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FDA Approves CSL's Gene Therapy To Treat Adults With Hemophilia B

CSL (CSL.AX) said that the U.S. Food and Drug Administration approved Hemgenix (etranacogene dezaparvovec-drlb), the first and only one-time gene therapy for appropriate adults with hemophilia B. Hemgenix is approved for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes.

The approval provides a new treatment option that reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy and generates elevated and sustained factor IX levels for years after a one-time infusion, the company said in a statement.

CSL Behring, a CSL business, will make Hemgenix available for eligible people with hemophilia B as soon as possible.

In the ongoing clinical trial, Hemgenix reduced the rate of annual bleeds and 94 percent of patients discontinued factor IX prophylaxis and remained prophylaxis-free.
Hemgenix is still currently under assessment by other regulatory agencies.

Hemophilia B is a life-threatening rare disease. People with the condition are particularly vulnerable to bleeds in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage. Current treatments for moderate to severe hemophilia B include life-long prophylactic infusions of factor IX to temporarily replace or supplement low levels of the blood-clotting factor.

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