Roctavian (valoctocogene roxaparvovec)

A one-time gene therapy for severe hemophilia A, FDA-approved in June 2023 and voluntarily withdrawn from the market by BioMarin in February 2026. The withdrawal was a business decision and was not related to how well the therapy worked or how safe it was.

Roctavian uses a harmless virus (AAV5) to deliver a working copy of the Factor VIII gene into liver cells. Once inside, the liver cells read the new gene and begin producing Factor VIII protein on their own. This can reduce or eliminate the need for regular Factor VIII infusions.

Liver enzyme elevations are very common and require close monitoring. Headache, nausea, fatigue, and infusion-related reactions occur frequently. Immunosuppressive therapy (corticosteroids) may be needed to manage liver inflammation. Not recommended for patients with pre-existing AAV5 antibodies.

Given as a single one-time intravenous infusion. Requires pre-screening for AAV5 antibodies. Patients need close liver monitoring for at least a year after treatment, with corticosteroid management as needed.

The FDA approved Valoctocogene roxaparvovec (Roctavian) in 2023 for Hemophilia A, but BioMarin Pharmaceutical has since stopped selling it, so it is no longer available.

Five-year follow-up data from the GENEr8-1 trial showed average Factor VIII activity of about 24% of normal on one standard test (one-stage assay) and about 14% on another (chromogenic assay), well above the severe hemophilia threshold of less than 1%. Most patients reduced or eliminated Factor VIII prophylaxis, though factor levels may gradually decline over time.

No. BioMarin voluntarily withdrew Roctavian from the market in February 2026, so it is not available to new patients. When it was available, it was approved for adults with severe hemophilia A who did not have pre-existing antibodies to the AAV5 viral vector, and people with significant liver disease or active Factor VIII inhibitors were excluded. BioMarin said the withdrawal was a commercial decision, not a safety or effectiveness one.

The AAV5 vector delivers the Factor VIII gene to liver cells, which can trigger immune-mediated liver inflammation. Liver enzyme elevations are very common and may require corticosteroid treatment. Close monitoring for at least one year after infusion is essential to manage this response.

Roctavian treats hemophilia A (Factor VIII deficiency) while Hemgenix treats hemophilia B (Factor IX deficiency). Both use AAV5 vectors to deliver genes to the liver, but they target different clotting factors. They cannot be used interchangeably.

Roctavian provides sustained but potentially declining Factor VIII production over years. While it dramatically reduces or eliminates the need for factor infusions, it is described as a functional improvement rather than a permanent cure. Long-term data beyond 5 years is still being collected.

Roctavian is given as a single one-time intravenous infusion at a specialized treatment center. The infusion itself takes several hours. Patients then enter a monitoring phase with frequent blood tests and liver function checks, along with corticosteroid management as needed.

BioMarin has committed to continuing support for people already treated, including ongoing monitoring and follow-up care, and to honoring existing outcomes-based agreements with payers. If you were treated with Roctavian, keep up with your scheduled liver monitoring and talk with your hematologist. Providers with questions can reach BioMarin Medical Information at 1-800-983-4587.