FDA approves Exjade in patients with genetic blood disorder

Wed Jan 23, 2013 3:30pm EST

* Exjade approved for non-transfusion-dependent thalassemia

* FDA also approves use of FerriScan as companion diagnostic

* Exjade approved for NTDT under accelerated approval program

By Toni Clarke

Jan 23 (Reuters) - U.S. regulators said on Wednesday they have approved Novartis AG's drug Exjade to remove excess iron in patients over the age of 10 who have a genetic blood disorder known as non-transfusion-dependent thalassemia, or NTDT.

Exjade was approved in the United States in 2005 to treat chronic iron overload resulting from blood transfusions in patients ages two and older. The new approval allows Novartis to also market the drug for NTDT.

Patients with thalassemia have an abnormal form of hemoglobin, the protein in red blood cells that carries oxygen. The malfunction leads to the presence in the body of fewer red blood cells than normal. As a result patients may develop severe anemia and need regular blood transfusions.

NTDT is a milder form of thalassemia that does not require frequent transfusions. Patients with the condition are nonetheless at risk of iron overload, which can lead to damage of various organs and potentially premature death.

The U.S. Food and Drug Administration also authorized the use of FerriScan, made by Resonance Health of Australia, as a companion imaging diagnostic to measure liver iron concentrations and help select patients for Exjade therapy and monitor their response to the drug.

The new approval was given under the FDA's accelerated approval program, which gives patients earlier access to promising new treatments intended to treat serious or life-threatening illnesses while the company conducts additional studies to confirm its clinical benefit.

Exjade was approved based on clinical data showing it can reduce liver iron concentrations, which the FDA judged reasonably likely to predict a clinical benefit to patients.

Citing the National Heart, Lung, and Blood Institute, the FDA said about 1,000 people in the United States have thalassemia. Exjade is the first product to be approved to treat patients with NTDT who develop an iron overload.