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Drug evaluation: Deferitrin for iron overload disorders.

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 clinical 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 clinical trial that began in September 2003 has reportedly completed recruitment.
AuthorsJames C Barton
JournalIDrugs : the investigational drugs journal (IDrugs) Vol. 10 Issue 7 Pg. 480-90 (Jul 2007) ISSN: 1369-7056 [Print] England
PMID17642018 (Publication Type: Journal Article)
Chemical References
  • 4'-hydroxydesazadesferrithiocin
  • Carboxylic Acids
  • Iron Chelating Agents
  • Thiazoles
Topics
  • Animals
  • Carboxylic Acids (adverse effects, chemistry, pharmacokinetics, therapeutic use)
  • Clinical Trials as Topic
  • Drug Evaluation, Preclinical
  • Humans
  • Iron Chelating Agents (adverse effects, chemistry, pharmacokinetics, therapeutic use)
  • Iron Overload (drug therapy)
  • Molecular Structure
  • Structure-Activity Relationship
  • Thiazoles (adverse effects, chemistry, pharmacokinetics, therapeutic use)

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