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DATE

May 4, 2026 at 4:30 p.m. ET

CALL PARTICIPANTS

  • Chief Executive Officer — Alexander Hardy
  • Chief Financial Officer — Brian Mueller
  • Chief Commercial Officer — Cristin Hubbard
  • Chief Research & Development Officer — Gregory Friberg

TAKEAWAYS

  • Revenue -- $766 million total for the quarter, driven by increased patient demand in enzyme therapies and Voxogo, partly offset by order timing and lower revenues from Rocktadian, Kuvan, and royalties.
  • Enzyme Therapies Revenue Growth -- Increased 6% year over year, led by Vimizim, Naglazyme, and Brineura.
  • Voxogo Patient Growth -- Number of children treated increased by more than 20% year over year, with a majority of new U.S. patients under age two.
  • Cost of Sales -- Increased by $31 million due to an unsuccessful Naglazyme manufacturing campaign, impacting quarterly margin but not product supply.
  • R&D and SG&A Expenses -- Non-GAAP R&D expense rose, driven by BMN 401 development and other pipeline programs; SG&A increased due to commercial expansion and Amicus pre-close costs.
  • Non-GAAP Diluted EPS -- $0.76, significantly impacted by operating expense increases, the Naglazyme charge, and Amicus-related pre-close costs; combined, these impacts were a $0.20 share reduction.
  • 2026 Revenue Guidance Update -- Total revenue guidance raised to $3.825 billion-$3.925 billion inclusive of Amicus, projecting approximately 20% growth at midpoint, with enzyme therapies guidance increased to $2.725 billion-$2.775 billion (about 30% growth midpoint) including Galafold, Pombility, and Upholda.
  • Voxogo 2026 Guidance -- Unchanged at $975 million-$1.025 billion, representing high single-digit growth at the midpoint.
  • 2026 Non-GAAP EPS Guidance -- Updated to $4.85-$5.05, noting that the Amicus acquisition will be slightly dilutive for the full year 2026 but accretive in the first twelve months and substantially accretive from 2027.
  • Revenue and EPS Timing -- More than 55% of 2026 revenue and approximately two-thirds of 2026 earnings per share expected to be recognized in the second half of the year due to order dynamics and integration effects.
  • Palynziq Label Expansion -- U.S. adolescent patient base expanded following February FDA approval for ages 12 and up, with broad uptake seen in new patient starts and momentum expected to continue.
  • Voxogo sNDA Status -- Submission for full approval has been completed, with FDA review timing expected to be communicated in the coming months.
  • Voxogo Clinical Evidence -- Long-term efficacy and safety demonstrated with more than 10,000 patient-years of exposure and sustained improvements in arm span, height, and bone health.
  • Pipeline Readouts -- Two pivotal data readouts expected in the second quarter: Voxogo for hypochondroplasia and BMN 401 for ENPP1 deficiency, with the latter’s ENERGY-3 study including two co-primary endpoints.
  • Amicus Acquisition Integration -- Integration initiated with planned commercial, diagnostic, and geographic expansion for Galafold and Pombility/Upholda, and further details on revenue synergies and peak sales to be provided in Q2.

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RISKS

  • Naglazyme Manufacturing Charge -- CFO Brian Mueller stated a "$31 million charge associated with an unsuccessful process qualification campaign to extend Naglazyme manufacturing capability" elevated cost of sales and temporarily reduced margins.
  • Amicus Acquisition EPS Impact -- CFO Brian Mueller said, "the acquisition of Amicus will be slightly dilutive for the full year 2026."
  • Geopolitical Uncertainty Allowance -- CFO Brian Mueller noted that guidance reflects "an allowance for a modest amount of disruption" in the Middle East.

SUMMARY

The addition of Amicus (NASDAQ: FOLD) and its therapies Galafold and Pombility/Upholda has led to an upward revision of BioMarin Pharmaceutical (BMRN +2.59%)'s full-year revenue guidance by $500 million at the midpoint and now targets approximately 20% growth for 2026. Management expects over 55% of revenue and the majority of earnings per share to be recognized in the second half of the year as Amicus products make their first contributions and order timing remains skewed. The integration plan aims for rapid commercial expansion of the new assets, with key performance metrics and synergies to be outlined next quarter. BioMarin Pharmaceutical is awaiting two pivotal readouts for Voxogo (hypochondroplasia) and BMN 401, signaling near-term catalysts impacting future growth and market positioning.

