FDA Approves First Gene Therapy for Adults With Hemophilia B

Benefits include decreased need for routine factor IX replacement prophylaxis and reduction in annualized bleeding rate
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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.

Hemgenix, an adeno-associated virus vector-based gene therapy, is administered via intravenous infusion as a one-time, single dose.

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.

"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."

Approval of Hemgenix was granted to CSL Behring.

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