Orphan status for ChemGenex

By Kate McDonald
Tuesday, 20 January, 2009

Melbourne’s ChemGenex has been granted orphan drug status for its apoptopic drug omacetaxine mepesuccinate from the US FDA.

The orphan drug status is for use in myelodysplastic syndromes, a range of bone marrow stem cell disorders. May sufferers die from infection while some develop acute myelogenous leukaemia.

The drug is also currently in Phase II trials for chronic myeloid leukaemia. The drug induces apoptosis by inhibiting protein synthesis and is being trialled in people with a mutation in the T3151 gene, who are often resistant to the front-line drug imatinib (Glivec).

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