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

  • The FDA approved a treatment that cost $3.5 million, making it the world’s most expensive drug.
  • Hemgenix has effectively treated several patients with the hemophilia B blood disorder in studies.
  • An independent study said a fair price for the drug would be about $2.9 million.

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

The 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 that results from missing or insufficient levels of blood clotting factor IX.

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

In a study, Hemgenix, distributed by CSL Behring, reduced the number of expected bleeds over a year by 54%. It also eliminated the need for factor IX infusions in 94% of patients, saving them significant 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 an important advancement in the development of innovative therapies for those experiencing a high burden of disease associated with this form of hemophilia.”

However, the drug will have a list price of $3.5 million per dose, Managed Healthcare Executive reported, making it the world’s most expensive drug by some distance.

A spokesperson for CSL told the publication: “We are confident that this award will provide significant cost savings to the overall healthcare system and significantly reduce the economic burden of hemophilia B by reducing the number of annual bleeds, reducing or eliminating prophylactic therapy and generate elevated FIX (factor of 9) levels that persist for years.”

The price exceeds the figure of approximately $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 hemophiliacs “live in constant fear of bleeding.”

The list price for Hemgenix surpasses Bluebird Bio’s Zynteglo, which treats the blood disorder beta thalassemia, which cost $2.8 million earlier this year.

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