The FDA approved a treatment at a cost of $3.5 million, making it the most expensive medicine in the world.
Hemgenix has effectively treated several 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 medicine in the world.
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 that results from 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 the Hemgenix study, distributed by CSL Behring, the number of expected bleeding events over a one-year period decreased by 54%. It also eliminated the need for 94% of patients to receive Factor IX infusions, 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 Biologics Evaluation and Research.
“Today’s approval provides a new treatment option for patients with hemophilia B and represents an important advance in the development of innovative therapies for those with a high disease burden associated with this form of hemophilia.”
However, the drug will have a list price of $3.5 million per dose, the Managed Health Executive reported, making it the world’s most expensive medicine in some time.
A spokesperson for CSL told the publication: “We are confident that this price point will generate significant cost savings for the entire healthcare system and significantly reduce the economic burden of hemophilia B by reducing annual bleeding rates, reducing or eliminating prophylactic therapy and generate 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 chief executive of Loncar Investments, told Bloomberg that he thought the treatment would succeed because the existing drugs were also very expensive and hemophilia patients “always live in fear of blood .”
The list price surpasses that for Hemgenix Zynteglo Bluebird Bio, which treats the blood disorder beta thalassemia, which was priced at $2.8 million earlier this year.
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