BioMarin Pharmaceutical Inc. announced that it received notice from the 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."
Global Regulatory Status
The FDA completed a Pre-License Inspection of the manufacturing facility in early December 2022.
The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec in March 2021. RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, which are expected to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy designation, which the company received for valoctocogene roxaparvovec in 2017.
In addition to the RMAT designation and Breakthrough Therapy designation, BioMarin's valoctocogene roxaparvovec also received Orphan Drug designation from the European Medicines Agency (EMA) and FDA for the treatment of severe hemophilia A. Orphan Drug designation is reserved for medicines treating rare, life-threatening, or chronically debilitating diseases. The European Commission (EC) granted conditional marketing authorization to valoctocogene roxaparvovec gene therapy under the brand name ROCTAVIAN on August 24, 2022.
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