BioMarin Pharmaceutical announced the US Food and Drug Administration’s (FDA) approval of valoctocogene roxaparvovec-rvox (Roctavian), the first gene therapy available for adults with severe hemophilia A.

The approval, announced via news release, means that the one-time, single-dose infusion approved by the European Medicines Agency in August 2022 will now be available in the United States.

“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.”

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Hemophilia A is a rare hereditary disease caused by a mutation in the gene that produces FVIII, a protein required for blood clotting. Patients with hemophilia experience painful and potentially life-threatening bleeds and typically require life-long infusions of clotting factors at regular intervals.

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Valoctocogene roxaparvovec-rvox is an adeno-associated virus vector-based therapy that delivers functional copies of the gene that produces FVIII, enabling the body to produce sufficient levels of FVIII and eliminating the need for ongoing infusions.

The FDA approval is based on the results of the global phase 3 GENEr8-1 trial, in which 134 patients received valoctocogene roxaparvovec-rvox and were followed for at least 3 years. The patients with baseline annualized bleeding rates experienced a 52% reduction in bleeds after the treatment compared with baseline.

The patients also reported a notable reduction in spontaneous bleeds and joint bleeds, and most patients have continued to respond to the therapy through year 3 without needing supplemental prophylaxis.


U.S. Food and Drug Administration approves BioMarin’s ROCTAVIAN™ (valoctocogene roxaparvovec-rvox), the first and only gene therapy for adults with severe hemophilia A. News release. BioMarin Pharmaceutical, Inc.; June 29, 2023.