First oral iron chelator approved

Star: Novartis announced the approval of Deferasirox – the first and only once-daily oral iron chelator – by the US Food and Drug Administration. It has been approved for the treatment of chronic iron overload due to blood transfusions in adults and children age two and older.
Iron overload is a potentially life-threatening and unavoidable consequence of frequent blood transfusions used to treat certain types of rare chronic blood disorders, including thalassemia and sickle cell disease, as well as other rare anaemias and myelodysplastic syndromes.
Signs of iron overload may be detected after transfusion of about 20 units of blood. If left undiagnosed or untreated, the excess iron in the body is likely to lead to damage to the liver, heart and endocrine glands. The body has no inherent mechanism to remove excess iron, so iron chelation is used as an effective treatment for transfusion-related iron overload.
Deferasirox is the only iron chelator administered as a drink (the tablets are dispersed in a glass of orange juice, apple juice or water).
This is much more convenient for patients than the current standard of care, which often requires a subcutaneous infusion lasting eight to 12 hours per night, for five to seven nights a week for as long as the patient continues to receive blood transfusions or has excess iron within the body.
As a result, many patients may have stopped or avoided iron chelation therapy, thus risking the toxic effects of iron overload.
“The oral chelating agent will actually help,” says Prof Dr Zulkifli Ismail, President of the Thalassaemia Association of Malaysia, “making it so patients don’t have to have infusions any more. They don’t have to be stuck with tubes to their tummies eight hours a day. They can stop having needle phobia that they had before.”
Prof Dr Zulkifli adds: “It is long awaited. We have been waiting a long time for a once-daily oral chelator. A once a day oral medication is a great help.”
The approval of the drug is expected to greatly enhance the acceptance of iron chelation therapy, especially for children, and offer a new alternative to continuous infusion therapy.
“The approval by the US FDA is extremely good news for the Malaysian thalassaemia community. What we have been hoping for and waiting for has finally come to fruition, i.e. an effective oral chelator which will replace the burden of needles and daily infusions,” says Prof Chan Lee Lee.
She continues: “All the problems with patient refusal and poor compliance will dramatically change. As doctors we look forward to seeing happy thalassaemia patients who experience effective iron chelation leading to improved lifestyles and extended life spans.
“The drug should also prove to be useful for other patients who receive regular blood transfusions like patients with myelodysplastic syndromes.
“We hope that in the near future, many of our patients will be able to benefit from this new oral iron chelator.”
Designated an orphan drug in the US, Switzerland, Australia and the EU, Deferasirox has also been granted a priority review in Canada, Australia, and New Zealand. Additional regulatory submissions have been made around the world.