BioMarin Pharmaceutical announced that it received notice this afternoon from the U.S. Food and Drug Administration that the agency has extended review of the company’s Biologics License Application for ROCTAVIAN gene therapy for adults with severe hemophilia A. The FDA determined that the submission of the three-year data analysis from the ongoing Phase 3 GENEr8-1 study constituted a Major Amendment due to the substantial amount of additional data and set a new PDUFA Target Action Date of June 30, 2023. The company had previously communicated that this data submission could be qualified as a Major Amendment. The Phase 3 study, which included 134 participants, is the longest and largest to date for a gene therapy in hemophilia.
Published first on TheFly
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