* Exjade approved for non-transfusion-dependent thalassemia
* FDA also approves use of FerriScan as companion diagnostic
* Exjade approved for NTDT under accelerated approval
By Toni Clarke
Jan 23 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,
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.