Posted by Michael Wonder on 29 Jun 2023
US FDA approves BioMarin's Roctavian (valoctocogene roxaparvovec-rvox), the first and only gene therapy for adults with severe haemophilia A
29 June 2023 - Roctavian's approval was based on durability, efficacy and safety results from the largest and longest Phase 3 study for a gene therapy for haemophilia.
BioMarin Pharmaceutical today announced that the US FDA approved Roctavian (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults with severe haemophilia A (congenital factor VIII deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 detected by an FDA approved test.
