FDA Grants Priority Review to BridgeBio's Dwarfism Drug Filing
BBIO wins FDA priority review for infigratinib, potentially making it the first approved oral therapy for achondroplasia.
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BioMarin develops and commercializes therapies for rare diseases, including enzyme replacements, gene therapies, and treatments for conditions like MPS, PKU, and hemophilia, serving global markets through collaborations and specialty distribution.

Voxzogo is a daily injection approved for increasing growth in children with achondroplasia, driving BioMarin's revenue growth through strong market adoption and strategic commercial focus.
In its most significant M&A activity to date, BioMarin Pharmaceutical Inc. entered into a definitive agreement to acquire fellow rare disease drugmaker Amicus Therapeutics for approximately $4.8 billion in an all-cash transaction. This strategic move is intended to accelerate BioMarin's revenue growth and strengthen its portfolio with the addition of two growing products, Galafold for Fabry disease and Pombiliti-Opfolda for Pompe disease.
In a major strategic move to accelerate growth and diversify its revenue, BioMarin announced its agreement to acquire Amicus Therapeutics. The deal significantly expands BioMarin's portfolio with two marketed therapies for Fabry and Pompe diseases, strengthening its position as a global leader in treatments for rare genetic conditions.
BBIO wins FDA priority review for infigratinib, potentially making it the first approved oral therapy for achondroplasia.
Ms. Bauer Brings More Than 25 Years of Experience in Human Resources, Across Biotechnology and Technology Industries Appointment Effective Oct. 5, 2026 SAN RAFAEL, Calif., Oct. 5, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that Cara Bauer, Ph.D.
A BioMarin combination therapy helped adults with late-onset Pompe disease maintain walking distance over five years of continuous therapy. Respiratory function remained relatively stable, with no new safety signals identified.
Long-term data reinforce durable efficacy and safety of POMBILITI + OPFOLDA in late-onset Pompe disease SAN RAFAEL, Calif., Oct. 2, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new data evaluating the efficacy and safety of POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) in late-onset Pompe disease (LOPD) were presented at the 31st Annual Congress of the World Muscle Society (WMS), in Hiroshima, Japan.
Overall rating: B
| PEER | PRICE | |
|---|---|---|
VRTX | $504.43 | COMPARE |
MRNA | $189.85 | COMPARE |
RVMD | $198.93 | COMPARE |
ALNY | $222.39 | COMPARE |
INSM | $99.22 | COMPARE |
EXEL | $57.94 | COMPARE |
ASND | $224.44 | COMPARE |
HALO | $110.28 | COMPARE |