FERRIPROX® (deferiprone) is an iron chelator indicated for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate. The approval in second line is reasonable both because the studies for approval were done in second line and because Ferroprox may be inferior to Exjade in first line (Cemak et al). This is not an innocuous drug; the most serious side effect seen in about two percent of patients treated with Ferriprox was the development of agranulocytosis, a serious and potentially life-threatening reduction in the number of granulocytes (a type of white blood cell that fights infection). The therapy 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 the drug’s clinical benefit. The accelerated approval program allows the agency to approve a drug to treat a serious disease based on clinical 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).
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.
For Lay version see here
At this time, there is not sufficient literature support for the use of Ferriptox in sickle cell disease.
Cermak, Jaroslav,2011, http://www.eventure-online.com/eventure/publicAbstractView.do?id=161926&congressId=4634
Ferriprox, Prescribing Information, 2013