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Valoctocogene roxaparvovec is an investigational AAV5 gene therapy for the treatment of severe hemophilia A. It has been approved for conditional use in the European Union and is marketed as ROCTAVIAN™ (valoctocogene roxaparvovec). It is not approved for use in the United States.

Hemophilia A is a genetic disease caused by the deficiency of clotting factor VIII. It is the most common type of hemophilia and occurs much more frequently in males; incidence is estimated at 1 in 4,000-5,000 male births.

People born with hemophilia produce little or no clotting factors. The two main types of hemophilia are A and B. People with hemophilia A are missing or have low levels of clotting factor VIII. People with hemophilia B are missing or have low levels of clotting factor IX. These proteins work with platelets in the clotting process. When blood vessels are injured, clotting factors help platelets stick together to plug cuts and breaks on the vessels and stop bleeding. Many patients with hemophilia experience spontaneous bleeding events that result in progressive and debilitating joint damage.

The current standard of care for most hemophilia A patients who are severely affected today is a prophylactic regimen of intravenous infusions three times per week. Even with prophylactic regimens, many patients still experience spontaneous bleeding events.

Gene Therapy Research and Hemophilia A

Image: BioMarin scientists
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