Novartis drinks to Exjade after EU approval

By Gregory Roumeliotis

- Last updated on GMT

Swiss drugmaker Novartis is celebrating the approval of Exjade in
the EU for the treatment of patients with transfusional iron
overload, hoping for a blockbuster as the drug offers an oral
single daily-dose alternative to cumbersome infusions via pump.

A breakthrough in iron chelation therapy according to Novartis, Exjade, already available in the US, is administered once-daily as a drink, as opposed to the company's Desferal, which requires nightly infusions by needle and pump, often lasting eight to twelve hours per night for five to seven nights a week, as long as the patient continues to receive blood transfusions or has excess iron within the body.

Iron chelation is often necessary to prevent potentially life-threatening complications of excess iron in patients who receive regular blood transfusions for diseases such as thalassemia, myelodysplastic syndromes, sickle cell disease and other anemias.

Tens of thousands of children and adults around the world have these diseases. For many, the need for transfusions and chelation are life-long. A single dose of Exjade works throughout the entire day, removing excess iron - including highly toxic labile plasma (unbound) iron - from key organs such as the liver and heart.

"The approval of Exjade in the Europe Union as a new therapy for transfusional iron overload is most welcome, as this allows for the first time an effective, once daily oral monotherapy for transfusional iron overload,"​ said Professor John Porter from the Department of Haematology of University College London.

"This is also the first oral treatment available for transfusional iron overload where the dose response effect on iron balance has been systematically studied on a scale not previously undertaken with iron chelation therapy."

Due to the burdensome administration of deferoxamine, compliance with standard chelation therapy is poor.

However, with Exjade, the tablets are dispersed by stirring in a glass of water, orange juice, or apple juice (100-200 mL) until a fine suspension is obtained.

After the suspension has been swallowed, any residue must be resuspended in a small volume of water or juice and swallowed.

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