Exjade Approved to Remove Excess Iron in Patients with Non-Transfusion-Dependent Thalassemia

Drug Alerts , News / October 1, 2016

The FDA has expanded the approved use of Exjade (deferasirox) 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 transfusion. However, over time, some patients with NTDT are still at risk for iron overload than can lead to damage to vital organs. Generally, 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 that 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 milligrams of iron per gram of dry liver tissue weight.

The safety and effectiveness of Exjade to treat chronic iron overload in patients with NTDT were established in two 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.

Exjade is marketed by East Hanover, N.J.-based Novartis.

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