Devimistat gets orphan drug desig in EUMarch 7, 2019
The European Medicines Agency (EMA) has granted orphan drug designation to devimistat (CPI-613), for the treatment of metastatic pancreatic cancer.
Devimistat, developed on Rafael Pharma’s Altered Metabolism Directed (AMD) platform, targets the altered regulation of metabolic processes specific to cancer cells. It is highly specific, simultaneously attacking multiple targets, minimally toxic and has broad spectrum activity across a wide variety of cancers, the company said.
Devimistat is currently being evaluated in 7 trials as a single agent, as well as in combination with standard drug therapies for hematological malignancies and solid tumours.
In pancreatic cancer, devimistat in combination with modified folfirinox exhibited an objective response rate of 61%, median overall survival of 19.9 months and median progression-free survival of 9.9 months.
Devimistat also exhibited a good safety profile both as a single agent and in combination with other standard-of-care drugs.
Devimistat has previously been granted orphan drug designation for pancreatic cancer, AML, MDS, peripheral T-cell lymphoma and Burkitt lymphoma by the US FDA.