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FDA approves new gene therapy treatment for hemophilia
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FDA approves new gene therapy treatment for hemophilia

FDA approves Hemgenix for Hemophilia B. Hemgenix provides a new treatment option.

Where: United States

The Facts

The US Food and Drug Administration (FDA) approved Hemgenix, a gene therapy product for the treatment of adults with Hemophilia B.
Peter Marks, director of FDA’s Center for Biologics Evaluation and Research stated, “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.”
Missing or insufficient levels of blood clotting Factor IX results in Hemophilia B.
The FDA release states, “Hemgenix is a one-time gene therapy product given as a single dose by IV infusion.”
Experts expect that the drug will come with a super-high price point.

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