New Drug to Treat Thalassemia May Get Accelerated FDA Approval

A new drug to treat Thalassemia could soon be available.

On September 14, Oncologic Drugs Advisory Committee (ODAC) voted 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 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.

The Cooley’s Anemia Foundation (CAF), the only national non-profit dedicated solely to fighting the genetic blood disorder thalassemia, applauds this decision.

Ferriprox is used “for the treatment of patients with transfusional iron overload when current chelation therapy is inadequate.” It helps 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.

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.

“I cannot urge the FDA strongly enough to act on this Committee’s recommendation and approve this new drug application,” said CAF National President Anthony J. Viola. “Thalassemia is a very challenging disorder to treat…The availability of more chelating options is crucial for that individualization.” He stated that the drugs Desferal and Exjade are not effective for a significant portion of the patient population.

“The Cooley’s Anemia Foundation and the US 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,” added Viola.
Founded in 1954, the Cooley’s Anemia 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.