The Cooley’s Anemia Foundation (CAF), the only national non-profit dedicated solely to fighting the genetic blood disorder thalassemia, applauds the September 14 decision of the Oncologic Drugs Advisory Committee (ODAC) to recommend to the Food and Drug Administration (FDA) approval of the new drug application of the oral iron chelator, Ferriprox. Thalassemia is much more common among people of Greek descent than among the general population.
The indication for Ferriprox is “for the treatment of patients with transfusional iron overload when current chelation therapy is inadequate.”
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.
“For more than a decade, the Foundation and the thalassemia patients 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.”
Ferriprox 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 which is administered orally. Ferriprox is also administered orally.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.
Dozens of letters and emails were sent from thalassemia patients 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, 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 significant 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 patients 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.”
Oral testimony presented by representatives from CAF at the ODAC meeting can be found at https://tinyurl.com/CAFFDA1.