Cooley's Anemia Foundation Announces Support Of Deferiprone; Cites Two New Publications Demonstrating Safety And Efficacy.

Business Editors, Medical Writers

FLUSHING, N.Y.--(BUSINESS WIRE)--Aug. 16, 2002

The National Cooley's Anemia Foundation (CAF) and its patient advocacy arm, the Thalassemia Action Group (TAG), announced their support for deferiprone, an orally active iron chelator that is expected to be submitted to the federal Food and Drug Administration (FDA) for approval this year. The Foundation is encouraged in its efforts by a pivotal study published today in a leading medical journal, the Lancet, as well as by an article to be published in Blood, September 2002, both of which support the efficacy and the safety of the drug.

"We are particularly encouraged by the article published today in Lancet that indicates reduced iron in the hearts of thalassemia patients and improved heart function in patients treated with deferiprone. Clearly, if deferiprone can be effective at removing heart iron, it will make an enormous difference in the health and life expectancy of our patients," stated Frank Somma, National President of the Cooley's Anemia Foundation.

Cooley's anemia, also known as thalassemia, is a genetic blood disorder. Patients with thalassemia require frequent red blood cell transfusions. Excess iron from the transfusions accumulates in the heart, liver and other organs. In the United States, patients have only one treatment option for the removal of excess iron - the subcutaneous infusion of an iron chelator (desferrioxamine) over a period of 8-12 hours every night. Compliance with this treatment regime has proven difficult.

The study, published today in Lancet and entitled "Improved myocardial iron levels and ventricular function with oral deferiprone compared with subcutaneous desferrioxamine in thalassemia," was authored by a team led by a cardiologist, Dudley Pennell of the Royal Brompton Hospital, London England. Professor Pennell utilized a new, validated Magnetic Resonance Imaging (MRI) technique to determine the degree of toxic iron loading in the heart and found that the enhanced removal of iron from the heart was associated with improved heart function in the deferiprone patients.

Prior to Prof. Pennell's work, there was no accurate assessment of iron overload in the heart. Previous measurements of iron overload were based upon hepatic iron concentration or serum ferritin. The new MRI technique shows that liver iron concentrations do not predict heart iron concentrations in chelated patients.

"Predicting the survival of thalassemia patients by using liver iron concentrations is likely to be misleading because most deaths occur from heart disease," stated Pennell.

"A new and critical finding in this study was that we were able to establish, using this new MRI technology, that deferiprone was more effective than subcutaneous desferrioxamine in reducing cardiac iron," stated Dr. Beatrix Wonke, a thalassemia expert and a co-author of the study.

"This news is particularly important because the most frequent cause of death among thalassemia patients is cardiac failure, occurring among 52 percent of the patient population and leading to death at a median age of 26 years," said Ralph Colasanti, immediate past president, Thalassemia Action Group.

"We are anxious for the FDA to review the merits of this drug, particularly its benefit to the heart. An orally administered chelator, such as deferiprone, provides an alternative that may greatly improve compliance and increase life expectancy," stated Colasanti.

A second study, available online in Blood (and to be published in the September print edition of Blood) was commissioned by the International Safety Monitoring Committee that oversaw clinical trials of deferiprone. The study, "Lack of Progressive Hepatic Fibrosis during Long Term Therapy with Deferiprone in Subjects with Transfusion-Dependent Beta Thalassemia," examined a claim that deferiprone may worsen hepatic fibrosis in patients with thalassemia. An analysis of the largest collection of liver biopsies reported to date in patients receiving deferiprone demonstrates no evidence of deferiprone-induced progression of hepatic fibrosis during long-term therapy. The lead author of the study is Dr. Ian Wanless, liver pathologist at Toronto General Hospital.

"Combined, these studies indicate that deferiprone can be a safe and effective approach to offsetting complications of iron overload among our patients," stated Somma. "We are told that Apotex Inc., of Toronto, Canada plans to file a New Drug Application with the FDA in September 2002. The Foundation looks forward to a fair and thorough review of these data when the application is submitted."

For more information, contact Craig Butler, 718-321-2873 ([e-mail]) or visit our press office at http://www.cooleysanemia.org/sections.php?sec=156&node=156