BioMarin Pharmaceutical Inc.’s (BMRN) investigational AAV5 gene therapy, Valoctocogene roxaparvovec, proposed for the treatment of adults with hemophilia A, is under priority review by the FDA, with a decision expected on August 21, 2020.Hemophilia A is a condition of increased tendency to bleed due to an inherited deficiency of a clotting protein known as Factor VIII (FVIII). The current standard of care for most hemophilia A patients is a prophylactic regimen of intravenous infusions three times per week. Valoctocogene roxaparvovec, an adeno-associated virus serotype 5 (AAV5) mediated gene therapy, is designed to work by delivering a B-domain-deleted variant of human coagulation Factor VIII gene to cells in the liver, enhancing Factor VIII expression.If approved, Valoctocogene roxaparvovec will be the first gene therapy in the U.S. for the treatment of any type of hemophilia. This product is also under review by the European Medicines Agency.BMRN closed Friday’s (Jul.24, 2020) trading at $122.95, down 1.24%.