U.S. FDA Approved World's Most Expensive Drug Costs $3.5-M Per Patient

US regulators have approved a hemophilia drug that will cost $3.5 million per patient, making it the world's most expensive medicine while an independent study said a fair price for the drug would be about $2.9 million, Ryan Hogg reported for Business Insider.

Photo Insert: Factor IX

The Food and Drug Administration (FDA) announced on Tuesday that it had approved Hemgenix, 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.

The list price for Hemgenix overtakes Bluebird Bio's Zynteglo, which treats the blood disorder beta thalassemia, that was priced at $2.8 million earlier this year.

FDA said the condition affects one in 40,000 people, mostly men and accounts for about 15% of all hemophilia cases. 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.

"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.

"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."

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