The US biotech claimed conditional marketing approval from the European Commission for Roctavian (valoctocogene roxaparvovec) as a treatment for adults with severe haemophilia A, who don’t have a history of developing the antibodies that inhibit the activity of blood-clotting protein Factor VIII.
Roctavian delivers a working copy of the Factor VIII gene into cells via an adeno-associated virus (AAV) vector, and offers a one-shot, potentially curative therapy for haemophilia A.
