Benefits include decreased need for routine factor IX replacement prophylaxis and reduction in annualized bleeding rate<\/p>\n<\/h3>\n
By Lori Solomon HealthDay Reporter<\/b><\/p>\n
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MONDAY, Nov. 28, 2022 (HealthDay News) — The U.S. Food and Drug Administration has approved Hemgenix (etranacogene dezaparvovec) for the treatment of hemophilia B in adults.<\/p>\n
Hemgenix, an adeno-associated virus vector-based gene therapy, is administered via intravenous infusion as a one-time, single dose. <\/p>\n
The approval was based on two studies of adult men (ages 18 to 75 years) with severe or moderately severe hemophilia B. Outcomes included increases in factor IX activity levels, a decreased need for routine factor IX replacement prophylaxis, and a 54 percent reduction in the annualized bleeding rate compared with baseline. Adverse reactions included liver enzyme elevations, headache, mild infusion-related reactions, and flu-like symptoms.<\/p>\n
“Gene therapy for hemophilia has been on the horizon for more than two decades,” Peter Marks, M.D., Ph.D., director of the FDA Center for Biologics Evaluation and Research, said in a statement. “Today’s approval provides a new treatment option for patients with hemophilia B and represents important progress in the development of innovative therapies for those experiencing a high burden of disease associated with this form of hemophilia.”<\/p>\n
Approval of Hemgenix was granted to CSL Behring.<\/p>\n