This article is more than 5 years old. Information may no longer be current.
FDA approves Exjade for chronic iron overload resulting from NTDT
The FDA has expanded the approved use of deferasirox to treat patients aged at least 10 years with chronic iron overload as a result of non-transfusion-dependent thalassemia.
Deferasirox (Exjade, Novartis) was previously approved for treatment of chronic iron overload caused by blood transfusions in patients aged at least 2 years, and this approval extended its use to treat patients with non-transfusion-dependent thalassemia (NTDT) who exhibit iron overload.
Deferasirox has been approved for use in patients with NTDT who have a liver iron concentration of at least 5 mg of iron per gram of dry liver tissue weight.
Deferasirox’s new indication has been approved under the FDA’s accelerated approval program, based on clinical data demonstrating it can diminish liver iron concentration to less than 5 mg/g dry weight, a surrogate endpoint that is judged reasonably likely to predict a clinical benefit to patients.
Richard Pazdur
“Using our accelerated approval process, FDA is able to expedite the availability of this drug to patients who need to reduce excess iron,” Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, said in a press release. “Exjade is the first drug approved to treat patients with NTDT who show iron overload.”
The safety and effectiveness of deferasirox to treat chronic iron overload in patients with NTDT were established in two clinical trials designed to measure the number of patients whose liver iron concentration 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 deferasirox, 10 mg/kg deferasirox or a placebo daily. Results showed 15% of patients receiving 5 mg/kg deferasirox and 27% of patients receiving 10 mg/kg deferasirox achieved the target liver iron concentration vs. 4% in placebo-treated patients. The second trial included 133 patients from the first study who received an additional year of deferasirox treatment or switched from placebo to deferasirox treatment — 35% of patients in this extension trial achieved the target liver iron concentration.