Zevra to appeal EMA negative opinion on Arimoclomol for Niemann-Pick disease type C

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.