FDA approves Exjade (Novartis) to treat Non-Transfusion-Dependent Thalassemia

The FDA on 23 January 2013 expanded the approved use of Exjade (deferasirox) from Novartis, to treat patients ages 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. The FDA is also authorizing marketing of FerriScan 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.

This new indication is being approved under the FDA�s accelerated approval program, which provides patients earlier access to promising new drugs intended to treat serious or life-threatening illnesses while the company conducts additional studies to confirm the drug�s clinical benefit.