FDA Approves First Gene Therapy for Severe Hemophilia A
BioMarin Pharmaceutical Inc., a global biotechnology company dedicated to transforming lives through genetic discovery, announced that the US Food and Drug Administration (FDA) has approved ROCTAVIAN (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity of 1 IU/dL) without antibodies to adeno-associated virus. The one-time, single-dose infusion is the first gene therapy licensed in the United States for severe hemophilia A. ROCTAVIAN was approved by the European Medicines Agency in August 2022.
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