An Exhaustive Review of Deferiprone (DB08826): From Physicochemical Properties to Clinical Application and Risk Management

Executive Summary

Deferiprone (DrugBank ID: DB08826) is an orally active, small-molecule iron chelator that occupies a critical and specialized niche in the management of transfusional iron overload. This report provides a comprehensive analysis of Deferiprone, synthesizing data on its chemical nature, pharmacology, clinical efficacy, safety profile, regulatory history, and comparative standing against other iron chelators.

Chemically identified as 3-hydroxy-1,2-dimethylpyridin-4-one, Deferiprone is a bidentate ligand that forms a stable, neutral 3:1 complex with ferric iron, facilitating its excretion primarily through the urine. Its classification as a Biopharmaceutics Classification System (BCS) Class 1 drug, characterized by high solubility and high permeability, underpins its excellent oral bioavailability—a significant advantage over the parenteral administration required for the first-generation chelator, Deferoxamine. Furthermore, its small molecular size and lipophilic character enable it to cross cellular membranes, including the blood-brain barrier and, most critically, the myocardial cell membrane.

This ability to access intracellular iron pools is the cornerstone of Deferiprone's unique clinical value. It has demonstrated particular efficacy in removing iron from the heart, a key site of toxicity and the leading cause of mortality in patients with transfusional iron overload, such as those with thalassemia major and sickle cell disease. Clinical evidence, increasingly supported by advanced imaging techniques like T2* magnetic resonance imaging (MRI), confirms its superior cardioprotective effect compared to other chelators, solidifying its role as a vital agent for patients with or at high risk of cardiac siderosis.