thalassemia
The FDA has approved deferiprone (Ferriprox) to treat iron overload in thalassemia patients who had an inadequate response to prior chelation therapy.
Deferiprone is the first treatment for transfusional iron overload to be approved since 2005, said Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.
The drug’s approval is based on a review of data from 12 clinical studies in 236 patients. Patients participating in the studies did not respond to prior iron chelation therapy.
Deferiprone was considered a success if patients experienced at least a 20% decrease in serum ferritin. And half of the patients included in the review experienced at least a 20% decrease in ferritin levels.
The most common side effects of the drug 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. The most serious side effect, seen in about 2% of patients, was the development of agranulocytosis.
Deferiprone has been approved under the FDA’s accelerated approval program, which was designed to provide patients with earlier access to promising new drugs, followed by further studies to confirm the drug’s clinical benefit.
ApoPharma, the company that manufactures deferiprone, has agreed to several post-marketing requirements and commitments. One commitment includes further study of the use of deferiprone in patients with sickle cell disease who have transfusional iron overload.
