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FDA approves deferiprone for patients with iron overload
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The FDA has today approved deferiprone to treat transfusional iron overload in patients with thalassemia who have exhibited inadequate responses to prior chelation therapy, according to a press release.
Safety and efficacy of deferiprone (Ferriprox, ApoPharma) was based on an analysis of 12 clinical studies involving 236 patients. The therapy was considered successful among patients who experienced at least a 20% decrease in serum ferritin levels. The most commonly reported side effects were nausea, vomiting, abdominal and joint pain, chromaturia, neutropenia and an increase in the level of a liver enzyme that may be indicative of tissue or liver damage at unsafe amounts. Half of the patients in the studies achieved those reduced ferritin levels. About 2% of patients, however, developed agranulocytosis, the agency said.
"Ferriprox represents the first new FDA-approved treatment for this disorder since 2005," Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research, said in the press release.
Deferiprone is being approved under the FDA's accelerated approval program, designed to provide patients with earlier access to promising new drugs followed by further studies to confirm clinical benefit. ApoPharma has made several postmarketing commitments, including the further study of deferiprone use in patients with sickle cell disease who have transfusional iron overload.
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