Public Citizen urges US FDA to not approve ApoPharma's iron overload drug

The US Food and Drug Administration 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 (a unit of Canadian generics firm Apotex), failed to demonstrate that the drug is safe and effective in the intended patient population, said consumer advocacy group Public Citizen in a letter sent 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 USA 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.