Regulatory authorities have approved the world’s most expensive drug, costing $3.5 million per patient

Male hand in blue protective gloves holds test tube with blood sample against background of microscope.  Performs express analysis for aids in the presence of the patient in the hospice chemical laboratory

Hemgenix can be used to treat adults with haemophilia.Ivan Balvan/Getty Images

  • The FDA approved a treatment that costs $3.5 million, making it the world’s most expensive drug.

  • Hemgenix effectively treated several patients with the blood disease hemophilia B in trials.

  • 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 had approved Hemgenix, the first gene therapy to treat adults with hemophilia B, a genetic bleeding disorder caused by 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 bleeding events over a year by 54%. It also eliminated the need for 94% of patients to receive infusions of factor IX, 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 important progress in the development of innovative therapies for those experiencing a high disease burden associated with this form of hemophilia.”

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

A CSL spokesperson told the publication: “We are confident that this price point will generate significant cost savings for the overall healthcare system and significantly lower the financial burden of hemophilia B by reducing annual bleeding rates, reducing or eliminating prophylactic therapy, and generating elevated 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 biotech investor and CEO of Loncar Investments, told Bloomberg that he believed the treatment could be successful because existing drugs were also very expensive and hemophiliacs “live in constant fear of bleeding.”

The listing price for Hemgenix overtakes Bluebird Bio’s Zynteglo, which treats the blood disease beta-thalassemia, which was priced at $2.8 million earlier this year.

Read the original article on Business Insider

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