Clinical Evaluation of Deferasirox (Exjade^®, ICL670)

Deferasirox is a once-daily oral iron chelator that is now available in more than 50 countries, including the European Union and the United States, for the treatment of chronic transfusional iron overload in adults and children. Approval of this novel agent was based on the results from a comprehensive series of clinical studies, the largest ever undertaken for an iron-chelating agent, involving more than 1,100 patients with a wide range of transfusion-dependent anemias. Achieving an effective, well-tolerated, and convenient therapy that provided 24-hour chelation coverage had proved difficult with previous therapies, making patients vulnerable to organ loading between doses of chelator. The long half-life and maintained plasma levels of deferasirox offer the potential to optimize treatment by improving the duration of chelation coverage. Dose-dependent decreases in both liver iron concentration and serum ferritin, with good tolerability across a range of transfusion-dependent anemias (children and adults), show that transfusional iron overload can be controlled with an acceptable therapeutic safety margin using deferasirox.