Iron-chelating therapy with the new oral agent ICL670 (Exjade)

Abstract

Iron-chelation therapy is essential in iron-overloaded patients. The long-term efficacy of this approach has been demonstrated unequivocally with deferoxamine, which effectively controls iron concentrations, thereby limiting organ damage and preventing premature death. However, as deferoxamine is administered parenterally, poor compliance is a significant limitation to successful patient management. ICL670 (Exjade) is a highly efficient and selective oral iron chelator. The results of phase I and phase II clinical studies indicate that ICL670 is at least as effective as current standard therapy (deferoxamine), with a tolerability and safety profile suitable for chronic, once-daily administration. These encouraging results have provided the rationale for the ongoing phase III programme, and offer physicians and patients the promise of an oral, once-daily, iron-chelating agent for the prevention and management of iron overload.