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FDA approves gene therapy for hemophilia B, now world's costliest medicine

Added 11-24-22 03:03:01pm EST - “The Food and Drug Administration has approved a gene therapy treatment for adults with hemophilia B that costs $3.5 million per dose, making it the world's most expensive medicine.” - Washingtontimes.com

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Posted By TheNewsCommenter: From Washingtontimes.com: “FDA approves gene therapy for hemophilia B, now world’s costliest medicine”. Below is an excerpt from the article.

The Food and Drug Administration has approved a gene therapy treatment for adults with hemophilia B that costs $3.5 million per dose, making it the world’s most expensive medicine.

“Today’s approval provides a new treatment option for patients with hemophilia B and represents important progress,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said Tuesday in an announcement.

Biopharmaceutical firm CSL Behring manufactures the gene therapy treatment Hemgenix, which allows patients with hemophilia to produce a protein that makes their blood clot in open wounds and halt prolonged bleeding.

Each dose of Hemgenix costs $3.5 million, but the gene therapy treatment only requires a single dose, according to the FDA.

Some experts see the price as high but acknowledge that existing treatments for hemophilia are also expensive and say the fear of bleeding complications will drive patient demand.

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