FDA Approves Roctavian (valoctocogene roxaparvovec-rvox) Gene Therapy for Adults with Severe Hemophilia A
BioMarin Pharmaceutical Inc., announced 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. 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. Safety results for 134 patients have been reported over three years, demonstrating that ROCTAVIAN was well-tolerated.
Hemophilia A is a lifelong, genetic condition caused by a mutation in the gene responsible for producing a protein called FVIII, which is necessary for blood clotting. When severely deficient in amount, the condition puts people with hemophilia A at risk for painful and potentially life-threatening bleeds, which can occur spontaneously. With the current standard of care, individuals undergo lifelong preventative therapy, receiving infusions or injections at burdensome routine intervals to maintain enough clotting factor in the bloodstream to prevent bleeds. Roctavian is designed to replace the function of the mutated gene, allowing people with severe hemophilia A to produce their own FVIII and thereby limit bleeding episodes.
As part of the development of Roctavian, BioMarin has worked with private and public payers in the U.S. in parallel to enable access, with the goal of ensuring that every eligible adult interested in Roctavian is able to receive treatment.
It is estimated that there are approximately 6,500 adults living with severe hemophilia A in the U.S. BioMarin expects approximately 2,500 of those adults to be eligible to receive Roctavian with this initial approval.
