SILVER SPRING, Md., Oct. 14, 2011 /PRNewswire-USNewswire/ -- The U.S. Food and Drug Administration today approved Ferriprox (deferiprone) to treat patients with iron overload due to blood transfusions in patients with thalassemia, a genetic blood disorder that causes anemia, who had an inadequate response to prior chelation therapy.
Patients with thalassemia have excess iron in the body from the frequent blood transfusions (transfusional iron overload), a condition that is serious and can be fatal. These patients also have a risk of developing liver disease, diabetes, arthritis, heart failure or an abnormal heart rhythm.
The standard of care to treat transfusional iron overload is chelation therapy – chemical agents that are used to remove heavy metals from the body. Ferriprox is intended for use when chelation therapy is inadequate.
"Ferriprox represents the first new FDA-approved treatment for this disorder since 2005," said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research.
The safety and effectiveness of Ferriprox is based on an analysis of data from twelve clinical studies in 236 patients. Patients participating in the study did not respond to prior iron chelation therapy. Ferriprox was considered a successful treatment for patients who experienced at least a 20 percent decrease in serum ferritin, a protein that stores iron in the body for later use. Half of the patients in the study experienced at least a 20 percent decrease in ferritin levels.
The most common side effects seen in patients who received Ferriprox included nausea, vomiting, abdominal and joint pain, urine discoloration (chromaturia), a decrease in the number of white blood cells (neutropenia), and an increase in
|SOURCE U.S. Food and Drug Administration|
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