FDA approves deferasirox to remove excess iron in patients with genetic blood disorder

FDA has expanded the approved use of deferasirox (Exjade, Novartis) to treat patients aged 10 years and older who have chronic iron overload resulting from a genetic blood disorder called non-transfusion-dependent thalassemia (NTDT). 

NTDT is a milder form of thalassemia that does not require individuals to get frequent red blood cell transfusions. However, over time, some patients with NTDT are still at risk for iron overload that can lead to damage to vital organs.

FDA is also authorizing marketing of FerriScan (Resonance Health) as an imaging companion diagnostic for Exjade. The agency previously cleared FerriScan for measuring liver iron concentration (LIC), but its use in Exjade clinical studies to select patients for therapy, and to manage therapy, defined its role as an imaging companion diagnostic necessary for Exjade’s safe and effective use. FerriScan measures LIC non-invasively using magnetic resonance imaging.

About 1,000 people in the United States have thalassemia, according to the National Heart, Lung, and Blood Institute. Thalassemia conditions can cause the body to make fewer healthy red blood cells and less hemoglobin, a protein that carries oxygen to all parts of the body and returns carbon dioxide to the lungs so it can be exhaled. Some patients with thalassemia require frequent transfusions of red blood cells to maintain an acceptable level of hemoglobin. Iron overload is common in these patients.

Exjade was previously approved for treatment of chronic iron overload due to blood transfusions in patients aged 2 years and older, and this approval extends its use to treat patients with NTDT who show iron overload. Exjade should be used in patients with NTDT who have an LIC of at least 5 mg of iron per gram of dry liver tissue weight.

Exjade was approved under FDA’s accelerated approval process. Its approved is based on clinical data showing it can reduce LIC to less than 5 mg/g dry weight, a surrogate endpoint that is judged reasonably likely to predict a clinical benefit to patients.

The safety and effectiveness of Exjade to treat chronic iron overload in patients with NTDT were established in 2 clinical trials designed to measure the number of patients whose LIC was reduced to less than 5 mg/g dry weight after 52 weeks of treatment. In the first trial, 166 patients were randomly assigned to receive 5 mg/kg of Exjade, 10 mg/kg of Exjade, or a placebo daily. Results showed 15% and 27% of Exjade-treated patients achieved the target LIC, respectively, compared with 4% in placebo-treated patients. The second trial contained 133 patients from the first study who received an additional year of Exjade treatment or switched from placebo to Exjade treatment. Thirty-five percent of the evaluable patients in this extension trial achieved the target LIC.