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Orphan drug designation granted by US FDA for Ceflatonin

Melbourne | Wednesday, March 22, 2006, 08:00 Hrs [IST]

The United States Food and Drug Administration (FDA) has granted Orphan Drug status for ChemGenex Pharmaceuticals Limited's advanced drug, Ceflatonin, for the treatment of chronic myeloid leukaemia (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 leukaemia (CML), myelodysplastic syndromes (MDS) and acute myeloid leukaemia (AML).

According to the company release, 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 leukaemia (AML).

"The decision by the US 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.
Ceflatonin (HHT) is a potent inducer of apoptosis (programmed cell death) in myeloid cells and inhibits angiogenesis (blood vessel formation).

Recent studies have demonstrated that Ceflatonin has potential in CML patients who are developing resistance to tyrosine kinase inhibitor (TKI) therapy, including Gleevec, and in-combination with TKI's in early stage CML to increase the cytogenetic and molecular response rate.