FDA Gives Nod to Gene Therapy for Hemophilia A
The FDA approved valoctocogene roxaparvovec (Roctavian) as the first gene therapy for the treatment of adults with severe hemophilia A, drug developer Biomarin announced on Thursday. Patients with severe disease are eligible for the one-time, single-dose treatment if they don’t have antibodies to adeno-associated virus serotype 5 (AAV5), as detected by an FDA-approved test. FDA […]