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FDA Licenses First Concentrate for Factor I Deficiency

The U.S. Food and Drug Administration (FDA) granted a license to CSL Behring’s RiaSTAP™, for the treatment of patients with congenital fibrinogen deficiency, a rare bleeding disorder that can be life-threatening. It is usually diagnosed at birth when newborns hemorrhage from the umbilical cord stump, genitourinary tract or central nervous system.

RiaSTAP™ is indicated for patients with afibrinogenemia (the absence of the protein fibrinogen), also called factor I deficiency. Fibrinogen, a protein made in the liver, is needed for proper blood clotting. The disorder is very rare, occurring in individuals who inherit an abnormal gene from both parents. Incidence has been reported at about one out of every million people. There are approximately 300 patients with factor 1 deficiency in the U.S. Symptoms include easy bruising, nose and mouth bleeds and soft tissue bleeding. Miscarriage can occur in women.

The therapy is also indicated for hypofibrinogenemia, lower than normal levels of fibrinogen, with bleeding symptoms that may be mild, moderate or severe. It is not indicated for patients with dysfibrinogenemia, malfunctioning fibrinogen. These patients rarely experience bleeds but can be susceptible in some cases to thrombosis, or blood clots.

Until now, the treatment for fibrinogen deficiency has been intravenous infusions of fibrinogen-rich cryoprecipitate or plasma. Standard practice requires that donors of plasma are screened.  However, it should be noted that cryoprecipitate or “cryo,” a component of blood separated from thawed plasma as a precipitate, is not treated to kill viruses. It potentially could be infectious, despite donor screening using nucleic acid testing.

In a study of 15 patients with afibrinogenemia, all achieved target levels of fibrinogen and 14 had improvement in clot formation. Side effects included headache and fever. RiaSTAP™ is the first factor I concentrate to become available in the U.S. A version of it has been available in Europe for years.

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

Designated an orphan drug by the FDA, RiaSTAP™ is an intravenous fibrinogen concentrate derived from the plasma of healthy human blood donors. The FDA’s Office of Orphan Products Development oversees and grants “orphan drug status,” to drugs seeking FDA approval. The designation allows for accelerated approval, so that much-needed drugs or biologics become available to patients with rare, life-threatening diseases who need effective treatments. More than 1,400 drugs and biologics have been designated as orphan drugs. Since 1983, more than 250 of them have been approved for marketing.

Source: FDA and CSL Behring news releases dated January 16, 2009


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