The U.S. Food and Drug Administration (FDA) has approved BioMarin’s ROCTAVIAN™ (valoctocogene roxaparvovec-rvox) gene therapy product for the treatment of adults with severe hemophilia A without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved diagnostic test. This companion test is designed to help health care providers identify patients who may benefit from receiving ROCTAVIAN. It effectively screens for pre-existing anti-AAV5 antibodies, which may render the therapy less effective or ineffective.

FDA approval was based largely on the positive durability, efficacy, and safety results from the global Phase 3 GENEr8-1 study, which has been fully enrolled since November 2019 with 134 participants ultimately receiving a one-time dose of therapy. Results showed an overall 82.9% reduction in treated bleeds and a 96.8% reduction in FVIII usage overall when compared to baseline data. Trial participants will continue to be closely monitored.

"Adults with severe hemophilia A face a lifelong burden, with frequent infusions and a high risk of health complications, including uncontrolled bleeding and irreversible joint damage," said Dr. Steven Pipe, professor of pediatrics and pathology at the University of Michigan and an investigator in the Phase 3 study.  "The approval of ROCTAVIAN, as the first gene therapy for severe hemophilia A, has the potential to transform the way we treat adults based on years of bleed control following a single, one-time infusion."

“Hereditary hemophilia A is a potentially serious bleeding disorder. Severe cases of hemophilia A can cause life-threatening health issues due to increased risk of uncontrolled bleeding,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research. “Today’s approval represents an important advance in providing treatment options for patients with this bleeding disorder, and treatment with gene therapy may reduce the need for ongoing routine therapy.”

Read the full FDA release for more context, including additional information on the safety and efficacy data that supported the approval.

Source: FDA news release, Cision (PR Newswire), both dated June 29, 2023