FDA approves first gene therapy for hemophilia

The Food and Drug Administration on Tuesday approved the first gene therapy for a type of hemophilia, giving people with the inherited disorder a treatment option that could potentially keep their bleeding in check for years while also allowing them to skip the infusions that have been the standard of care.

The therapy, called Hemgenix and developed by the Dutch biotechnology company UniQure, is for the less common “B” form of hemophilia, which is estimated to represent about 15% of all patients with the disease.

“Gene therapy for hemophilia has been on the horizon for more than two decades,” said Peter Marks, head of the FDA’s Center for Biologics Evaluation and Research, in a Tuesday statement. “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.” 

Australian drugmaker CSL, which licensed Hemgenix from UniQure and will market the drug, set the treatment’s list price at $3.5 million, making it the most expensive medicine in the U.S. on a single-use basis. 

In a statement, the company said it is confident that the price “will generate significant cost savings for the overall healthcare system and significantly lower the economic burden of hemophilia B.” 

The Institute for Clinical and Economic Review, an influential nonprofit that assesses drug costs, previously estimated treatment with Hemgenix could be cost effective at a price of $2.9 million. The group cited the treatment’s benefit, as well as the high cost of current treatment.

This is breaking news. Check back soon for updates.

 

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