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Amy Celento Testimony to ODAC in Support of New Drug Application #021825 for Ferriprox Good morning. My name is Amy Celento and I am the parent of a child with thalassemia major, as well as the volunteer Vice President of the Cooley’s Anemia Foundation. The Foundation has received minimal event sponsorship funding from Apo-Pharma. Thank you for your time and interest in my remarks supporting FDA approval of Ferriprox. My 13-year old son James is currently using the FDA-approved oral chelator Exjade, and has done so for 4 years. Prior to Exjade, James used Desferal for 4 years. While James is doing well on Exjade, I cannot say that either one of these options is ideal. The difficulty of administering Desferal is well known and inevitably leads to serious compliance issues which in turn compromise the drug’s effectiveness. While much easier to administer, there are drawbacks with Exjade which, especially in children, can impact compliance. For James, he had trouble taking Exjade every morning; he was resistant to its taste and texture and the need to wait a half hour before eating. It was only after he enrolled in a study to see whether Exjade could be taken with food, or with a wider variety of liquids, that James was able to consistently tolerate Exjade. We’re fortunate James’ problems were resolved, but it just emphasizes how, even after a drug has been approved, there are obstacles that can interfere with effectiveness. As is well documented, both of the currently approved chelators have the potential to cause a wide range of side effects. My son undergoes a battery of tests to determine if there are complications arising from chelation. Some side effects, such as hearing or vision loss, are not life threatening, but would have a significant impact on his life quality. Other significant concerns that have been reported in the thalassemia community include severe GI problems, sometimesrequiring cessation of therapy due to bleeding ulcers. For roughly 35% of the patients, efficacy issues with Exjade mean that Desferal is currently their only chelation option. I am acutely aware of the Institute for Safe Medication Practices 2009 study of frequent suspect drugs in reported patient deaths, which led the FDA to strengthen the warning label for Exjade. This shows that even a meticulously studied drug can demonstrate unforeseen dangerous side effects. When looking at Ferriprox, I believe that its long term clinical use demonstrates safe responsible use of the drug. The 1% potential for neutropenia leading to agranulocytosis, while frightening ,can be managed through weekly blood counts which we support. This side effect is not only rare but predictable, manageable and reversible and therefore should not impede the drug’s approval. It’s well known that most medications come with the potential of causing some side effect in some individuals. The risk:benefit ratio is always a consideration. However, I have seen that the existence of potential side effects can be less important than having a working system for monitoring and handling such side effects; this Ferriprox clearly has, and I request that you recommend approval of Ferriprox to the FDA. Thank you.