FDA accepts BioMarin's Biologics License Application (BLA) for valoctocogene roxaparvovec AAV gene therapy for adults with severe haemophilia A

13 October 2022


BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) has announced that the U.S. Food and Drug Administration (FDA) accepted the Company’s resubmission of the Biologics License Application (BLA) for its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with severe haemophilia A. The Prescription Drug User Fee Act (PDUFA) target action date is March 31, 2023. At this time, the FDA has not communicated any plans to hold an advisory committee meeting. If approved, valoctocogene roxaparvovec would be the first gene therapy in the U.S. for the treatment of severe haemophilia A.

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