US regulators have approved a hemophilia drug that will cost $3.5 million per patient, making it the world’s most expensive medicine.<\/p>\n
The Food and Drug Administration (FDA) announced <\/a>that it had approved Hemgenix<\/a>, the first gene therapy to treat adults with hemophilia B, a genetic bleeding disorder resulting from missing or insufficient levels of blood-clotting Factor IX.<\/p>\n The FDA said the condition affected one in 40,000 people, mostly men. It accounts for about 15% of all hemophilia cases.<\/p>\n In a study Hemgenix, distributed by CSL Behring, cut the number of bleeding events expected over a year by 54%. It also removed the need for 94% of patients to receive infusions of Factor IX, saving them considerable time and money.<\/p>\n “Gene therapy for hemophilia has been on the horizon for more than two decades,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research.<\/p>\n “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.”<\/p>\n However, the drug will have a list price of $3.5 million per dose, Managed Healthcare Executive<\/a> reported, making it the world’s most expensive medicine by some distance.<\/p>\n A spokesperson for CSL told the publication: “We are confident this price point will generate significant cost savings for the overall healthcare system and significantly lower the economic burden of hemophilia B by reducing annual bleed rates, reducing or eliminating prophylactic therapy and generating elevated FIX (factor 9) levels that last for years.”<\/p>\n The price is higher that the figure of about $2.9 million recommended in an independent review<\/a> by the Institute for Clinical and Economic Review.<\/p>\n