  • Integration of the Amicus business will account for only eight months in 2026, compressing the initial growth impact, but management cited high organic revenue performance for Galafold and Pombility/Upholda so far this year.
  • Cristin Hubbard stated, "are already seeing a 90% increase in the number of hypochondroplasia patients that we have identified, as well as a 70% reduction in the age." of diagnosis, indicating commercial readiness for anticipated Voxogo expansion.
  • BioMarin Pharmaceutical reiterated that Amicus acquisition synergies and peak revenue trajectories for Galafold and Pombility/Upholda will be quantified in the Q2 update, with peak potential possibly exceeding prior estimates.
  • Management has completed submission of the sNDA for Voxogo's full approval and expects pivotal data for pipeline programs to drive future growth and regulatory progress this year.

INDUSTRY GLOSSARY

  • Achondroplasia: A rare genetic disorder resulting in dwarfism due to FGFR3 mutations, targeted by therapies such as Voxogo.
  • ENPP1 Deficiency: A rare genetic condition affecting vascular, skeletal, and soft tissue systems, addressed by BMN 401.
  • sNDA (Supplemental New Drug Application): Regulatory submission seeking changes to the label or approved use of an existing therapy, such as expanding Voxogo's indications.
  • AGV (Annualized Growth Velocity): Measurement of height growth over one year, key endpoint in pediatric skeletal disorder clinical trials.
  • AUC (Area Under Curve): Pharmacokinetic measure of drug exposure over time, referenced for CNP analogs in trial design.
  • DEXA Scan: Imaging test measuring bone mineral density, used to assess outcomes in hypochondroplasia trials.
  • FGFR3: The gene coding for fibroblast growth factor receptor 3, mutated in skeletal dysplasias targeted by BioMarin Pharmaceutical drugs.
  • ITC (International Trade Commission): U.S. agency referenced in the context of IP enforcement regarding biologic competitors.
  • Energy-3 Study: Pivotal phase three clinical trial for BMN 401 in ENPP1 deficiency patients.

Full Conference Call Transcript

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 Pommability and Unfolda 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. 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 roadmap as well as more detail on BioMarin Pharmaceutical Inc.'s growth acceleration with the addition of Galafold and Ponbility and Opholder 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 Pongility and OptFolder to the portfolio. Moving to our skeletal conditions business unit, we saw strong patient demand for Boxogo, with new patient starts increasing across all regions in the first quarter. In the U.S., a majority of new patient starts were from the under age two cohort.

These results reflect our focused investments in increasing adoption of VoxoGo, particularly among younger patients. Building on our leadership in achondroplasia, we are pleased to have submitted the sNDA for full approval of VoxoGo. 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 VoxoGo 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 Oddfolder 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 Voxago. And as expected, those organic growth drivers were partly offset by order timing dynamics as well as lower revenue from Rocktadian, Kuvan, and royalties. Enzyme therapies revenue increased 6% year over year, led by growth in Vimizin, Naglazide, 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 12–18-year-old population gains momentum and patients continue to titrate to their maintenance dose. Oaxogo 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 Voxogo 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 transaction.

The 2025 development activities for Voxelgo for hypochondroplasia, EMN 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 VoxoGo, 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 Opholda, 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 Voxago, 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 our 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 Pommibility and Ophfolda 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.

And 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 Upholda 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? Thank you, Brian.

