BioMarin Provides Update on FDA Review of ROCTAVIAN™ (Valoctocogene Roxaparvovec) Gene Therapy for Adults with Severe Hemophilia A

BioMarin Pharmaceutical, a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that it received notice this afternoon from the United States Food and Drug Administration (FDA) that the agency has extended review of the company’s Biologics License Application (BLA) for ROCTAVIAN™ (valoctocogene roxaparvovec) 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.

“We are continuing to work closely with FDA and appreciate the agency’s active engagement as we seek to deliver this important therapy to patients with severe hemophilia A,” said Hank Fuchs, M.D., president of Worldwide Research and Development of BioMarin. “The three-year data enhance our application and further reinforce our belief that ROCTAVIAN has the potential to fundamentally transform care for people with hemophilia A.”