SCDAA Statement on Exjade® (deferasirox) Filing

The Sickle Cell Disease Association of America, Inc. (SCDAA) is excited by the news that Novartis has filed regulatory submissions with the US Food and Drug Administration for Exjade® (deferasirox), the first and only once-daily oral iron chelator for the treatment of chronic iron overload due to blood transfusions. The availability of a once-daily oral iron chelator has the potential to transform the treatment paradigm for people affected by Sickle Cell Disease (SCD).

Sickle cell disease is managed with supportive care—care that is aimed at improving a person’s quality of life by focusing on the psychological, emotional and physical concerns associated with the disease. Staying well hydrated, use of medication and regular blood transfusions are all important parts of the supportive care of SCD.

Regularly scheduled blood transfusions treat SCD by increasing the number of normal red blood cells in circulation, which in turn helps to reduce episodes of pain, risk of stroke and other complications. The value of transfusion is increasingly recognized in helping SCD patients. Based on the results of a pair of studies, the National Heart, Lung, and Blood Institute (NHLBI) recently confirmed that blood transfusions help to prevent strokes in children with SCD who are at risk of strokes.

However, repeated transfusions also cause excess iron to accumulate in the heart, liver, and other organs. This often leads to a potentially life-threatening condition called iron overload. If iron overload is left undiagnosed and untreated, it can lead to debilitating and life-threatening consequences, including damage to the liver, heart and endocrine glands—causing many clinicians, patients and parents to be concerned about transfusion therapy.

Excess iron can be removed by using an iron chelator, a medical treatment that binds to the iron and helps to remove it from the body. While patients have access to an infused iron chelation therapy, Desferal® (deferoxamine), many of them have been unable to comply with the treatment due to the lengthy and frequent subcutaneous infusions, which may cause pain, bruising and scarring.

Because of its convenience and ease of use, the SCDAA believes that the availability of Exjade will present a significant potential advancement in the treatment of chronic iron overload, and could help many patients who are unable to comply with the current standard of care, or who are not being treated for iron overload.

The SCDAA encourages sickle cell patients and their families to keep abreast of news regarding the availability of Exjade, and to use this as an opportunity to speak with their healthcare provider about how regular blood transfusions and iron chelation therapy fit into their treatment plans.

For More Information, please contact:
Sickle Cell Disease Association of America
16 S. Calvert St, STE 600
Baltimore, Maryland 21202
410-528-1555
scdaa@sicklecelldisease.org