Swiss drug giant Novartis AG (NVS) on Friday said the European Commission approved its drug Exjade for iron overload in patients with non-transfusion-dependent thalassemia or NTDT syndromes.
The company noted that Exjade (deferasirox) is the first oral treatment approved in the EU for chronic iron overload in patients with these types of thalassemia. These patients require chelation therapy when deferoxamine therapy is contraindicated or inadequate in patients aged 10 years and older.
Hervé Hoppenot, President, Novartis Oncology said, "This approval is a critical milestone for patients with NTDT syndromes. For the first time, Exjade will be available to thalassemia patients who are not regularly transfused but still suffer from the life-altering effects of excess iron."
The approval is based on results from the first prospective placebo-controlled study of iron chelation in NTDT patients, THALASSA, which showed a significant dose-dependent decrease in iron burden compared to placebo.
In this pivotal study, Exjade significantly reduced the concentration of iron in the liver, known as liver iron concentration, as well as the amount of iron anywhere in the body, measured by serum ferritin. Novartis added that overall adverse event rate for Exjade was similar to the placebo arm.
According to Novartis, Thalassemia refers to a diverse group of genetic disorders that affect red blood cell production, causing anemia. Those patients with NTDT syndromes don't receive regular transfusions, a significant cause of chronic iron overload.
Worldwide, at least three quarters of a million people have NTDT, which can lead to severe and life-altering complications due to excess iron accumulation.
Maria Domenica Cappellini, MD, Department of Internal Medicine, University of Milan, said, "NTDT syndromes can be complicated to manage. Without proper treatment, patients with NTDT can suffer severe and life-changing complications from chronic iron overload. The THALASSA data show that these patients now have an effective oral treatment option to help reduce iron concentration in the body."
Exjade is approved in more than 100 countries, including the US,Switzerland, Japan and countries comprising the EU. The approved indication may vary depending upon the individual country.
In Zurich, Novartis shares closed Thursday's trading at 57.95 Swiss francs, down 0.50 francs or 0.86 percent, on a volume of 3.8 million shares. On the NYSE, the shares settled at $63.76, down $0.19 or 0.30 percent.
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