FDA Approves New Treatment Option for Patients with Thalassemia

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The Cooley's Anemia Foundation Applauds FDA Approval of Ferriprox, Iron Chelator for People with Thalassemia. The Foundation Believes a Wider "Menu" of Treatment Options is Crucial for Each Individual Patient to Receive Optimal Treatment

The Cooley’s Anemia Foundation (CAF), the only national non-profit dedicated solely to fighting the genetic blood disorder thalassemia, applauds a Food and Drug Administration (FDA) decision today to approve the new drug application for the oral chelator, Ferriprox. This action follows a 10-2 vote on September 14, 2011 by the Oncology Drugs Advisory Committee to recommend the approval of this drug.

“As the premier voice of the thalassemia community in the United States, we are thrilled that our patients will have the benefit of this drug which has proven beneficial to patients throughout the world,” says CAF National President Anthony J. Viola. “The FDA clearly responded to the overwhelming need for this drug in our patient population and has provided those patients needing daily drug therapy to remove iron, an option that has improved cardiac health and prolonged life in thousands of patents over the past decade.”

Ferriprox is an iron chelator, which is a drug that is used to help rid the body of excess iron, a serious and often fatal complication in thalassemia. Because individuals with the severe form of thalassemia are born with a life threatening anemia, they require lifelong blood transfusions as often as every two weeks. These transfusions overload the body with iron; if it is not removed, it settles in the organs, causing heart and liver failure, as well as numerous other complications.    
For many years, the only FDA-approved chelator was Desferal, which must be administered by pumping the drug into the body for 8-12 hours, 5-7 nights per week. In 2005, the FDA approved Exjade, a chelator that is administered orally.

Ferriprox is also an oral chelator. In addition to being easier to administer than Desferal, Studies published in Europe demonstrate that use of Ferriprox has been shown to protect the heart from iron accumulation, a crucial concern for individuals with thalassemia.
“The major cause of death in our patient population is iron-related heart failure,” says Viola. “And with a very significant percentage of our population unable to use either Desferal or Exjade, there is a vital need for another option; that option is Ferriprox.”

“We have seen too many patients die too young,” Viola concludes. “Our patients’ lives depend upon having more treatment options available to them. The FDA addressed the unmet medical needs of these desperately ill patients and provided approval for a drug that will help them. Their decision has the opportunity to extend our patient’s lives and significantly improve their quality of life.”

Founded in 1954, the Cooley’s Anemia Foundation (http://www.cooleysanemia.org; (212-279-8090) is the only national non-profit organization dedicated solely to thalassemia. The Foundation’s mission is advancing the treatment and cure for this fatal blood disease, enhancing the quality of life of patients and educating the medical profession, trait carriers and the public about Cooley's anemia/thalassemia major.

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Gina Cioffi
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