EMA has recommended granting a conditional marketing authorisation in the European Union (EU) for Roctavian (valoctocogene roxaparvovec) for the treatment of severe haemophilia A in adults who do not have factor VIII inhibitors (auto-antibodies produced by the immune system which make factor VIII medicines less effective) and no antibodies to adeno-associated virus serotype 5 (AAV5).
Patients with haemophilia A cannot produce factor VIII (an essential protein required for blood to clot and stop bleeding); they are more prone to bleeding and have prolonged bleeding, e.g. after injury or surgery. Haemophilia A is a rare debilitating disease affecting approximately 0.7 in 10,000 people in the EU. It is life long and may be life threatening when bleeding occurs in the brain, the spinal cord or the gut…
