BioMarin posts first $1 billion quarter, sets VOXZOGO PDUFA date for hypochondroplasia

BioMarin Pharmaceutical reported third quarter 2026 results on November 2, 2026, for the period ended September 30, 2026. The FDA set a PDUFA target action date of May 30, 2027 for BioMarin's supplemental NDA to expand VOXZOGO into hypochondroplasia (HCH), which the company called the first potential targeted therapy for the condition.¹ The European Medicines Agency also validated BioMarin's submission for VOXZOGO in HCH.¹

On the existing VOXZOGO indication, the application for full approval in children with achondroplasia remains under FDA review with a PDUFA date of February 28, 2027, and includes long-term safety and efficacy data such as final adult height.¹ The Phase 2/3 study of BMN 333, a long-acting CNP candidate for achondroplasia, continues to enroll, with dose-response data expected in 2027.¹

Pipeline additions were a theme. BMN 450, an investigational long-acting enzyme replacement therapy with potential to be the first subcutaneous ERT for Fabry disease, is now in a global Phase 1/2 study.¹ BMN 440 for hypophosphatasia, a potential first-in-class oral therapy, is in a Phase 1/2a study in healthy volunteers and HPP patients, with data expected in 2027.¹ BMN 820 for FSGS, a first-in-class oral CCR2 inhibitor under BioMarin's exclusive U.S. commercialization rights, has a fully enrolled Phase 3 ACTION 3 trial with pivotal data expected in 2028.¹

On other clinical updates, five-year PROPEL open-label extension results for POMBILITI + OPFOLDA, presented at the World Muscle Society Congress in October, showed durable motor function and relative pulmonary stabilization in late-onset Pompe disease patients, including those with a mean 7.6 years of prior enzyme replacement therapy, with no new safety signals.¹ For BMN 351 in Duchenne muscular dystrophy, the company expects a program update by year-end.¹

Financially, total revenue was $1,025 million, up 32% year over year, driven largely by GALAFOLD and POMBILITI + OPFOLDA, acquired April 27, 2026, and 18% growth in VIMIZIM.¹ More than 75% of VOXZOGO revenue came from outside the U.S., and a large international order previously expected in Q3 is now anticipated in Q4.¹ Full-year guidance was revised: total revenue, metabolic conditions, and VOXZOGO guidance were updated to reflect uncertainty in the timing of multiple orders from a single international customer.¹ BioMarin also said the Alesta acquisition and BMN 450 license agreement are expected to result in pre-tax charges of about $320 million and $83 million, respectively, for full-year 2026.¹