FDA Approves Ferriprox to Treat Excess Iron in the Body

The drug Ferriprox (deferiprone) was approved today by the US Food and Drug Administration (FDA) as a treatment for patients suffering from excess iron in their bodies.

This is usually observed as a result of blood transfusions in patients with thalassemia (a genetic blood disorder that causes anemia) who had an inadequate response to prior chelation therapy.

Thalassemia patients suffer often from iron overload resulting from the frequent blood transfusions their disease requires. Transfusional iron overload is severe and can be fatal.

In addition to suffering from iron excess, such patients may also be at risk of developing liver disease, diabetes, arthritis, heart failure, or an abnormal heart rhythm.

The typical treatment of transfusional iron overload is chelation therapy, a process which involves removing heavy metals from the body using chemical agents. When this therapy is ineffective, Ferriprox is supposed to be a substitute.

Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research reported, “Ferriprox represents the first new FDA-approved treatment for this disorder since 2005.”

The FDA evaluated the data from 12 clinical studies involving 236 patients in order to assess the safety and efficacy of Ferriprox.

The participants of the studies did not respond to prior iron chelation therapy. The researchers involved determined Ferriprox to be a successful treatment for patients who experienced at least a 20% decrease in serum ferritin, a protein that stores iron in the body.

About 50% of the participants achieved at least a 20% decrease in ferritin levels.

The most frequent side effects observed included nausea, vomiting, abdominal and joint pain, urine discoloration, a decrease in the number of white blood cells, and an increase in the level of a liver enzyme (a possible indication of tissue or liver damage at unsafe amounts).

The most severe side effect observed in roughly 2% of the participants on Ferriprox was the development of agranulocytosis, a serious and potentially life-threatening reduction in the number of infection-fighting white blood cells known as granulocytes.

The drug has been approved as a part of the FDA’s accelerated approval program in order to offer patients fast access to the promising new treatment; further studies will be performed to substantiate its clinical advantage.

ApoPharma Inc. of Toronto, marketer of Ferriprox, has made an agreement with the agency to pursue further study of the use of the drug in patients with sickle cell disease who have transfusional iron overload.