The U.S. Food and Drug Administration has approved Hemgenix, a gene therapy, to treat certain patients with hemophilia B, a genetic bleeding disorder.
The drugs manufacturer, Pennsylvania-based CSL Behring, set the price of the one-time intravenous infusion at $3.5 million, making it the most expensive drug in the world.
Symptoms of hemophilia B, which results from missing or insufficient levels of blood clotting Factor IX, include prolonged or heavy bleeding, in severe cases without clear cause.
Hemgenix delivers a viral vector carrying a gene for clotting Factor IX. The gene helps the liver produce the Factor IX protein, ultimately limiting bleeding episodes.
“Gene therapy for hemophilia has been on the horizon for more than two decades,” said Peter Marks, M.D., Ph.D., director of the FDAs Center for Biologics Evaluation and Research.
“Todays approval provides a new treatment option for patients with Hemophilia B and represents important progress in the development of innovative therapies,” Marks said.
