voloctocogene roxaparvovec

Voloctocogene roxaparvovec (brand name: Roctavian) is an adeno-associated virus serotype 5 (AAV5)-based gene therapy developed by BioMarin Pharmaceutical for the treatment of severe hemophilia A (congenital factor VIII deficiency). It delivers a functional complementary DNA (cDNA) encoding the B-domain deleted SQ form of human coagulation factor VIII (hFVIII-SQ) under the control of a liver-selective promoter. This enables hepatocytes to endogenously produce and secrete functional factor VIII, restoring the coagulation cascade and reducing or eliminating the need for prophylactic factor VIII infusions. Administered as a single intravenous infusion, it was conditionally approved in the European Union in August 2022 and fully approved in the United States in June 2023.

Brand names

Roctavian

Other names

valoctocogene roxaparvovecvaloctocogene roxaparvovec-rvoxValroxAAV-hFVIII-SQ