ChemGenex Pharmaceuticals Limited announced that its investigational drug Ceflatonin (homoharringtonine, HHT) has been awarded fast track status by the US Food and Drug Administration (FDA). The fast track designation covers Ceflatonin for the treatment of patients with chronic, accelerated and blast-phase chronic myeloid leukaemia (CML) who have failed imatinib mesylate (Gleevec) and have the T315I bcr-abl point mutation.
Under the FDA Modernization Act of 1997, the fast track programme facilitates interactions with the FDA before and during the submission of a New Drug Application (NDA) for therapeutics being investigated as a treatment of serious or life-threatening diseases where there is an unmet medical need. The fast track designation will enable ChemGenex to file a New Drug Application (NDA) on a rolling basis as data becomes available. This permits the FDA to review the filing as it is received, rather than waiting for the entire document prior to commencing the review process.
"The FDA decision to award fast track status to Ceflatonin was based on recent clinical data and the high unmet need for new treatments for patients failing Gleevec and Sprycel" said Greg Collier, Ph.D., chief executive officer and managing director of ChemGenex. "As we enter the final stages of clinical development, with significant interest in our trials being conducted in both the US and Europe, we look forward to collaborating closely with the FDA to expedite the development and approval of Ceflatonin."
Ceflatonin (HHT) is a potent inducer of apoptosis (programmed cell death) in myeloid cells and inhibits angiogenesis (blood vessel formation). In phase 2 studies, Ceflatonin has demonstrated clinical activity in patients with CML, both as a single agent and in combination with other chemotherapeutic drugs. ChemGenex is developing Ceflatonin for the treatment of CML, and pilot studies are underway in myelodysplastic syndrome (MDS) and in acute myeloid leukaemia (AML).
Ceflatonin has a different mechanism of action than tyrosine kinase inhibitors (TKI's), and ongoing and proposed clinical studies will seek to determine:
Efficacy in treatment of CML patients who have developed resistance to tyrosine kinase inhibitor (TKI) therapy due to development of the T315I bcr-abl kinase domain point mutation. The T315I bcr-abl mutation, which develops in some CML patients treated with TKI's, is associated with resistance to Gleevec and Sprycel.
Efficacy in CML patients who have failed therapy with two tyrosine kinase inhibitors, e.g., Gleevec and Sprycel; and Efficacy as combination therapy with Gleevec, for the treatment of residual disease and to prolong Gleevec sensitivity in CML patients who have developed resistance to Gleevec.
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.
Ceflatonin is a registered trade-mark of ChemGenex Pharmaceuticals Limited. Gleevec is a registered trade-mark of Novartis AG. SprycelTM is a registered trade-mark of the Bristol-Myers Squibb Company.