ApoPharma, a pharmaceutical company specializing in the discovery and development of new medicines for critical diseases, announced that Health Canada has granted approval of Ferriprox (deferiprone), an oral iron chelator for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate.

"We are very pleased that Health Canada has responded positively to the extensive clinical data and track record associated with Ferriprox," says Dr Michael Spino, president, ApoPharma, Inc. "This drug will provide a critically important new treatment option for individuals who have not had a good response with another therapy."

At any given time in Canada, approximately 400 patients with the inherited blood disorder thalassemia require blood transfusions for survival. While repeated blood transfusions are life-saving for many patients, they result in an accumulation of iron in blood and in organs such as the heart and liver. Without effective treatment, iron overload ensues and can lead to organ failure and early death.

Not all patients have their iron burden successfully controlled by the currently available treatment options. Approximately one quarter of thalassemia patients fall into this category, according to research conducted by the NIH-funded Thalassemia Clinical Research Network.

Clinical studies and more than 15 years of international post-marketing surveillance totaling approximately 58,000 patient years of exposure have demonstrated that Ferriprox can control the iron burden in patients who are transfusion dependent. In the pivotal clinical trial Ferriprox was twice as effective as deferoxamine in removing cardiac iron. Analysis of studies sponsored by ApoPharma shows that one year of Ferriprox therapy met the targeted reduction of serum ferritin levels (a measure of total body iron) in approximately half of the patients who had failed previous therapy.

Ferriprox is currently approved in more than 65 countries worldwide for the treatment of iron overload in patients with thalassemia major when the standard therapy is contraindicated or inadequate. With the present approval, patients whose current therapy is unsatisfactory can be treated with Ferriprox as an established alternative.

One to two per cent of Ferriprox patients develop agranulocytosis, a decline of certain white blood cells (neutrophils) that may put them at risk of serious infections. ApoPharma is introducing a programme designed to mitigate the potential risks among patients taking Ferriprox, similar to a programme implemented in the US in 2011.