Valoctocogene roxaparvovec (Roctavian; Biomarin Pharmaceutical Inc.) 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 international unit/deciliter [IU/dL]) without pre-existing antibodies to adeno-associated virus serotype 5 detected by a U.S. Food and Drug Administration (FDA)-approved test.
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