The Cooley's Anemia Foundation testified before the US Food and Drug Administration (FDA) for the approval of the iron chelator deferasirox (Exjade) because of the dire need within the thalassemia community for an alternative to treatment by subcutaneous infusion of deferoxamine for the removal of transfusional iron overload.
Individuals born with thalassemia have been struggling for decades to maintain their health and extend their lives utilizing deferoxamine. For most, the burdensome method of administering deferoxamine has had a significant detrimental effect on their ability to maintain compliance with treatment; the drug is safe, but the method of administration seriously impacts its effectiveness.
In addition to the sheer physical difficulty of complying with deferoxamine treatment, many patients often experience psychosocial complications related to the drug and its administration. They feel "chained" to the infusion pump, which affects the image they have of themselves and of how they relate to the world around them. Living with this chronic disease makes them feel different from those around them, as if they do not fit in. The fact that their daily therapy has been more visible and obvious than simply taking an oral compound has added to their self-consciousness. They seek to hide the fact that they have an illness and develop a heightened sense of guilt or shame as a result. This contributes to the difficulty of maintaining compliance, further diminishing the efficacy of the prescribed chelation treatment.
The data pertaining to deferasirox that was submitted to the FDA indicated that efficacy would not be an issue. The Foundation and the thalassemia community looked to the FDA to verify this assessment. More importantly, we looked to the agency to evaluate the drug's safety profile and provide an independent assessment of the data. As long as deferasirox could be determined to be safe, we believed that the relative ease of administration would ensure significantly increased rates of compliance within the patient population.
The decision by the FDA to grant accelerated approval to deferasirox for use in the treatment of patients over the age of 2 with chronic iron overload due to blood transfusions was greeted with hope by members of the thalassemia community, who were excited that a new era of oral chelation had finally reached the United States.
Although this patient population in the United States is small, their needs are great and can vary significantly from patient to patient. Thus, there is a demand for a menu of drugs for alleviating transfusional hemosiderosis, from which physicians may tailor treatment that suits the needs of each patient. We know from experience in other countries, which have for some time had access to an oral chelating alternative, that the existence of options results in more effective treatment.
The approval of deferasirox is an important step forward in making individualized treatment regimens possible in this country. Physicians now have the option of two chelating agents; ideally, they will one day have even more options, as do doctors in Europe, who can choose between deferoxamine, deferasirox, and deferiprone when determining the most appropriate method of removing excess iron from an individual patient.
1. Borgna-Pignatti C, Cappellini MD, De Stefano P, et al: Cardiac morbidity and mortality in deferoxamine- or deferiprone-treated patients with thalassemia major. Blood 107:3733-3737, 2006.
2. Wood JC, Tyszka JM, Carson S, et al: Myocardial iron loading in transfusion-dependent thalassemia and sickle cell disease. Blood 103:1934-1936, 2004.
3. Drug Safety: Improvement Needed in FDA's Postmarket Decision-making and Oversight Process (Report to Congressional Requesters), pp 1-62. Washington, DC; US Government Accountability Office (GAO-06-402); March 2006.