FDA approves $3.5M Hemgenix gene therapy drug

The FDA approved the $3.5 million gene therapy drug Hemgenix as a treatment for hemophilia B. Photo courtesy of U.S. Food and Drug Administration/<a href="">Flickr</a>
The FDA approved the $3.5 million gene therapy drug Hemgenix as a treatment for hemophilia B. Photo courtesy of U.S. Food and Drug Administration/Flickr

Nov. 23 (UPI) -- The most expensive drug in the world has been approved by the U.S. Food and Drug Administration, estimated to cost $3.5 million for a single dose.

Hemgenix is a gene therapy treatment for adults with hemophilia B. It allows patients to avoid ongoing, repeated treatments, instead needing the single dose.


"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 burden of disease associated with this form of hemophilia," Peter Marks, director of the FDA's Center for Biologics Evaluation and Research, said Tuesday in a statement.

Hemophilia B is a genetic disorder that causes prolonged and heavy bleeding. Most symptomatic hemophilia B patients are men. The condition is present in about one of every 40,000 people. Only 15% of people with hemophilia have hemophilia B. Without treatment it is a lifelong condition.

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Before Hemgenix, repeated treatments of Hemophilia B required intravenous infusions. Hemgenix was tested on 57 men between 18 and 75 years old. The subjects had "severe or moderately severe hemophilia B," the FDA report said.


The new drug is manufactured by CSL Behring.

"As part of our promise to patients, CSL is committed to delivering innovative and groundbreaking solutions to address unmet medical needs, and we are proud to introduce the next wave of breakthrough medicines for people living with hemophilia B," said Paul Perreault, CSL CEO and Managing Director.

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According to the press release by CSL Behring, Hemgenix reduced annual bleeds by 94%.

Existing gene therapies have a track record of being especially expensive. Zynteglo, a therapy that helps produce a hemoglobin component, costs about $2.8 million. Zolgensma, which treats spinal muscle atrophy, was introduced with a price point of about $2.1 million.

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