Novartis’ Exjade (deferasirox) has received European approval as a treatment for non-transfusion-dependent thalassaemia (NTDT) syndromes, a group of genetic disorders characterised by red blood cell problems and anaemia.
The main treatment for thalassaemias is regular blood transfusions, which is a safe and effective therapy but tends to leave too much iron in the body.
Exjade (deferasirox) acts as an iron chelator, binding to and removing excess iron from the body and thereby preventing iron overload, which can lead to debilitating symptoms such as liver fibrosis and cirrhosis, blood clots, bone disease, pulmonary hypertension, and vascular and endocrine diseases.
The drug has already been used for some years to treat thalassaemia patients who depend on transfusions, but recent clinical trials have shown the product can also significantly decrease iron burden in NTDT patients compared to placebo, with similar overall adverse event rate.
The approval makes Exjade the first oral treatment available for NTDT in Europe, although the drug is already approved in more than 100 other countries to treat iron overload due to frequent blood transfusions, achieving $633m in sales in the first nine months of 2012.
“This approval is a critical milestone for patients with NTDT syndromes,” said Hervé Hoppenot, president of Novartis Oncology.
“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,” he added.
Approval in thalassaemia adds to the peak sales potential for Exjade and puts the medicine on track to meet its target of $1bn-plus in peak annual sales. The drug was first launched onto the market in 2005, becoming the first oral alternative to Novartis older injectable product Desferal (deferoxamine).