Orphan Drug Designation Granted by the U.S. FDA for Ceflatonin®
MELBOURNE, Australia, and MENLO PARK, California U.S.A. (March 20, 2006).
ChemGenex Pharmaceuticals Limited (ASX:CXS and NASDAQ:CXSP) announced today 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.”
Gleevec® is a registered trademark of the Novartis Pharmaceuticals Corporation.
About Ceflatonin®
Ceflatonin® (homoharringtonine or HHT) is a potent inducer of apoptosis (programmed cell death) in myeloid cells and inhibits angiogenesis (blood vessel formation). In phase 2 studies, Ceflatonin® 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, myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML).
Ceflatonin has a different mechanism of action than tyrosine kinase inhibitors (TKIs), which are widely used in the treatment of CML. A registration-directed, international phase 2/3 clinical study is currently in progress, investigating the effectiveness of Ceflatonin® in the treatment of CML patients who have developed resistance to TKI therapy due to the T315I Bcr-Abl point mutation. Ceflatonin® was recently granted Fast Track status by the U.S. Food and Drug Administration (FDA) for this clinical indication. Additional studies will assess the activity of Ceflatonin® in CML patients who are refractory to, or intolerant of treatment with TKIs and investigate if combination therapy with Ceflatonin®, TKIs and other agents increases the cytogenetic and molecular response rates in CML patients.
Ceflatonin® is not approved by the FDA as a treatment in any indication and is being evaluated in clinical trials for efficacy and safety for future regulatory applications.
About Chronic Myeloid Leukemia
Chronic Myeloid Leukemia (CML) is a cancer of the blood cells caused by an acquired genetic defect called the bcr-abl mutation. This defect occurs when genetic material from two chromosomes (9 and 22) swaps places, creating the so-called Philadelphia chromosome. The bcr-abl mutation interferes with normal cell replication processes, leading to an abnormal proliferation of white blood cells.
CML usually occurs in adults and typically progresses through three phases. Patients generally are diagnosed in ‘chronic phase’, progress through an ‘accelerated phase’ and then may die if the disease progresses to ‘blast phase’. CML incidence is relatively consistent occurring at about 1 to 2 per 100,000 people and the global CML market for therapeutics is estimated at in excess of US$2 billion.
Patients with chronic phase CML have been effectively treated in recent years by the drug Gleevec. However, over time many patients become resistant to the therapeutic effects of the drug and the disease progresses.
About ChemGenex Pharmaceuticals Limited
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. ChemGenex harnesses the power of genomics for target discovery and validation, and in clinical trials to develop more individualized therapeutic outcomes. ChemGenex's lead compound, Ceflatonin®, is currently in phase 2/3 clinical trials for chronic myeloid leukemia and Quinamed® is in phase 2 clinical development for prostate, breast and ovarian cancers. The company has a significant portfolio of anti-cancer, diabetes, obesity and depression programs, several of which have been partnered with international pharmaceutical companies. ChemGenex currently trades on the Australian Stock Exchange under the symbol "CXS" and on NASDAQ under the symbol "CXSP".
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