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FDA Approves First Gene Therapy for Hemophilia B

Posted on: 29 November 2022, source: FDA
The FDA has approved etranacogene dezaparvovec for the treatment of adults with Hemophilia B who are currently using Factor IX prophylaxis therapy, have a history of life-threatening hemorrhaging, or have serious spontaneous bleeding episodes. The adeno-associated virus vector-based therapy is the first-of-its-kind for the treatment of Hemophilia B. “Gene therapy for hemophilia has been on the horizon for more than two decades,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in a press release. “Despite advancements in the treatment of hemophilia, the prevention and treatment of bleeding episodes can adversely impact individuals’ quality of life.”