BioMarin axes $270M asset after missing phase 3 endpoint
BioMarin axes $270M asset after missing phase 3 endpoint
BioMarin Pharmaceutical
BioMarin Pharmaceutical is a global biotechnology company focused on developing and commercializing therapies for serious rare diseases. Its commercial portfolio includes Vimizim, Naglazyme, Aldurazyme, Brineura, and Voxzogo (vosoritide) for achondroplasia. The company's pipeline spans enzyme replacement therapies, gene therapy (Roctavian for hemophilia A), and skeletal-disorder treatments.
Company Info
Sector:Rare-disease biotechnology
Headquarters:San Rafael, California, USA
Founded:1997
Website:www.biomarin.com
Recent News
U.S. Food and Drug Administration Accepts BioMarin's Supplemental New Drug Application for Full Approval of VOXZOGO® (vosoritide) for Children With Achondroplasia
Application based on long-term safety and efficacy data from three ongoing studies, including adult height and additional clinical outcomes beyond linear growth, including body proportionality and arm span evaluated over long-term follow-up FDA PDUFA target action date of Feb. 28, 2027 SAN RAFAEL, Calif., July 13, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted the company's supplemental New Drug Appli
Leadership Team
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CEO
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