On Jun. 29, 2023, the U.S. Food and Drug Administration approved Roctavian, an adeno-associated virus vector-based gene therapy for the treatment of adults with severe hemophilia A without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test. Roctavian was developed by BioMarin Pharmaceutical.

Hemophilia A is a rare genetic bleeding disorder that occurs due to a mutation on the gene which produces factor VIII (FVIII), a protein that enables blood to clot. The disorder primarily affects males. This deficiency in FVIII causes affected individuals to have uncontrolled bleeding and bleed longer than people who do not have the condition. The frequency and severity of bleeding episodes depends on how much FVIII protein a person produces.

Roctavian is a one-time gene therapy product administered as a single dose by intravenous infusion. Roctavian consists of a viral vector carrying a gene for clotting Factor VIII. The gene is expressed in the liver to increase blood levels of FVIII and reduce the risk of uncontrolled bleeding. 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.