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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