance in the quarter-over-quarter revenue. This is why we emphasized the underlying patient demand growth. There are no significant price drivers—this is entirely an order timing dynamic.

Philip Nadeau: Good afternoon. Thanks for taking our questions. Two from us. First, could you give an update on the ITC hearing and, in particular, your thoughts on the ITC pretrial brief that was recently posted online? Second, in the press release, there is a note that there will be some data from BMN-333’s phase 2/3 trial in 2027. Could you provide a bit more detail around what data will be released at that time?

Alexander Hardy: Thanks very much, Phil. Overall, we believe that seeking an exclusion order at the ITC and enforcing our IP is the most expeditious way to protect our IP in the United States. We recently completed the ITC evidentiary hearing, and post-hearing briefs are now being submitted to the presiding Chief Administrative Law Judge. We expect to receive a decision on whether Ascendis’ product infringes our patent on or about August 21. If the full Commission decides to review the decision, then the final decision is expected on or about December 21. On completion of the ITC process, we would expect to also enforce our patent in the federal district court, where monetary damages are available.

Gregory Friberg: Thanks for the question about BMN-333. As a reminder, this is our long-acting CNP analog to release continuous, potentially higher AUC exposures of CNP when administered on a weekly basis. In our phase one, we saw over 10x increases in the AUC levels achieved safely in healthy volunteers. We have recently initiated our phase 2/3 study in children with achondroplasia. This is a multiregional clinical trial currently open in a variety of countries around the world, and we are enrolling as we speak. Our goal is to run the phase two portion where one of three doses of BMN-333 will be administered to children, and there will be one arm that has Voxzogo as well. There is no placebo in this study. In 2027, we will report annualized growth velocity at the six-month time point and use that data, along with other anthropometric measures and safety and well-being, to make a decision—based on a Bayesian analysis—of which dose to bring forward into a phase three to run head-to-head against Voxzogo, looking for a superiority profile in AGV, with the presumption that more AGV will drive more improvements in the measurements of health and wellness that we all are familiar with.

Christopher Raymond: Thanks. Just a question on the pivotal trial for BMN-333. I know this has come up before, but I want to ask in a more pointed way about the decision to go with a superiority trial versus noninferiority. I think I have heard what you have said around BMN-333 providing two to three times free CNP and that should translate into higher efficacy, but maybe just strategically, if a noninferiority trial could show that data, why take the risk and run a superiority study? Thanks.

Gregory Friberg: Thanks for the question. Our goal with BMN-333 is to evolve this space, not just make a more convenient version of Voxzogo. That 3x target was an at least 3x. We are actually testing approximately 3x, 5x, and greater than 7x AUC exposure. We believe strongly that BMN-333 is the right reagent to test this hypothesis. From a noninferiority standpoint, it is a natural question to ask if it would be easier, but from a mathematical standpoint, it is actually much harder, and the study would be upwards of 10 times the size in order to show noninferiority versus a drug like Voxzogo. There is a practicality of designing the study. We will have an opportunity to look at the data after our phase two portion, and if there need to be adjustments, the Bayesian model gives us an update that could prompt reevaluation.

That said, we are very clear with what we want out of this molecule: a superior CNP product that can be the cornerstone for future therapies for achondroplasia.

Alexander Hardy: I would just add, as Greg said, we think the opportunity and the need here is around superiority in efficacy—AGV and benefits beyond linear growth. Also, by this study design, we have an active control with Voxzogo. So the size of the study is smaller than it would be if it were a noninferiority design, as Greg has covered. We also think the proposition to both caregivers and physicians makes this a study that is very attractive to potential patients to enroll in, which supports speed of recruitment—extremely important for achieving milestones.

Gregory Friberg: Placebo-controlled studies, when there are active, safe, and effective therapies, are no longer really possible to run, nor is it the right thing to do.

Analyst: Great. This is Chen Shui for Mohit. Thank you for taking our question. We just want to double-click on the competitive landscape a bit. Competitors are advancing their weekly CNP analog together with growth hormone, potentially reporting a higher AGV. How should we think about BioMarin Pharmaceutical Inc.’s view of the evolving treatment landscape and the role that BMN-333 could potentially play over time? Thank you.

