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FDA Alerts
Hematology

FDA approves gene therapy for Hemophilia B

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The U.S. Food and Drug Administration has approved Hemgenix (etranacogene dezaparvovec), an adeno-associated virus vector-based gene therapy for the treatment of adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, according to a press release.

Etranacogene dezaparvovec was evaluated in 2 studies of men between the ages of 18 and 75 years with severe or moderately severe Hemophilia B. A 54% reduction in annualized bleeding rate compared to baseline was reported in 1 study of 54 participants. Increases in Factor IX activity levels and a decreased need for routine Factor IX replacement prophylaxis were also noted.

The most common adverse reactions associated with Hemgenix 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. Despite advancements in the treatment of hemophilia, the prevention and treatment of bleeding episodes can adversely impact individuals’ quality of life,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research in a press release. “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.”

Read the full press release here.

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