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Male hand in blue protective gloves holding test tube with blood sample against microscope background.  Perform an express analysis for tools in the hospice's chemistry lab in the presence of the patient

Hemgenix may be used to treat adults with hemophilia. Ivan Balvan/Getty Images

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

  • Hemgenix has been effective in treating several patients with the blood disorder hemophilia B in studies.

  • An independent study says 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 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 absent or inadequate levels of blood clotting factor IX.

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

In a study distributed by CSL Behring, Hemgenix reduced the number of expected bleeding events over a year by 54%. In addition, 94% of patients eliminated the need to receive factor IX infusions, saving them significant time and money.

“Gene therapy for hemophilia has been on the cards for more than two decades,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research.

“Today’s approval offers a new treatment option for patients with hemophilia B and represents an important advance in the development of innovative therapies for patients suffering from 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 far.

A CSL spokesperson told the publication, “We are confident that this price point will result in significant cost savings across the healthcare system and significantly reduce the economic burden of hemophilia B by lowering annual bleeding rates, reducing or eliminating and increasing prophylactic therapy.” FIX (factor 9) levels that last for years.”

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

The story goes on

Brad Loncar, a biotechnology investor and CEO of Loncar Investments, told Bloomberg he believes the treatment could be successful because existing drugs are 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 was priced at $2.8 million earlier this year.

Read the original article on Business Insider

Regulators Approve World’s Most Expensive Drug Costing $3.5M Per Patient – Yahoo! Voices – S Chronicles

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