The U.S. FDA approved BioMarin Pharmaceutical Inc.’s Roctavian (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test.
The one-time, single-dose infusion is the first approved gene therapy for severe hemophilia A in the U.S. Roctavian was first approved by the European Medicines Agency in August 2022.
The FDA approval is based on data from the global Phase 3 GENEr8-1 study, the largest Phase 3 trial of any gene therapy in hemophilia.
