Tradename: ROCTAVIAN

Proper Name: valoctocogene roxaparvovec-rvox

Indication: Valoctocogene roxaparvovec-rvox is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity <1 IU/dL) without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test.

Approval Date: 06/29/2023

Manufacturer: BioMarin Pharmaceutical Inc.