Novartis' Exjade is now available in the UK for the removal of iron in patients with a certain form of the genetic blood disorder thalassaemia, expanding its use in the country.

Following a decision by European regulators last year, Exjade (deferasirox) is now available in the UK to combat iron overload in patients aged 10 years and over with non-transfusion-dependent thalassaemia (NTDT), the current standard treatment desferrioxamine is contraindicated or inadequate.

Exjade first won European approval back in 2006 for thalassaemia patients with iron overload as a result of regular blood transfusions, and is also indicated for use in those with irregular transfusions in certain circumstances.

Excess iron can also build up in NTDT patients via intestinal absorption, leaving them at risk from debilitating health complications such as liver fibrosis and cirrhosis, blood clots, bone disease, pulmonary hypertension and vascular and endocrine diseases.

First oral option

Until now, the only option for these patients has been treatment with desferrioxamine, but this requires slow infusions under the skin and "many patients with this form of anaemia find [the therapy] difficult to take", said Professor John Porter, Consultant Haematologist at University College London.

“The recent licensing of Exjade for the treatment of these patients offers an orally available treatment for the first time," and this "should allow better control of iron overload and fewer complications from excess body iron,” he noted.

“The availability of Exjade in the UK marks an important milestone in improving the outcomes of patients with NTDT," added Panos Alexakos, Oncology General Manager, Novartis UK & Ireland, stressing that it "has the potential to lessen the debilitating health complications that can result from chronic iron overload".