Cristin Hubbard: We were encouraged by the commercial execution across our portfolio so far this year, with strong patient demand across enzyme therapies and VoxoGo. We look forward to providing a more detailed commercial update on our plans to maximize the potential of both Galafold and Prombility and Uphold 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 Upholda 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 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, Maglazyme, 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 Voxsogo 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 VOXZOVO 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, SOLO 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 Voxago 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 Voxova 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 Voxsogo'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 Voxovu into hypochondroplasia. As we get closer to the phase three data and potential launch, our precommercialization 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 first half 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 Voxsogo 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 these sustained benefits of Voxogo 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 Voxago'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 Voxago's well-established long-term safety profile. Taken together, this extensive body of evidence reinforces Voxago'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 Voxogo 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 Voxago'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 Voxogo 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 buxogo 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 1 to 12 includes two co-primary endpoints. The first is change from baseline in plasma measured of 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 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 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 milligram per kilogram 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 maybe there is a roadmap for future growth acceleration. We were just wondering if you could help to comment—specifically for Pambulio and Napolda—what levers do 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, of course 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 again that in addition to growing bone length and stimulating endochondral bone formation, that bone is healthy and of a strength that we would like to see and, again, 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, so we mentioned that it is in the second quarter. We are eager to see those results. And again, I think 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 National Children's the kind of growth that certainly would meet the criteria for a statistically significant improvement that our study is developed to measure. And 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. And 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 have mentioned before, but I will 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 Amicus has really started to build momentum. But we feel that using BioMarin Pharmaceutical Inc.'s capability in this regard, we can really continue to drive that forward.

On the Pombility and OpFoldus side, that is more about a switching opportunity, and we really do 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. So we are really working on initiatives that, again, Amicus has started, and that is around starting new therapy, looking at patients that 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.

So again, 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.

Operator: We will move to our next question from Salveen Richter at Goldman Sachs.

Analyst: This is Tommy on for Salveen. Just a quick one on Voxsogo. 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 really does remain strong. Our enrollments in Q1 exceeded the average of the second half last year, and that trend continued into April. So as we see it now, we are really excited. As you know, we are very much focused on the 0–2 population based on the consensus guidelines. We are seeing real momentum there. Not only were our new patient starts weighted, with more than half of them being in that 0–2 population in the first quarter, but we are also seeing a reduction in the time we have from diagnosis to treatment in that population.

That has reduced by 10% already. So we know that the real focus therein is working. In addition, we are focused on the patients that are already on treatment and making sure that those patients for whom they are doing well on treatment understand the totality of the evidence that exists for Voxsogo and continue to have a positive experience in terms of the benefit. So we are very pleased with how Q1 went and look forward to continuing on that trajectory.

Operator: We will go next to Paul Matteis at Stifel.

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 in terms of 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. Appreciate the question. We wanted to highlight today having just closed the transaction a week ago a brief update on the integration to date, which after a week 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 the 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 PONBILITY and Upholda. But 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. So that is what we will share.

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

Operator: We will move next to Eliana Merle at Barclays.

Jasmine Alexandra Fels: Hi. This is Jasmine on for Ellie. Thank you so much for taking our question. So just for Voxelgo and hypochondroplasia, what is the latest on what you are thinking will be good data and what you are looking to see on AGV? And also, how are you thinking about the contribution to Voxoga 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—absolutely looking forward to that. Success is a statistically significant improvement in growth as compared to the control arm. In this world, of course, we are measuring a variety of other measures. We are looking at other anthropomorphic measures as well as the safety profile. Really, any statistically positive improvement in growth—we think that is a win for these patients.

I will just add that we have seen pretty dramatic accelerations of recruitment both for the older children as well as, as I mentioned in the prepared remarks, the infants in our hypochondroplasia study, really suggesting that this is a market that is 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, and so from that standpoint, and of course built upon the data that Dr. Dauber has seen, we are quite excited to see what those end results will be. I think there was a question also about the marketplace, and I am going to hand that one off to Cristin.

Cristin Hubbard: Thank you. Overall, our goal, of course, is to continue the growth of Voxsogo into the mid and hypochondroplasia provides an important component of that. Really where we are focused today on our prelaunch 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.

So our goal at this point in time prior to launch really is around increasing the number of hypochondroplasia patients that have been identified, getting physicians to understand the genetic testing and make sure that they are putting orders for that, and 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.

Gregory Friberg: And, Jasmine, if I could just add one other factor from the medical affairs standpoint, we are 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. There are probably three prongs to that: getting patients to be referred sooner to be evaluated; testing; as well as doing work on the genetic side to reclassify so-called VUSs, variants of uncertain significance. In that world, we are anticipating again that there will be patients available and ready for this therapy should it become available.

Cristin Hubbard: Just to throw out a few components, if I could, quickly. On the success we have had already when looking at the diagnosis program that we are running to date and these non–prelaunch activities, we are already seeing a 90% increase in the number of hypochondroplasia patients that we have identified, as well as a 70% reduction in the age. Those are precisely the types of numbers that we are driving for and want to continue throughout our launch period.

