The U.S. Food and Drug Administration on January 16, 2009
licensed RiaSTAP, an orphan drug for the treatment of bleeding
in patients with a rare genetic defect known as congenital
fibrinogen deficiency. Without treatment, these patients are at
risk of potentially life-threatening bleeding.
People with congenital fibrinogen deficiency are unable to make
sufficient amounts of fibrinogen, which plays an important role in
blood coagulation by helping to form blood clots and prevent
bleeding. Fibrinogen is manufactured in the liver and circulates in
the blood plasma in a normal concentration of 250-400 mg/dL.
Fibrinogen deficiency affects only 150 to 300 people in the
United States and is usually diagnosed at birth when newborns
bleed from their umbilical cord site. Children with the defect need
to curtail activities because of risk of bleeding from minor trauma.
RiaSTAP is an intravenous fibrinogen concentrate made from the
plasma of healthy human blood donors. The product is indicated
for patients who have no fibrinogen or low levels of the
substance, an abnormality known as afibrinogenemia, or for
those patients whose fibrinogen levels are below 50 mg/dL, an
abnormality known as hypofibrinogenemia. The product is not
indicated for patients with dysfibrinogenemia, who may have
normal fibrinogen levels but defective fibrinogen function.
Patients such as these are at risk for both bleeding and clotting
The licensing of RiaSTAP was supported by a study of 15
patients with afibrinogenemia who achieved the target level of
fibrinogen expected to prevent bleeding after they received 70
mg/kg of the drug. In addition, plasma from 14 of the 15
patients showed increased maximum clot firmness, a surrogate
marker likely to predict clinical benefit. Fever and headache were
the most common adverse reactions.
FDA Approves RiaSTAP for Treatment of Bleeding in Patients
with Rare Genetic Defect FDA News January 16, 2009
THIS WEBSITE TALKS ABOUT THE SIDE EFFECTS AND THE POTENTIAL HEALTH BENEFITS OF HERBS, SUPPLEMENTS,