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News: Ceflatonin receives U.S. FDA orphan drug designation for treatment of chronic myeloid leukemia

04-20-2006

ChemGenex Pharmaceuticals Limited (CXSP) announced that the U.S. Food & Drug Administration (FDA) has granted orphan drug status for the company's most advanced drug, Ceflatonin, for the treatment of chronic myeloid leukemia (CML).

Ceflatonin is the first of a new class of novel drugs that induces apoptosis (programmed cell death) and inhibits angiogenesis (new blood vessel formation). Ongoing or soon to be initiated clinical trials are and will be evaluating Ceflatonin in a broad range of conditions, including: chronic myeloid leukemia (CML), myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML).

Orphan drug status is granted by the FDA to promote the development of drugs for diseases affecting less than 200,000 people in the United States. Orphan drug status entitles ChemGenex to 7 years of market exclusivity for the use of Ceflatonin in the treatment of CML; protocol assistance by the FDA to optimize drug development in the preparation of a dossier that will meet regulatory requirements; and reduced fees associated with applying for market approval.

A similar designation for Ceflatonin in the European market, with similar advantages, was granted by the European Union on September 9, 2004, based on a positive recommendation by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMEA). The European Union granted Ceflatonin orphan status on October 20, 2004 for the treatment of acute myeloid leukemia (AML).

A multinational phase II/III study in CML patients harboring the T315I point mutation, known to confer complete resistance to tyrosine kinase (TKI) inhibitors like Gleevec, is scheduled to begin in Q2, 2006. A second multinational phase II/III study in CML patients resistant to TKI inhibitors is scheduled to begin soon thereafter. Each study will include centers in the United States, Germany, France and Italy.

Ceflatonin is not approved by the FDA as a treatment in any indication and is currently being evaluated in clinical trials for efficacy and safety for future regulatory applications.

ChemGenex Pharmaceuticals is a pharmaceutical development company dedicated to improving the lives of patients by developing therapeutics in the areas of oncology, diabetes, obesity, and depression.

This article was prepared by Clinical Oncology Week editors from staff and other reports. Copyright 2006, Clinical Oncology Week via NewsRx.com.