First Gene Therapy for Hemophilia A in USA Approved by FDA—Outcomes Warranty Part of Commercialization

Publicly traded BioMarin Pharmaceutical Inc. (BMRN), dedicated to transforming lives through genetic discovery, reports that the United States Food and Drug Administration (FDA) approved 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 approval is based on the results of a Phase 3 GENEr8-3 study led by several trial sites, all academic medical centers, across multiple continents. The company offers an outcomes-based warranty to all U.S.-based payers as part of its commercialization scheme.

What is Hemophilia A?