FDA Approves World’s Most Expensive Drug Costing $3.5 Million a Patient

  • The US Food and Drug Administration has approved a treatment that costs $3.5 million, making it the most expensive drug in the world.
  • Hemgenix effectively treated many patients with the blood condition hemophilia B in trials.
  • An independent study said a fair price for the drug would be around $2.9 million.

US regulators have approved a hemophilia drug that will cost $3.5 million per patient, making it the most expensive drug in the world.

The U.S. Food and Drug Administration (FDA) announced Tuesday that it has approved Hemgenix, the first gene therapy to treat adults with hemophilia B, a genetic bleeding disorder caused by insufficient or decreased levels of blood clotting factor IX.

The Food and Drug Administration said the condition affected about 1 in 40,000 people, most of them men. It accounts for about 15% of all cases of hemophilia.

In a study, Hemgenix, distributed by CSL Behring, reduced the number of expected bleeding events over the course of a year by 54%. It also eliminated the need for 94% of patients to receive factor IX injections, saving them a lot of 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 Biology Evaluation and Research.

“Today’s approval provides a new treatment option for patients with hemophilia B and represents an important advance in developing innovative therapies for those with a high disease burden associated with this form of hemophilia.”

However, the drug’s price tag will be $3.5 million per dose, the director of managed health care said, making it the most expensive drug in the world in terms of cost.

A CSL spokesperson told the publication: “We are confident that this price point will deliver significant cost savings to the overall health care system and significantly reduce the economic burden of hemophilia B by reducing annual bleeding rates, reducing or eliminating prophylaxis and generating high FIX (Factor 9) levels that last for years.”

The price is higher than the figure of about $2.9 million recommended in an independent review by the Institute for Clinical and Economic Review.

Brad Loncar, a biotechnology investor and CEO of Loncar Investments, told Bloomberg that he thought the treatment could be successful because existing drugs were also very expensive and hemophilia patients “always live in fear of bleeding.”

Hemgenix’s list price beats Bluebird Bio’s Zynteglo, which treats the blood disorder beta thalassemia, which had a $2.8 million price tag earlier this year.


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