Ferriprox® (deferiprone) is used for the treatment of iron overload in patients with thalassemia. Such patients require long-term blood transfusions to treat their profound anemia; however, red blood cells are loaded with iron, and there is no physiologic pathway to eliminate excess iron from the body. Consequently, these patients are at risk for developing very high levels of iron in their circulation and vital organs. As the level of labile iron (iron not bound to proteins) rises, it begins to react with its environment to generate free radicals, which are toxic to proteins and membranes. The liver can tolerate fairly high levels of iron before it exhibits toxicity, but the heart and endocrine glands (pancreas, thyroid, hypothyroid, gonads, etc.), exhibit toxicity at much lower levels. Ferriprox works by binding to labile iron in the tissues and circulation, thereby inactivating it, and then removing it from the body, mostly via the urine. With this treatment, patients can continue to receive the blood transfusions they need to keep them alive without building up their body iron levels.
Currently, there are three iron chelators on the market. From a biochemical and pharmacological perspective, the Ferriprox molecule is unique in that it is much smaller than the other two, has a neutral charge, and can readily penetrate membranes to enter and remove labile iron from intracellular sites. Because there is a risk that some patients may experience agranulocytosis (a sudden decline in circulating neutrophils, a critical white blood cell for fighting infection), Ferriprox is approved as second line therapy in most countries. Depending on the country, Ferriprox is available in either tablet format, liquid format, or both.