FDA approves new treatment for rare blood-clotting disorder


ROCKVILLE, Md. The Food and Drug Administration has approved a new biologic for treating a rare blood-clotting disorder.

The FDA announced Friday that it approved GTC Biotherapeutics' ATryn (antithrombin alfa), a treatment for hereditary antithrombin deficiency that the agency had designated an orphan drug due to the rarity of the disease, which affects about one-in-5,000 people in the United States. The drug is produced using genetically engineered goats. 

"This product offers an important new treatment option for patients with hereditary antithrombin deficiency, preventing life-threatening clots that otherwise frequently occur during high-risk situations," FDA Center for Biologics Evaluation and Research director Jesse Goodman said in a statement. 

Hereditary AT is usually first diagnosed in teenagers and young adults when they develop clots in their blood vessels, especially during pregnancy, surgery or prolonged bed rest.

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