Novartis received the European Commission (EC) approval for Exjade (deferasirox) as a new treatment to help patients with transfusional iron overload in all 25 member states of the European Union (EU).
The approval brings to children and adults with a broad range of transfusion-dependent anaemias the only oral iron chelator to provide continuous chelation coverage with a single daily dose. The current standard of care is a cumbersome infusion via pump that often lasts eight to 12 hours and must often be done daily, a Novartis press release stated.
Exjade is the first oral iron chelator approved in the EU for use in patients with transfusional iron overload who have a wide range of underlying diseases. Exjade is approved in the EU for the treatment of chronic iron overload due to frequent blood transfusions in patients age six and older with beta thalassemia major.
It is also indicated in the EU for the treatment of chronic iron overload when the medicine deferoxamine is contraindicated or considered inadequate in patients with other anaemias, children age two to five years and patients with beta thalassemia major with iron overload due to infrequent blood transfusions
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 anaemias.
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.
A breakthrough in iron chelation therapy, Exjade is administered once-daily as a drink. Exjade was developed specifically to meet the high unmet medical need for iron chelation despite the availability of deferoxamine, the standard iron chelator used around the world.
Already approved in 29 countries, Exjade was granted approval after the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMEA) issued a positive opinion recommending marketing authorization. Designated an orphan drug in the EU, US, Switzerland and Australia, additional regulatory submissions for Exjade have been made around the world.