FDA accepts Sarepta's sNDAs for AMONDYS 45 and VYONDYS 53 with February 2027 target action date

BioMarin Pharmaceutical reported positive Phase 3 CANOPY-HCH-3 results for VOXZOGO in children with hypochondroplasia and submitted a supplemental FDA application to expand the drug's use, while also adding Bristol Myers Squibb research leader Robert Plenge, M.D., Ph.D., to its Board and Science and Technology Committee. The late-stage data cover an indication with no approved treatments, and the company said the combination of the results and the board appointment underscores its focus on deepening its rare disease franchise and scientific oversight. BioMarin's narrative projects $5.0 billion revenue and $1.3 billion earnings by 2029, requiring 14.0% yearly revenue growth and about a $1.2 billion earnings increase from $73.0 million today, with a $91.42 fair value cited, a 43% upside to its current price. More pessimistic analysts warn that heavy reliance on a few rare disease drugs and uncertainty around future indications could leave BioMarin short of pre-news expectations of about US$4.7 billion in revenue and roughly US$509.5 million in earnings by 2029.

BioMarin Pharmaceutical said it is progressing with the integration of Amicus Therapeutics and expects $200 million in annual non-GAAP cost savings, with most synergies realized in 2027 and full realization in 2028. Speaking at Morgan Stanley's Global Healthcare Conference, President and CEO Alexander Hardy said Amicus had about 505 employees and BioMarin expects to retain approximately 192 over the long term, with about 70% of the savings coming from general and administrative expenses and the remainder from duplicated research and development functions. Chief Financial Officer Brian Mueller said the deal should be accretive in its first year, with substantial accretion beginning next year, and that leverage is now expected to fall below 2.5 times by the middle of 2027, nearly a year earlier than the initial target. Hardy said VOXZOGO delivered 20% quarterly patient growth despite U.S. competition and is expected to reach $1 billion in revenue this year, which would make it BioMarin's first blockbuster product, while a potential FDA approval in hypochondroplasia is not incorporated into its 2026 outlook. BioMarin projects peak sales of approximately $1.4 billion for GALAFOLD and $1.2 billion for POMBILITI + OPFOLDA by the mid-2030s, and said PALYNZIQ grew 27% to $135 million while VIMIZIM has the potential to exceed $1 billion in revenue.

AstraZeneca said the FDA accepted its regulatory filing for the investigational enzyme replacement therapy efzimfotase alfa in patients aged two years and older with hypophosphatasia, or HPP, and granted the application priority review, shortening the review period by four months, with a final decision expected during the first half of 2027. The filing is supported by data from three phase III studies: MULBERRY met its primary endpoint in treatment-naive children aged two to less than 12 years old, showing a significant improvement in bone health, and CHESTNUT showed that patients switching from Strensiq could maintain the benefits of treatment, while HICKORY did not meet its primary endpoint in treatment-naive individuals aged 12 years and older, though AstraZeneca reported a numerical improvement, particularly in patients with pediatric-onset disease. The company said efzimfotase alfa was generally well-tolerated with an acceptable safety profile across the three studies. The commercial angle rests on Strensiq, the established HPP treatment AstraZeneca gained through its 2021 acquisition of Alexion, which was approved by the FDA in 2015 as the first bone-targeted enzyme replacement therapy for HPP and generated $1.05 billion in sales in the first half of 2026, up 41% year over year. Efzimfotase alfa is designed to require lower injection volumes and substantially less frequent dosing, once every two weeks, compared with Strensiq's three- or six-times-weekly regimen. Elsewhere in the space, BioMarin Pharmaceutical entered HPP through its acquisition of Alesta Therapeutics, completed earlier this month, paying $275 million upfront with up to $215 million in additional milestone payments for ALE1, an oral small-molecule therapy in a phase I/IIa study, while Recursion Pharmaceuticals is developing the oral ENPP1 inhibitor REC-102, formerly REV102, which remains in IND-enabling studies with a data-driven decision on a phase I study expected before the end of this year.