Ferriprox for treatment of sickle cell anemia

FERRIPROX® (deferiprone) is an oral medication designed to chelate, or remove,  iron overload from transfusions due to thalassemia syndromes when other chelation therapy, most commonly Exjade,  does not work. The studies for approval were done for teh situation when teh initial chaltion did not work (second line) because Ferroprox may be inferior to Exjade in first line. This is not an innocuous drug; the most serious side effect seen in about two percent of patients treated with Ferriprox was low white counts, a serious and potentially life-threatening complication. This drug is being approved under the FDA’s accelerated approval program, designed to provide patients with earlier access to promising new drugs, that had not been fully studied and thatmust  followed by further studies to confirm the drug’s benefit. The accelerated approval program allows the agency to approve a drug to treat a serious disease based on initial data showing that the drug has an effect on an endpoint that is reasonably likely to predict a clinical benefit to patients, or on an effect on a clinical endpoint other than survival or irreversible morbidity (illness). The assumption that these parameters can be counted on to predict ultimate effectiveness is not universally accepted.

ApoPharma has agreed to several post-marketing requirement and commitments. One commitment includes further study of the use of Ferriprox in patients with sickle cell disease who have transfusional iron overload.  One such study is: Absorption, Metabolism, and Excretion of a Single Dose of Ferriprox® in Patients With Sickle Cell Disease,  NCT01835496.

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