The U.S. Food and Drug Administration (FDA) has approved CSL Behring’s hemophilia B gene therapy, a one-off infusion treatment with a list price of $3.5 million, making it the world’s most expensive medicine.
The approval of CSL Behring’s Hemgenix “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,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research.
Hemgenix is used for treating hemophilia B patients who currently use factor IX prophylaxis therapy or suffer from serious spontaneous bleeding episodes or have had a life-threatening hemorrhage. Based on clinical trials, the infusion reduced annual bleeds and “94 percent of patients discontinued factor IX prophylaxis and remained prophylaxis-free,” said the company, opening the possibility for the drug to eliminate the need for lifelong routine treatment in adult patients….