BioMarin Pharmaceutical Q3 Earnings Call Highlights

Key Points

• Q3 revenue topped $1 billion, rising 32% year over year, with metabolic-condition sales up 44%. Growth was led by Voxzogo, Galafold and Pombiliti + Opfolda, supported by broader rare-disease portfolio demand.
• BioMarin lowered 2026 revenue guidance to $3.8 billion–$3.9 billion and adjusted EPS guidance to $2.80–$3.10, citing roughly $70 million in deferred international orders and regional cash-flow disruption rather than weaker underlying demand.
• The company expanded its pipeline with BMN 450, an investigational long-acting Fabry disease enzyme therapy, and highlighted positive five-year data for Pombiliti + Opfolda. Management also expects Amicus integration synergies to reach approximately $220 million by 2028.

BioMarin Pharmaceutical NASDAQ: BMRN reported third-quarter revenue above $1 billion for the first time, driven by growth across its rare-disease portfolio and contributions from acquired Amicus Therapeutics products. The company said revenue increased 32% year over year, while its metabolic conditions business grew 44%.

Chief Executive Officer Alexander Hardy said the quarter reflected strong demand across BioMarin’s diversified portfolio, with Galafold and Pombiliti + Opfolda contributing more than $200 million of combined revenue. The company also added a new investigational Fabry disease program, BMN 450, to its clinical pipeline, its fourth new asset added during 2026.

Commercial portfolio growth

Chief Commercial Officer Cristin Hubbard said Galafold generated $152 million in third-quarter revenue, up 10% from the $138 million reported by Amicus in the third quarter of 2025. The number of patients receiving Galafold increased about 13% year over year.

Pombiliti + Opfolda revenue rose 58% to $49 million, while the number of treated patients increased approximately 69% year over year. Hubbard said treatment switching has been supported by the therapy’s clinical profile and an expanding body of clinical and real-world evidence. The product has become the market-share leader by patients on therapy in four European markets, she said.

BioMarin also began supporting some European Pompe disease patients affected by supply constraints for other therapies. Hubbard said those patients represented a meaningful share of new Pombiliti + Opfolda starts in certain markets during the quarter, though the company could not predict how long competing supply constraints would last or whether they would produce material revenue gains. Revenue from recent new starts is expected to build more materially in 2027 and beyond.

Elsewhere in the metabolic portfolio, Palynziq patient numbers increased 9% globally, supported by the U.S. adolescent launch, though revenue grew 2% as U.S. ordering timing normalized after a strong second quarter. Vimizim revenue rose 18%, benefiting from international orders, while Naglazyme revenue declined as expected following order-timing benefits in the prior quarter.

Voxzogo revenue increased 12% year over year to $245 million despite a large order that had been expected in the third quarter shifting into the fourth quarter. The number of children treated globally rose about 17%, and more than 75% of Voxzogo revenue came from outside the United States.

BioMarin said it received approval in three additional international markets for Voxzogo in achondroplasia. The company expects Voxzogo to continue growing in 2027, supported by international expansion, deeper penetration in existing markets and a planned hypochondroplasia launch that could offset some potential competitive share loss.

Guidance reduced on order timing and regional disruption

Chief Financial Officer Brian Mueller said BioMarin lowered its full-year 2026 revenue guidance to $3.8 billion to $3.9 billion. The update was driven by expected timing of orders rather than changes in underlying patient demand.

A single international customer indicated it intended to defer several anticipated orders totaling approximately $70 million. BioMarin expects to fulfill most of those orders between late in the fourth quarter and the first quarter of 2027, although Mueller said the precise timing remains uncertain. More than half of the affected $70 million in orders relates to Voxzogo, he said.

The company also cited increased customer liquidity and cash-flow disruption related to the prolonged Middle East conflict. Mueller said BioMarin had managed moderate disruption earlier in the year, but the impact had increased over the preceding two months.

BioMarin now expects 2026 non-GAAP diluted earnings per share of $2.80 to $3.10. The outlook includes the impacts of its recent Alesta acquisition and BMN 450 license agreement, as well as the revised revenue guidance. The Alesta deal is expected to reduce full-year non-GAAP diluted earnings per share by about $1.55, while the BMN 450 agreement is expected to reduce it by about $0.32.

The company reported non-GAAP diluted earnings per share of $0.23 in the third quarter, reflecting acquired in-process research and development charges from the Alesta acquisition. Third-quarter results included approximately $290 million in pre-tax expenses related primarily to acquired research and development.

Pipeline additions and clinical updates

BioMarin said BMN 450 is a phase I/II, long-acting enzyme replacement therapy for Fabry disease that could become the first subcutaneous enzyme therapy for the condition. The company believes the program could serve patients whose genetic variants are not amenable to Galafold and could potentially be mutation agnostic. BioMarin will assume responsibility for future development, manufacturing and commercialization outside Korea after the ongoing study.

The company also highlighted BMN 440, an oral investigational treatment for hypophosphatasia acquired through Alesta. BMN 440 is in a phase I/IIa study in healthy volunteers and adults with hypophosphatasia. BioMarin expects 2027 data on pharmacokinetics, pharmacodynamics and safety that could establish initial human proof of mechanism and help select doses for a subsequent study.

At the World Muscle Society Congress, BioMarin presented five-year open-label extension data for Pombiliti + Opfolda in 82 adults with late-onset Pompe disease. Chief Research & Development Officer Greg Friberg said motor function was maintained and pulmonary function remained relatively stable over five years, with no new safety signals reported.

BioMarin continues preparing for a potential Voxzogo launch in hypochondroplasia. Hardy said the product’s PDUFA date for that indication is May 30, 2027. The company estimates the global addressable population at approximately 14,000 children and said it is focusing on disease education, patient identification and payer engagement ahead of a potential launch.

Management said Amicus integration cost synergies are ahead of schedule. BioMarin continues to target approximately $220 million in non-GAAP cost synergies fully realized in 2028, while Mueller said the acquisition is now expected to become accretive within nine months of closing.

About BioMarin Pharmaceutical (NASDAQ:BMRN)

BioMarin Pharmaceutical Inc is a biotechnology company focused on developing and commercializing therapies for serious and life-threatening rare diseases. The company's research and development efforts center on genetic and metabolic disorders, where patient populations are often small and treatment options are limited.

BioMarin's marketed products include Voxzogo (vosoritide) for children with achondroplasia and open growth plates, Palynziq (pegvaliase) and Kuvan (sapropterin dihydrochloride) for phenylketonuria, Brineura (cerliponase alfa) for late-infantile neuronal ceroid lipofuscinosis type 2, Vimizim (elosulfase alfa) for Morquio A syndrome, and Naglazyme (galsulfase) for mucopolysaccharidosis VI.

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