alexa Drug evaluation: deferitrin (GT-56-252; NaHBED) for iron overload disorders.


Journal of Clinical & Experimental Cardiology

Author(s): Barton JC, Barton JC

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Abstract Deferitrin (GT-56-252) is the first drug in a class of desferrithiocin-derived hexadentate iron chelators. Genzyme Corp is developing this compound as an oral drug for the treatment of severe iron overload in people who require repeated erythrocyte transfusion for management of chronic anemia such as beta-thalassemia major. In phase I trials in adults with beta-thalassemia, deferitrin promoted iron excretion in a dose-related manner and was well tolerated as both a liquid and capsule in fed and fasted states. There were no serious adverse events or significant laboratory abnormalities. The author concludes that deferitrin may be useful as chelation monotherapy or as part of combination or doublet chelation therapy for the treatment of severe iron overload in patients with beta-thalassemia major if its favorable pharmacokinetic profile, efficacy, safety and tolerability are confirmed in more extensive clinical trials. A phase I/II trial that began in September 2003 has reportedly completed recruitment.
This article was published in IDrugs and referenced in Journal of Clinical & Experimental Cardiology

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