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FDA approves $3.5 million gene therapy for hemophilia

Hemophilia Drug
Posted at 9:24 AM, Nov 23, 2022

The Food and Drug Administration (FDA) approved a drug to treat adults with hemophilia B, a rare blood clotting disorder.

Hemgenix is a one-time gene therapy that will reportedly cost $3.5 million.

“Gene therapy for hemophilia has been on the horizon for more than two decades," said Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research. “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.”

According to The Associated Press, drugmaker CSL Behring justified the pricetag, claiming its drug would ultimately reduce health care costs for patients with hemophilia.

The FDA reports that about one in 40,000 people has hemophilia B. Most individuals who experience symptoms are men, health officials say.

Prior to the approval of Hemgenix, patients would typically need routine IV infusions to prevent bleeding episodes.

Hemgenix is also given through IV infusion. Although, it's only needed one time, according to the drugmaker.

“Over the years we have seen a variety of advancements for the hemophilia community, but gene therapy is the first treatment option to offer those living with hemophilia B--and caregivers--the possibility of freedom from the need for regular, ongoing infusions," said Kim Phelan, chief operating officer of The Coalition for Hemophilia B.