The CHMP backed a conditional marketing authorisation for Roctavian (valoctocogene roxaparvovec or valrox) for severe haemophilia A in adults without inhibitors – antibodies that make standard Factor VIII replacement medicines less effective.
Patients should also not antibodies to the adeno-associated virus serotype 5 (AAV5), the viral vector used to deliver the gene therapy in the body.
The positive opinion comes two years after the FDA rejected BioMarin’s marketing application for valrox in a surprise decision that scuppered BioMarin’s plans for a swift roll out for the gene therapy.
