Novartis’ Exjade - a breakthrough treatment for patients with iron overload as a result of blood transfusions - is about to hit shelves in the UK, following its regulatory nod in Europe at the end of August.

Iron overload is a potentially life-threatening condition that can manifest itself in several ways – heart failure, diabetes and osteoporosis, for example - in patients receiving regular blood transfusions, such as those with disorders like thalassaemia or sickle cell disease. “Every year people die from iron overload,” Dr Shah told delegates at Exjade’s (deferasirox) launch meeting in London yesterday, “but this is entirely preventable,” she stressed.

Exjade, which carries orphan drug status in Europe, has been hailed a breakthrough therapy as it is currently the only iron chelator on the market that provides continuous chelation coverage for the removal of excess body iron in a single dose. Moreover, the drug comes in the form of a once-daily drink, offering an important advantage over older therapies on the market, which come with difficult dosing regimens.

The main problem with earlier therapies lies in treatment compliance. While the previous standard of care, Novartis’ own Desferal (deferoxamine), is effective, the product requires an infusion via a pump and needle in the stomach that can last up to 12 hours a day, “and this really does affect treatment compliance,” Shah explained. Furthermore, effectiveness of the treatment drops dramatically if patients don’t stick to the heavy dosing regimen, which can involve infusions every night of the week in some cases. Therefore, an orally active alternative is expected to greatly appeal to patients and consequently boost treatment outcomes as well as grow the market.

But does this spell the end for Desferal? Not necessarily, says Ian Hayes, Medical Director for Oncology at Novartis. He told PharmaTimes that, while Exjade is undoubtedly the more attractive treatment option, some patients are still better suited to Desferal. It’s just a case of whatever suits the patient best, he said.

Exjade has been approved in 29 countries around the world, including the USA where it received the go-ahead last November, and has been designated an orphan drug in the EU, Switzerland, Australia and America.

A spokeswoman for Novartis told PharmaTimes that, in the UK, the cost of the medicine for an adult weighing 70kg is around £17,122 a year, while for that of a child weighing 35kg it is £8561. According to Professor John Porter, from the Department of Haematology at University College London, this puts the expense of the drug somewhere in between the two available treatment options for Desferal.

Desferal sales have stayed fairly flat over the last several years, bringing in upwards of $100 million dollars a year for the Swiss firm. But Exjade is thought to offer greater potential and should expand the market for iron chelators by extending treatment to patients unwilling to take the Desferal regimen, particularly children, according to Novartis.

The firm said last year that it was expecting peak sales of $650-$750 million for Exjade, but other predictions have placed estimates as high as $1 billion.