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).

Related News

Anti-inflammatory drug may help treat alcohol use disorder

A drug that is already FDA-approved for treating inflammatory conditions may help reduce both...

Osteoarthritis study uncovers new genetic links, drug targets

The genome-wide association study (GWAS) uncovered over 900 genetic associations, more than 500...

How brain cells are affected by Tourette syndrome

US researchers have conducted a cell-by-cell analysis of brain tissue from individuals with...


  • All content Copyright © 2025 Westwick-Farrow Pty Ltd