FDA Approves BioMarin's Gene Therapy for Severe Hemophilia A
The FDA has approved Roctavian, the first gene therapy for adults with severe hemophilia A. Hemophilia A is a rare genetic bleeding disorder caused by a deficiency in clotting factor VIII. Roctavian is a one-time gene therapy administered through intravenous infusion, which increases blood levels of factor VIII to reduce the risk of uncontrolled bleeding. The safety and effectiveness of Roctavian were evaluated in a study of adult men with severe hemophilia A, and it was found to significantly reduce bleeding rates. Common side effects include mild changes in liver function, headache, nausea, and fatigue. The approval of Roctavian provides a new treatment option for patients with severe hemophilia A.