The U.S. Food and Drug Administration (FDA) last week approved the first gene therapy to treat adults with hemophilia B, a genetic bleeding disorder resulting from missing or insufficient levels of a protein needed to make blood clot. About 15% of all hemophilia is hemophilia B. Other biopharmaceutical manufacturers are trying to create gene therapies for the more common type, hemophilia A, in which a different protein is missing.
FDA approved Hemgenix, manufactured by CSL Behring, on Nov. 22. According to several news organizations, an infusion will cost $3.5 million, making Hemgenix the world’s most expensive drug. “We are confident this price point will generate significant cost savings for the overall healthcare system and significantly lower the economic burden of hemophilia B,” the company told news outlets.
The U.S. Food and Drug Administration (FDA) last week approved the first gene therapy to treat adults with hemophilia B, a genetic bleeding disorder resulting from missing or insufficient levels of a protein needed to make blood clot.
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