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FDA approves fibrinogen concentrate for bleeding in patients with congenital fibrinogen deficiency
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Last week the FDA announced that fibrinogen concentrate had been granted licensing as an orphan drug for the treatment of bleeding in patients with congenital fibrinogen deficiency.
Patients with this disorder are unable to make sufficient amounts of fibrinogen. It affects between 150 and 300 people in the United States and is usually diagnosed at birth when bleeding occurs from the umbilical cord site.
IV fibrinogen concentrate (RiaSTAP, CSL Behring) is approved for patients with afibrinogenemia or hypofibrinogenemia, but not for patients with dysfibrinogenemia.
Data to support the approval came from a study of 15 patients with afibrinogenemia who received 70 mg/kg of fibrinogen concentrate and achieved a target level of fibrinogen expected to prevent bleeding. In addition, plasma from 14 of the 15 patients showed increased maximum clot firmness, a surrogate marker likely to predict clinical benefit of the drug.
Fever and headache were the most common adverse reactions. Postmarketing studies will include patients with afibrinogenemia and hypofibrinogenemia.