FDA approves new treatment for bleeding disease

Rockville, Md. The Food and Drug Administration has approved a drug to treat a bleeding disease that affects as few as 150 people in the United States.

The FDA licensed Marburg, Germany-based CSL Behring?s drug RiaSTAP (fibrinogen concentrate [human]), a treatment for the inherited disorder congenital fibrinogen deficiency. The agency had given the drug orphan drug status, which it grants to drugs for treating rare diseases that lack effective treatment options. Congentical fibrinogen deficiency affects between 150 and 300 Americans.People who have the disease face potentially life-threatening bleeding due to their livers? inability to make sufficient amounts of fibrinogen, which aids blood clotting. The bloodstream normally contains 250 to 400 mg per decaliter of fibrinogen."This product offers much-needed treatment for the small number of patients with congenital fibrinogen deficiency," FDA Center for Biologics Evaluation and Research director Jesse Goodman said in a statement. "If bleeding occurs in the brain or other organs and is left untreated, it may lead to blood loss, organ damage and death."RiaSTAP is an intravenous concentrate made from the plasma of healthy human blood donors.