HEMGENIX: First EU gene therapy to treat haemophilia B
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Hemgenix (etranacogene dezaparvovec) is now available on the European market for the treatment of severe and moderately severe haemophilia B in adults without factor IX inhibitors (autoantibodies produced by the immune system that make drugs against factor IX less effective). The recommendation for the granting of marketing authorization in EU countries is from the EMA.
Hemophilia B is an inherited disorder characterized 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.
Factor IX deficiency is the result of mutations in the respective coagulation factor gene. Prolonged bleeding episodes in patients with hemophilia B can lead to serious complications, such as bleeding into joints, muscles or internal organs, including the brain. Hemophilia B is a rare, debilitating disease that affects approximately 1 newborn in 20,000 to 50,000 males alive.
Currently, medicines licensed for the treatment of haemophilia B are aimed at preventing bleeding episodes or treating bleeding episodes that can occur during surgery or… read more
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