Ferriprox (deferiprone), an oral drug has been approved for treating Thalassemia patients with excess iron due to blood transfusions. Thalassemia, a genetic blood disorder that causes anemia, necessitates the affected patient to have frequent blood transfusions. This leads to a serious, often fatal condition called iron overload. Excess iron can have a number of deleterious effects on the body. The risk of developing liver disease, diabetes, arthritis, heart failure, or an abnormal heart rhythm is higher in patients with iron overload.
Transfusional iron overload is usually treated with chelation therapy. Chelation therapy uses chemical agents that can remove heavy metals from the body. The modality does not work for all patients. Ferriprox has been approved for use when chelation therapy is inadequate.
AdvertisementData from 12 clinical studies in 236 patients were analysed. Patients participating in the study did not respond to previous iron chelation therapy. Deferiprone was found to bring a 20% decrease in serum ferritin, a protein that stores iron in the body for later use. The drug was found to successfully reduce ferritin levels in half of the patients in the study.
The U.S. Food and Drug Administration (FDA), has listed the following side effects of Ferriprox:
• Abdominal and joint pain
• Urine discoloration (chromaturia)
• A decrease in the number of white blood cells (neutropenia)
• An increase in the level of a liver enzyme that may be indicative of tissue or liver damage at unsafe amounts
2% of patients treated with Ferriprox developed a serious and potentially life-threatening condition called agranulocytosis. The number of granulocytes (a type of white blood cell that fights infection) falls dangerously in agranulocytosis.
Further uses of the drug are being probed into. ApoPharma, the company behind Ferriprox, has committed to study the effect of the drug in patients with sickle cell disease who have transfusional iron overload.
Reference: U.S. Food and Drug Administration (FDA)