Gregory Friberg: Thanks for the question. The data in combination with growth hormone, of which we have seen about 12 months, does show in early studies that there is potentially additional growth by adding a second agent. But it is early days for the combination. Growth hormone has been around for quite a while, and in achondroplasia, it is only approved in one market that I am aware of—that is Japan. The reason is that growth stimulated with growth hormone ultimately has not historically resulted in increases in final adult height. It is growth that gets uncorked but at the expense of potentially closing the growth plates earlier, and that remains an open question. We need to see data at two to three years or more, not only to see safety—growth hormone, while it has a manageable safety profile, has its own set of challenges that need to be monitored by a physician—but also to see whether those gains are long-standing.

We are watching this closely. If there are opportunities and levers that can help CNP do its job better, we will evaluate those at the right time and place. We do not feel that is the correct time right now, and we are interested in seeing additional data before the paradigm shifts. This is consistent with what we have heard from many of our stakeholders as well.

Joseph Schwartz: Great. Thanks very much. I was wondering if we could get more perspective on your guidance raise for the Enzyme Therapies business. We see it increase by $500 million, and we estimate that Amicus generated around $450 million in revenue for Galafold and Pombility in the comparable eight months last year. That implies low double-digit growth around 11%, I think. But I heard you reference high double-digit percent growth, Brian. Can you help us reconcile that difference? Thanks.

Brian Mueller: Thanks, Joe. I would attribute the reconciliation difference to two elements. One, by doing a pro rata eight months of 2025, there are missing variables and a lack of precision—you cannot do a strict apples-to-apples comparison. Secondly, on a full-year basis, I pointed to the strong performance for the first four months of the year for both Galafold and Pombility and Opfolda. While not reported—this is internal management data—we thought it was important to add color to the full year. It is more important to compare the full-year annual cycle year over year. When we piece together the range implied today over the full-year $634 million that Amicus reported for 2025, we have a range of high teens to low 20s. I encourage you to anchor to that rather than trying to calculate intra-quarter math.

Jason Matthew Gerberry: Alex, just to follow up on the ITC question. Do you have any knowledge of Ascendis’ ability to do a manufacturing workaround, and can you help us think through scenarios if you get a positive ruling? Could a decision be stayed pending any appeals? And then, as a follow-up on Pombility, I believe you have inherited the asset basically launched into 15 country markets, and the goal is to get it into 80 country markets. How should we think about the phasing of that now that you have the asset, either 2026 or 2027? Thanks.

Alexander Hardy: Thanks very much for the questions. Unfortunately, I am not going to share any details of the ITC potential scenarios around it. I hope you can understand that we do not want to get into specifics, especially while the case is pending. I will hand it over now to Cristin for the second part.

Cristin Hubbard: Thank you for the question around Pombility. You are correct that currently we are reimbursed in 15 countries. Before the transaction was closed, we were doing deep-dive discovery sessions, looking at the business in each market for both Galafold and Pombility, understanding the dynamics therein, and then looking at our 80-country footprint to identify where the potential and opportunity would be. We are identifying opportunities for both Galafold and Pombility. Given that Pombility is much earlier in its launch trajectory, you can imagine that there will be a larger number of countries to look at there. It is important to note that we are not necessarily going to put it into our entire 80-country footprint, but will look at where we believe the potential opportunities are and then have a cadence to that which we will share more of on the Q2 call.

Operator: And that concludes the Q&A session. I will now turn the conference back over to BioMarin Pharmaceutical Inc.’s CEO, Alexander Hardy, for closing remarks.

Alexander Hardy: Thank you, operator, and thank you all for joining us today. This quarter marks an important inflection point for BioMarin Pharmaceutical Inc., with the recent close of the Amicus acquisition expanding our commercial reach, strengthening our 2026 revenue growth outlook to 20%, and enhancing our ability to serve more patients globally. We are encouraged by the robust patient demand observed across our portfolio. In Enzyme Therapies, we anticipate that the momentum from the Palynziq launch in adolescents 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 and several near-term catalysts ahead, including pipeline readouts, we are focused on translating this momentum into accelerated growth, broader patient impact, and meaningful value creation. We appreciate your continued support and look forward to updating you next quarter. Thank you.

Operator: And this concludes today’s conference call. Thank you for your participation. You may now disconnect.