EMA has recommended granting a conditional marketing authorisation in the European Union (EU) for Hemgenix (etranacogene dezaparvovec) for the treatment of severe and moderately severe haemophilia B in adults who do not have factor IX inhibitors (auto-antibodies produced by the immune system which make factor IX medicines less effective).
Haemophilia B is an inherited disorder characterised by an increased bleeding tendency due to a partial or complete deficiency of coagulation factor IX, a protein needed to produce blood clots to stop bleeding. The deficiency of factor IX is the result of mutations of the respective clotting factor gene. Prolonged bleeding episodes in patients with haemophilia B can lead to serious complications, such as bleeding into joints, muscles or internal organs, including the brain. Haemophilia B is a rare debilitating disease affecting approximately 1 in 20,000 to 50,000 live male newborns…
