Fresh data presented at the 50th American Society of Haematology (ASH) Annual Meeting and Exposition in San Francisco, California, this week have further demonstrated the efficacy and safety of Novartis’ Exjade in treating patients with excessive levels of iron.

Iron overload is a potentially life-threatening condition that can occur in patients receiving regular blood transfusions, such as in those with disorders like thalassaemia or myelodysplastic syndromes (MDS). Exjade (deferasirox), which carries orphan drug status in Europe, was launched in 2006 as a breakthrough therapy for chronic iron overload, offering continuous chelation coverage for the removal of excess body iron in a single, oral, once-daily dose.

Now, for the first time, a cardiac substudy of EPIC – which, with 1,744 participants, is currently the largest prospective trial in iron chelation ever undertaken – has shown that Exjade was able to remove iron from the heart in a significant number of patients with beta-thalassemia (69.5% of 114), thereby meeting a “critical” treatment goal, according to Dudley Pennell, MD, Professor of Cardiology, Royal Brompton and Harefield NHS Trust and Imperial College.

In addition, an analysis of 341 patients with MDS taking part in the EPIC study showed that the drug also significantly cut levels of serum ferritin – a key measure of iron in the body. This finding is also important as earlier studies of transfusion-dependent MDS patients have found that high levels of serum ferritin have an impact on overall survival.

"This study, which includes the largest number of MDS patients of any iron chelation study, shows deferasirox can effectively reduce iron burden and is generally well tolerated when used appropriately to treat these patients," commented Norbert Gattermann, MD, PhD, Haematology, Oncology and Clinical Immunology, Heinrich Heine University Medical Centre, Dusseldorf, Germany.

Exjade was awarded the prestigious Prix Galien award - considered the industry's highest accolade for pharmaceutical R&D – in the orphan drug category in September this year. The drug pulled in third-quarter 2008 sales of $148 million, a 51% rise on the prior quarter.