Innovative therapeutics


Valoctocogene Roxaparvovec (BMN 270) for Hemophilia A

Valoctocogene roxaparvovec is gene therapy designed for the treatment of Hemophilia A.

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.

BioMarin is developing gene therapy using an AAV-factor VIII vector, valoctocogene roxaparvovec, an investigational drug for the treatment of hemophilia A. In mouse models of hemophilia A, valoctocogene roxaparvovec restored factor VIII plasma concentrations to levels projected to be adequate for normal clotting in humans.

Gene therapy has the promise of delivering, in as little as a single administration, the missing gene needed to produce factor VIII. This provides a potential cure and may eliminate the need for ongoing treatments of factor VIII. The 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.

There are approximately 90,000 patients in territories where BioMarin has commercial operations.

Understanding Gene Therapy: Research for Hemophilia A

Gene Therapy and Hemophilia A

Valoctocogene roxaparvovec – Gene Therapy for Hemophilia A

Valoctocogene roxaparvovec – Gene Therapy for Hemophilia A

Click here to open the full-size pdf.



Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI, CLN2 disease) PKU, LEMS



Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361

Global Medical Information

Contact Global Medical Information