FDA approves a gene therapy for the inherited bleeding disorder hemophilia B

The Food and Drug Administration on Tuesday approved the first gene therapy to treat people with hemophilia B, an inherited bleeding disorder.

The one-time treatment, called Hemgenix, was developed by the Dutch biotech company UniQure and will be marketed by CSL Behring, an Australian pharmaceutical company.

Hemgenix will cost $3.5 million, making it the most expensive drug approved to date.

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