FDA approval of Ferriprox for treatment of transfusional iron overload due to sickle cell disease.- Chiesi Global Rare Diseases.

Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group),has announced that the FDA has approved Ferriprox (deferiprone) for the treatment of transfusional iron overload due to sickle cell disease (SCD) or other anemias in adult and pediatric patients 3 years of age and older. This FDA approval expands the use of Ferriprox for patients with SCD or other anemias as well as patients with thalassemia regardless of prior iron chelation exposure. SCD affects about 100,000 people in the U.S. and leads to a lower life expectancy by more than 20 years compared to the general population. SCD patients are typically diagnosed prior to 2 years of age and the more severe patients start to experience pain crises early in childhood. Many require hospitalization and chronic blood transfusions to manage disease complications. Disease complications include early onset stroke, acute chest syndrome, and multiorgan failure. Renal complications affect nearly 30-50 per cent of patients with sickle cell anemia and account for 16-18 percent of mortality in adults with SCD.