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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.

Valoctocogene roxaparvovec, is an investigational gene therapy in clinical trials for the treatment of Hemophilia A and has not been determined to be safe or effective. Valoctocogene roxaparvovec is administered as a single infusion. The ongoing clinical trials will determine if the new gene will enable the body to produce factor VIII. Following infusion, clinical trial subjects are being evaluated to determine the safety profile, changes in Factor VIII activity levels, changes in factor replacement usage, changes in reported bleeds requiring factor replacement, and quality of life measures, among other endpoints. 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.

Understanding Gene Therapy: Research for Hemophilia A

Gene Therapy and Hemophilia A

BioMarin

Diseases

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

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Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361
E-mail: support@biomarin-rareconnections.com

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