FDA Approves BioMarin’s Roctavian, the First Gene Therapy for Treating Severe Hemophilia A

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FDA has approved Roctavian (valoctocogene roxaparvovec-rvox), a gene therapy from BioMarin Pharmaceutical for treating severe hemophilia A in adults.

On June 29, 2023, FDA announced that it approved Roctavian (valoctocogene roxaparvovec-rvox), an adeno-associated virus vector-based gene therapy from BioMarin Pharmaceutical for treating severe hemophilia A. The therapy is indicated for adults without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test.

According to an agency press release, FDA’s decision marks the first approval for a gene therapy to treat adults with severe hemophilia A. Hemophilia A is a rare genetic bleeding disorder that primarily affects males and occurs when there is a mutation in the gene that produces factor VIII (FVIII), a blood-clotting protein. Severe hemophilia A manifests when there are especially low levels of FVIII (less than 1% in the blood); according to the release, this disorder represents approximately 60% of all cases.

Valoctocogene roxaparvovec-rvox is a one-time, single-dose gene therapy (viral vector carrying a gene for FVIII) that is given intravenously. It is designed to replace the function of the mutated gene, thus allowing severe hemophilia A patients to produce their own FVIII and limit bleeding episodes, according to a BioMarin company press release.

“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, director of the FDA’s Center for Biologics Evaluation and Research, in the agency’s press release. “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.”


According to BioMarin’s press release, valoctocogene roxaparvovec-rvox will be manufactured at the company's facility in Novato, Calif., from where BioMarin expects to meet commercial demand throughout the product’s lifecycle.

Source: FDA and BioMarin Pharmaceutical