The US Food and Drug Administration (FDA) has extended its Biologics License Application review of valoctocogene roxaparvovec (Roctavian), a novel gene therapy for individuals with severe hemophilia A, BioMarin Pharmaceutical announced.

The announcement noted that the FDA determined that the 3-year data analysis from the phase 3 GENEr8-1 clinical trial represented a major amendment and required a new Prescription Drug User Fee Act (PDUFA) date of June 30, 2023.

“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,” Hank Fuchs, MD, president of worldwide research and development at BioMarin, said in a press release. “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.”

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Hemophilia A is an X-linked hereditary condition caused by missing or defective factor VIII, a blood clotting protein. It can be passed from parents to children or caused by a spontaneous genetic mutation.

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Patients with hemophilia A are at risk of life-threatening bleeding from even minor injuries, and they currently require prophylactic intravenous infusions of factor VIII 2 to 3 times per week or treatment with a bispecific monoclonal antibody factor VIII replacement compound 1 to 4 times per month. Even with these regimens, many individuals with hemophilia A experience breakthrough bleeds and progressive joint damage.

Valoctocogene roxaparvovec is an investigational adeno-associated virus type 5 (AAV5)-based gene therapy that encodes human factor VIII for the treatment of patients with severe hemophilia A. The FDA granted valoctocogene roxaparvovec Regenerative Medicine Advanced Therapy designation in March 2021, in addition to Breakthrough Therapy designation in 2017.

BioMarin currently has several ongoing clinical trials on treatments for severe hemophilia A, including in patients with and without inhibitors.


BioMarin provides update on FDA review of Roctavian (valoctocogene roxaparvovec) gene therapy for adults with severe hemophilia A. News release. BioMarin Pharmaceutical Inc; March 6, 2023.