FDA Approves Human Fibrinogen Concentrate for Treatment of Bleeding in Congenital Fibrinogen Deficiency

Laurie Barclay, MD

January 28, 2009

January 28, 2009 — On January 16, the US Food and Drug Administration (FDA) granted accelerated approval to human fibrinogen concentrate (RiaSTAP, CSL Behring). This orphan drug is intended for use in the treatment of bleeding in patients with congenital fibrinogen deficiency.

Untreated patients who have this rare genetic defect are at risk for potentially life-threatening hemorrhage because they are unable to make sufficient amounts of fibrinogen. This coagulation factor normally is manufactured in the liver and is present in plasma at a concentration of 250 to 400 mg/dL.

"This product offers much-needed treatment for the small number of patients with congenital fibrinogen deficiency," Jesse Goodman, MD, MPH, director of the FDA's Center for Biologics Evaluation and Research, said in a news release. "If bleeding occurs in the brain or other organs and is left untreated, it may lead to blood loss, organ damage and death."

Human fibrinogen concentrate, an intravenous fibrinogen concentrate made from the plasma of healthy human blood donors, is indicated for patients with afibrinogenemia (no detectable fibrinogen) or hypofibrinogememia (fibrinogen levels < 50 mg/dL). However, it is not indicated for patients with dysfibrinogenemia, who have normal fibrinogen levels but defective fibrinogen function and who are at risk for both hemorrhagic and thrombotic complications.

Data leading to the licensing of the concentrate came from a study of 15 patients with afibrinogenemia who achieved the target level of fibrinogen expected to prevent bleeding after they were given human fibrinogen concentrate at a dose of 70 mg/kg. A secondary endpoint of maximum clot firmness, a surrogate marker thought to predict clinical benefit, was present in plasma from 14 of the 15 patients. The most frequently reported adverse reactions were fever and headache.

US prevalence of fibrinogen deficiency is only 150 to 300 people. Bleeding from the umbilical cord site usually leads to diagnosis at birth. Parents of children diagnosed with fibrinogen deficiency are counseled to keep them from sports or other activities that could cause minor trauma and lead to bleeding.

A postmarketing study of both afibrinogenemic and hypofibrinogenemic patients will further assess clinical benefit of human fibrinogen concentrate.

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