The U.S. Food and Drug Administration (FDA) approved BioMarin Pharmaceutical Inc.’s Roctavian (valoctocogene roxaparvovec-rvox), the first gene therapy for the treatment of adults with severe hemophilia A (congenital Factor VIII [FVIII] deficiency with FVIII activity.
Also on June 29, 2023, the FDA approved ARUP Laboratories’ AAV5 DetectCDx as a companion diagnostic to aid in the selection of adult patients eligible for treatment with Roctavian. Based on the label, BioMarin estimates that about 2500 patients may be eligible to receive Roctavian.
Roctavian consists of a viral vector carrying the F8 gene that encodes FVIII and is administered intravenously (IV) as a one-time dose. Roctavian is not intended for administration in women.
Due to its high cost, it has been recommended that payers implement prior authorization to reserve Roctavian’s use for patient populations most likely to benefit from this treatment.
