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Oct 12, 2011

Public Citizen Urges the FDA to Reject Approval of an Inadequately Tested Experimental Drug for Iron Overload

Serious Doubts Raised About the Drug’s Efficacy and Toxicity

WASHINGTON, D.C. – The U.S. Food and Drug Administration (FDA) should not approve an experimental drug proposed to treat patients who develop iron overload following multiple transfusions for anemia because data presented by the drug’s manufacturer, ApoPharma, failed to demonstrate that the drug is safe and effective in the intended patient population, Public Citizen said in a letter sent today to the agency. Public Citizen’s stance is in agreement with a similar conclusion by a noted Harvard researcher.

The drug, deferiprone, also known as Ferriprox, is taken orally and binds to iron in the blood, which is then excreted by the kidneys into the urine. It is intended for use in patients with certain types of anemia – such as thalassemia, sickle cell disease and myelodysplastic syndrome – who develop iron overload. In iron overload, excess iron accumulates in the liver, heart and other organs, causing damage. The standard FDA-approved treatment for iron overload in the U.S. is the deferoxamine, which has been well-studied in many clinical trials.

In November 2009, the FDA rejected approval of experimental drug deferiprone because data presented by ApoPharma failed to provide sufficient evidence that the drug was safe and effective. At that time, the FDA advised ApoPharma to conduct at least one additional prospective, randomized, controlled study of the drug.

The FDA subsequently acquiesced to pressure from ApoPharma and allowed the company to seek approval of deferiprone without conducting any additional prospective clinical studies. Instead, the agency granted accelerated approval status for the drug based solely on a retrospective analysis of data from a subset of subjects enrolled in 12 previously conducted clinical trials of deferiprone that varied widely in their study designs.

“FDA approval of deferiprone based on such inadequate data would set a recklessly dangerous precedent for drugs reviewed under an accelerated approval process in the future,” said Dr. Michael Carome, deputy director of Public Citizen’s Health Research Group.

In comments submitted in August to the FDA, Dr. David G. Nathan, a renowned hematology expert at Harvard Medical School with expertise on iron overload, stated, “I wish to state my firm opposition to such a decision [to approve deferiprone] because deferiprone has never been subjected to an appropriate clinical trial, and there are serious doubts about its efficacy and concerns about its toxicity … Since there is an adequate (albeit imperfect) oral iron [binder] available to U.S. patients that has undergone excellent clinical trials, I oppose the licensing of deferiprone until the company mounts a respectable clinical trial that persuades those of us who have devoted our careers to transfusion-induced iron overload that the drug is efficacious and safe.”

A decision by the FDA regarding deferiprone is considered imminent.

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