FDA approves CSL’s gene therapy for hemophilia

(Reuters) -A one-time gene therapy for hemophilia B from Australian drugmaker CSL Ltd has been approved by the U.S. Food and Drug Administration, potentially offering a long-term solution for patients with the blood clotting disorder.

The therapy, which will be branded Hemgenix, will be made immediately available in the United States at a list price of $3.5 million, the company said, making it the most expensive therapy in the world.

The approval makes it the first gene therapy for the rare inherited disorder that is usually treated by regular injections of clotting protein factor IX.

The therapy, developed in partnership with Uniqure NV, is expected to cut the rate of annual bleeding for hemophilia B patients, who represent about 15% of all patients with hemophilia.

The rare disorder is caused by a gene mutation that hampers the body’s ability to make factor IX, and current treatments from companies such as Biogen and Pfizer focus on regular infusions of the protein.

(Reporting by Sriparna Roy and Leroy Leo in Bengaluru; Editing by Sriraj Kalluvila and Krishna Chandra Eluri)


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