Operator: We will move to our next question from Cory Kasimov at Evercore.

Adithya Jayaraman: 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 Pommop? Is that conservative relative to the $600 million revenue that was generated in fiscal year 2025? And what are the key assumptions for adding these two assets into the guidance?

Brian Mueller: Hi, Adi. 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. I will frame it up for you. First of all, the $500 million represents a midpoint of a range that fits within the range of our existing enzyme therapies guide for 2026, but I will 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. In terms of your question around conservatism, I would say it is neither conservative nor aggressive. We think it is realistic.

We have 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. So both Galafold and Palmop together are off to a strong start in 2026. With that being said, again, the transaction closed just a week ago. We will come back next quarter with additional color, including the long-term potential. I will also share that 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 twenties.

We think it is really healthy growth, and we are excited that these assets are now part of our portfolio. Again, you see this 30% increase year over year as a result of layering this onto the BioMarin Pharmaceutical Inc. enzyme therapies, and total revenue is 20% at the midpoint. We are feeling good about it, and we will share more going forward.

Operator: We will go next to Jessica Fye at JPMorgan.

Analyst: This is Jose on for Jess. Thanks for taking our questions. How should we think about the adoption of Buxolvo 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 will address the first part of the question. In terms of uptake going in, I think we can consider it being similar to that of achondroplasia, but as I mentioned 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 are 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, but importantly that we continue on with that momentum because that ultimately is going to be what drives the shape of the adoption curve.

Gregory Friberg: And, 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 diagnosed a bit later. They are referred and diagnosed later. So 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 are doing to get these children in the hands of the right physicians who know what to do for them seems like a real early success.

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, Greg and Cristin. I will 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 are a couple of different layers to that, both of which we discussed last quarter, by the way. One was large international orders that were processed in Q4 that just did not recur in Q1. Then the second 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 Voxova.

It was not just about an extra week of inventory, but it was enough to show up as variance in the quarter-over-quarter revenue. This is why we wanted to emphasize the underlying patient demand growth. There are no significant price drivers in there, by the way. It is an entire order timing dynamic.

Operator: We will move next to Phil Nadeau at TD Cowen.

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, we are curious to get your thoughts on the ITC pretrial brief that was recently posted online? That is first. And then second, in the press release, there is a note that there is going to be some data from BMN 333's phase 2/3 trial in 2027. Could you provide a little bit more detail around what data will be released at that time?

Alexander Hardy: Thanks very much, Phil. With regard to the ITC, 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. 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' product infringes our patent on or about August 21. At that point, if the full commission decides to review the decision, then the final decision is expected on or about December 21. This is where we are and what we can expect going forward.

I will also add that 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 now hand it over to Greg.

Gregory Friberg: Thanks for the question, Phil, about BMN 333. As a reminder for those on the call, this is our long-acting CNP analog to release continuous, potentially higher AUC exposures of CNP when administered on a weekly basis. As a reminder, in our phase one we saw over 10x increases in the AUC levels that we were able to achieve safely in the healthy volunteers. We have recently initiated our phase 2/3 study in children with achondroplasia. This is a multi-regional clinical trial. It is currently open in a variety of countries around the world.

We are enrolling as we speak, and our goal is to run the phase two portion where one of three doses of BMN 333 will be administered to the children, and there will be one arm that has Voxogo as well. There is no placebo in 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 will be using that data along with other anthropomorphic measures and 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 three to run head to head against Foxogo, 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 are all familiar with.

Operator: We will go next to Chris Raymond at Raymond James.

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 maybe in a more pointed way on the decision to go with a superiority trial versus noninferiority. I think I have heard what you guys have said around 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 Voxsogo. I will highlight that the 3x target was an at least 3x. We are 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 will also add that from a noninferiority standpoint, it is a natural question to ask, would this be easier? From a pure mathematical standpoint, it is actually much harder, and the study would be upwards of 10 times the size in order to show noninferiority versus even a drug like Voxago.

There is a practicality of designing the study. I want to reassure you that we will have an opportunity to look at the data after our phase two portion, and if there need to be adjustments—as 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.

