ChemGenex Pharmaceuticals has announced that the United States Food and 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 seven 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).
"The decision by the U.S. FDA to grant Ceflatonin orphan drug status for CML is further incentive to move Ceflatonin as quickly as possible through the clinical development and regulatory approval process worldwide," said Greg Collier, ChemGenex chief executive officer and managing director. "We are very encouraged by the growing body of clinical data on Ceflatonin in resistant CML, MDS and AML, and by the ongoing support of key opinion leaders in the field. Now with the granting of Orphan Drug status by the FDA, we are fully committed to accelerate our development efforts to help address the unmet medical needs of CML patients worldwide."