SCDAA Responds Favorably to Positive Review of Exjade by FDA Advisory Committees

The Sickle Cell Disease Association of America (SCDAA) is excited by the news that the Blood Products Advisory Committee (BPAC) of the U.S. Food and Drug Administration (FDA) has given a positive review of Exjade® (deferasirox) for once-daily oral treatment of adults and children with chronic iron overload due to blood transfusions.

Specifically, the committee recommended use of Exjade for patients who undergo repeated blood transfusions to treat sickle cell disease, and may be at risk for iron overload. This recommendation brings us one step closer to the availability of a once-daily oral iron chelator, which has the potential to transform the treatment paradigm for people affected by Sickle Cell Disease (SCD).

As a demonstration of the SCDAA’s ongoing commitment to its constituents and their well being, Dr. Frempong, Chairman of the SCDAA Board and Dr. Willarda Edwards, president and COO, spoke on behalf of the Association at the FDA meeting during the public session. Their focus included the importance and benefits of having a wide range of medical options available for people affected by SCD and iron overload.

Sickle Cell Disease and Iron Overload

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.

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.