Committee Recommends Approval of Ferriprox to FDA

September 14, 2011 – The Oncologic Drugs Advisory Committee (ODAC) today recommended to the Food and Drug Administration (FDA) approval of the new drug application of the oral chelator, Ferriprox, a decision which CAF heartily applauds.


The indication for Ferriprox is “for the treatment of patients with transfusional iron overload when current chelation therapy is inadequate.” An iron chelator, Ferriprox is used to help rid the body of excess iron, a serious and often fatal complication in thalassemia.

The Committee voted 10-2 to recommend accelerated approval of Ferriprox; accelerated approval is employed when the drug is intended to treat a “serious or life-threatening illness” and carries with it the provision that the applicant will conduct clinical trials post-approval to provide additional information.

CAF National President Tony Viola

“For more than a decade, the Foundation and the thalassemia community in the United States have been waiting for this day,” says CAF National President Anthony J. Viola. “I am thrilled that this recommendation brings availability of this important treatment option for our patients and their doctors one step closer, and I cannot urge the FDA strongly enough to act on this Committee’s recommendation and approve this new drug application.”

During the presentation, Dr. Ellis Neufeld, Chair of CAF’s Medical Advisory Board and director of the thalassemia program at Children’s Hospital Boston, delineated the unmet medical need of the patient population and the clear need for more chelating options for this patient population. Dr. Neufeld pointed out that patients receiving chelation therapy continue to develop transfusion-related iron overload and that 25% of patients tretaed with 30-40 mg/kg of deferasirox for up to one year in the EPIC trial had no improvement in their ferritin level.

Dr. Neufeld also referenced Thalassemia Clinical Research Network data which show that 30% of patients on deferasirox and 25% of those on deferoxamine have inadequate chelation, and that 32% of patients have had clinical heart disease, as well as CAF data showing that 80 of 724 registered thalassemia patients died between 1998 and 2008.

More than 80 letters and emails were sent from the thalassemia community to the ODAC in support of this application, and representatives from CAF, including patients, parents and Board members, spoke at the meeting during the public comment section.

The Committee learned firsthand about the unmet patient need as patient/parent advocates described the ordeal involved with lifelong chelation therapy and the painful and often lonely struggle involved in living with thalassemia.

Nunzio Cazzetta

Nunzio Cazzetta, a CAF Board member and father of two deceased children with thalassemia, spoke eloquently of watching “his two sons die because chelating options came too late for them.”

“The last comment I heard from (my son Ralph’s) cardiologist concerned Ferriprox,” Mr. Cazzetta stated when relating his younger son’s death. “He said, ‘If we only had it sooner, he could have had a chance.’”

Another speaker, a 26-year-old woman who has obtained Ferriprox through a special “compassionate use” program, recalled the passing of a friend with thalassemia, with whom she had grown up and who “was never given the opportunity to turn his health around like I did (with Ferriprox)…I know that if he had had the options I have, he would be alive today.”


“Thalassemia is a very challenging disorder to treat,” says Viola. “In order to be effective, treatment must be specialized for each individual patient. The availability of more chelating options is crucial for that individualization, especially as many patients simply cannot remain compliant with Desferal and Exjade is not effective for a portion of the patient population.”

In addition, studies indicate that use of Ferriprox may provide a cardioprotective benefit; iron-related cardiac failure is a major cause of death in the thalassemia community.

“The Cooley’s Anemia Foundation and the U.S. thalassemia community have watched as the availability of Ferriprox in other countries has had a significant and major impact on quality and length of life,” says Viola. “We are excited that the FDA now has the opportunity to ensure that our patients enjoy similar benefits.”

The FDA will now consider the Committee’s recommendation and decide if it will act according to the recommendation; in most cases, the FDA generally follows the recommendations of its Committees.

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