Posted by Michael Wonder on 25 Aug 2022
First gene therapy for adults with severe haemophilia A, BioMarin's Roctavian (valoctocogene roxaparvovec), approved by European Commission
24 August 2022 - Maintains orphan drug designation in the EU providing 10 years of market exclusivity.
BioMarin today announced that the European Commission has granted conditional marketing authorisation to Roctavian (valoctocogene roxaparvovec) gene therapy for the treatment of severe haemophilia A (congenital Factor VIII deficiency) in adult patients without a history of factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5.
