One Rejected BioMarin's Hemophilia Gene Therapy Goes Under FDA Review

  • The FDA accepted BioMarin Pharmaceutical Inc's BMRN resubmission of the marketing application for its investigational AAV gene therapy, valoctocogene roxaparvovec, for severe hemophilia 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. to treat severe hemophilia A.
  • The European Commission granted conditional marketing authorization to valoctocogene roxaparvovec gene therapy under the brand name Roctavian.
  • The BLA resubmission incorporates the company's responses to all deficiencies identified in the FDA Complete Response Letter in August 2020, including two-year outcomes from all study participants in the global GENEr8-1 Phase 3 study. 
  • The GENEr8-1 Phase 3 study demonstrated stable and durable bleed control, including a reduction in the mean annualized bleeding rate and the mean annualized Factor VIII infusion rate. 
  • Price Action: BMRN shares are down 1.25% at $87.84 on the last check Thursday.
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