BioMarin provides update on BLA for valoctocogene roxaparvovec AAV gene therapy

BioMarin Pharmaceutical announced a progress update on its Biologics License Application for valoctocogene roxaparvovec AAV gene therapy for adults with severe Hemophilia A that is currently under review by the U.S. FDA. As part of their review of the BLA, the FDA has scheduled its Pre-Licensure Inspection of BioMarin’s gene therapy manufacturing facility, located in Novato, CA. As anticipated, the FDA also has requested that the company submit results from the upcoming three-year data analysis from the ongoing Phase 3 GENEr8-1 Study. While the FDA did not communicate a change to the current PDUFA target action date of March 31, 2023, the Agency stated that submission of these results may qualify as a Major Amendment, which would extend the action date by 3 months. FDA will evaluate the additional data prior to making this determination.