Deferiprone is in clinical development for patients with sickle-cell disorder (SCD) and other anaemias that are suffering from iron overload due to frequent transfusions to increase their red blood cell count. SCD is a group of inherited disorders where the red blood cells become hard and sticky and look like a C-shaped farm tool called a “sickle”. The sickle red blood cells die early, and patients often require blood transfusions. Iron overload is an effect of frequent transfusions in SCD. Excess iron in the body can be toxic to major organs like the heart and liver.
Deferiprone is administered orally and can reduce iron overload by binding to it, forming a complex, which can then be excreted in the urine. This reduces iron levels in the body, preventing iron toxicity. If licensed, deferiprone would offer an additional first-line treatment for transfusional iron overload in sickle-cell disease and other anaemias.