Roctavian

Roctavian is a medicine for treating severe haemophilia A, an inherited bleeding disorder caused by the lack of a clotting protein known as factor VIII. It is used in adults who do not have inhibitors (antibodies) against factor VIII and who have no antibodies against adeno-associated virus serotype 5 (AAV5). Roctavian contains the active substance valoctocogene roxaparvovec and is a type of advanced therapy medicine called a ‘gene therapy product’. This is a type of medicine that works by delivering genes into the body. Haemophilia A is rare, and Roctavian was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 21 March 2016. Further information on the orphan designation can be found here: [ema.europa.eu/medicines/human/orphan-designations/EU3161622](/en/medicines/human/orphan-designations/eu-3-16-1622).