Alexander Hardy: And I would just add, as Greg said, we think the opportunity and the need here is really around superiority in terms of efficacy—AGV and all the benefits beyond linear growth. Also, by this design of a study, we actually have an active control with VoxoGo. So the size of the study is smaller than it would be if it was a noninferiority design, as Greg has already covered. We also think the proposition to both caregivers and physicians means that this should be a study that is very attractive to potential patients to sign up to be included. Therefore, speed of recruitment—which is extremely important.

We think that the superiority profile we are aiming for from an efficacy standpoint together with rapid trial recruitment and milestones being achieved is really a compelling proposition.

Gregory Friberg: Placebo-controlled studies, when there are active safe and effective therapies, are no longer really possible to run. It is not the right thing to do either.

Operator: We will take our next question from Mohit Bansal at Wells Fargo.

Analyst: Great. This is Chen Shui from Mohit. Thank you for taking our question. I just want to double click on the development landscape a little bit. I think you guys discussed that BMN 333 is targeting improvement in achondroplasia. I am just wondering whether a competitor that is also advancing their weekly CNP analog together with growth hormone, potentially reporting a higher AGV—just how should we think about BioMarin Pharmaceutical Inc.'s view of the evolving treatment landscape and the role that 333 could potentially play over time? Thank you.

Gregory Friberg: Thanks for the question. It is true: I think the data in combination with growth hormone, of which we have seen about 12 months of data, does show in the early studies that there is potentially additional growth to be had by adding in a second agent. But I want to caution you that 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 for that is that growth that can be 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. That remains an open question. I think we do need to see data at 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—but we need to see whether or not those gains are long-standing.

I want to reassure you that this is something that we are watching very closely, and 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 do not feel that is the correct time right now, and we are certainly interested in seeing additional data before the paradigm shifts. This is consistent with what we have heard from many of our stakeholders as well.

Operator: We will move to our next question from Joe Schwartz at Leerink Partners.

Joseph Schwartz: 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 increase by $500 million, and we estimate that Amicus generated around $450 million in revenue for Galafold and Palmop 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. So I am just wondering if you can help us reconcile that difference. Thanks.

Brian Mueller: Thanks, Joe. Appreciate the question and your math there. I would probably attribute the reconciliation difference that you are trying to get at with two elements. One, by doing a pro rata eight months of 2025, there are missing variables there and a lack of precision such that we could not do a strict apples-to-apples comparison. Secondly, on a full-year basis—again, I pointed to that strong performance for the first four months of the year for both Galafold and PONVILITY UpholdUp—while not reported, this is internal management data.

We are not disclosing it because it did not go through the usual external reporting process like a regular quarter does, but we thought it was important to add color to that full year. I would say it is more important, and this goes to our own business when we think about some of these quarterly timing dynamics, to compare the full-year annual cycle year over year. That is where, when we piece together the range that is implied today over that full-year $634 million that Amicus reported for 2025, we have a range of high teens to low twenties. I encourage you to anchor to that rather than trying to calculate the intra-quarter math.

Operator: We will move next to Jay Gerberry at Bank of America.

Jason Matthew Gerberry: Alex, just to follow up on the ITC question. I am trying to get my head around whether you have any knowledge of Ascendis' ability to do a manufacturing workaround and help us think through scenarios if you get a positive ruling. Could a decision be stayed pending any sort of appeals? And then just as a follow-up on Palmop, I believe you have 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 have got 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 or the potential scenarios around it. I hope you can understand that we do not want to get into too many specifics, especially whilst the case is very much pending. I will hand it over now to Cristin for the second part.

Cristin Hubbard: Thank you very much for the question around Palmop. You are absolutely correct that currently we are reimbursed in 15 countries. Before the transaction was closed, we were doing significant and deep-dive discovery sessions, really looking at the business in each market for both Galafold and Palm Op, understanding what the dynamics are therein, but then importantly looking at, as you have mentioned, our 80-country footprint and identifying where the potential and the opportunity would be. We are identifying opportunities for both Galafold as well as PAMA. Given that PALMoP 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 we will share more of in 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, as you can tell, marks a really important inflection point for BioMarin Pharmaceutical Inc. The recent close of the Amicus acquisition expands 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 VoxoGo, 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.