HealthDay News — The iron chelator drug deferasirox (Exjade, Novartis) is approved to reduce iron overload in thalassemia patients aged 10 years and older, who do not require regular transfusions, the FDA announced in a press release.
The drug was previously approved to remove excess iron in transfusion-dependent thalassemia patients. The expanded indication now covers nontransfusion-dependent thalassemia (NTDT) patients with liver iron concentrations of at least 5 mg/g of dry liver tissue.
Thalassemia typically leads to the production of fewer red blood cells and less hemoglobin. NTDT is a milder form of thalassemia that, unlike other forms, does not require frequent blood transfusions. Thalassemia affects about 1,000 people in the United States, the FDA said.
The expanded indication was based on results from a clinical trial that involved 299 patients. The primary efficacy endpoint was liver iron concentration less than 5 mg/g. After one year, 15% of patients assigned to deferasirox 5 mg/kg and 27% of those assigned to a 10 mg/kg dose met the endpoint, compared with 4% in the placebo group.
The FDA approved Exjade under it’s fast track program. Drug maker Novartis is required to complete additional post-marketing studies to assess clinical benefit.