ApoPharma Inc. today announced that the Oncologic Drugs Advisory Committee (ODAC) to the U.S. Food and Drug Administration (FDA) voted 10 - 2 to recommend that the FDA grant accelerated approval of Ferriprox® (deferiprone), an oral iron chelator, for the treatment of patients with transfusional iron overload when current chelation therapy is inadequate.
"We are very pleased that ODAC members responded positively to the totality of the clinical data and to Ferriprox's established track record," said Dr. Michael Spino, President, ApoPharma Inc. "If approved, this drug will provide a critically important new treatment option to individuals whose current chelation is inadequate."
Under the Prescription Drug User Fee Act (PDUFA), the FDA is expected to act on the New Drug Application for Ferriprox by 14 October 2011. Although the FDA usually follows ODAC recommendations, it is not obligated to do so.
At any given time in the United States, approximately 500 to 1,000 patients with the inherited blood disorder thalassemia, require regular blood transfusions to survive. There also is a growing number of patients with sickle cell disease (SCD), who require transfusions to prevent complications associated with their condition. While repeated blood transfusions are life-saving for many patients, these transfusions can result in toxic iron levels in their blood and organs such as the heart, and liver. Without effective treatment, iron overload can lead to organ failure and early death.
Approximately one quarter of thalassemia patients are not able to manage iron overload with currently available treatment options, according to research conducted by the NIH-funded Thalassemia Clinical Research Network.
In clinical studies, publications and data generated by ApoPharma, including 34,000 patient years of exposure over more than a decade, Ferriprox has demonstrated that it can control the iron burden in patients who are transfusion dependent. In a review of patients who failed previous chelation in studies sponsored by ApoPharma, 52 percent were successfully treated with Ferriprox, as assessed by serum ferritin levels, a measure of total body iron.
In making their recommendation, the Committee noted that ApoPharma should conduct additional clinical trials of Ferriprox to confirm safety and efficacy in patients with iron overload, emphasizing the need for such studies in patients with SCD.
Ferriprox is approved in 61 countries worldwide for the treatment of iron overload in patients with thalassemia major when the standard therapy (deferoxamine) is contraindicated or inadequate. If the FDA accepts the recommendation of ODAC, the approval will permit the treatment of iron overloaded patients who have failed under current therapy to be treated with Ferriprox.
One-to-two percent of Ferriprox patients develop agranulocytosis, a decline of certain white blood cells (neutrophils) that may put patients at risk for infections. In the event that FDA follows ODAC's recommendation, ApoPharma has proposed to introduce an education program designed to mitigate the potential risks among patients taking Ferriprox similar to a successful program implemented in Europe.
SOURCE ApoPharma Inc.