The Food and Drug Administration (FDA) has granted Orphan Drug designation to CPI-613® (devimistat) for the treatment of biliary cancer.

Devimistat targets the mitochondrial tricarboxylic acid cycle selectively in cancer cells and is expected to increase the sensitivity of cancer cells to chemotherapeutic agents. The investigational drug may potentially be used in combination with lower doses of generally toxic drugs with greater efficacy and lower side effects. 

The Company is investigating the efficacy and safety of devimistat for the treatment of adults with advanced biliary tract cancer in an ongoing multicenter, randomized phase 1/2 trial ( Identifier: NCT04203160). Participants will receive either devimistat in combination with standard of care chemotherapy (gemcitabine plus cisplatin) or standard of care chemotherapy alone. 

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“Biliary cancer is often identified as an advanced stage cancer and considered aggressive with only modest response to existing treatment options for patients,” said Vaibhav Sahai, MBBS, MS, Associate Professor of Medical Oncology at The University of Michigan Medicine and the principal investigator on the phase 1b/2 clinical trial. “The Orphan Drug designation for devimistat showcases the importance of discovering these new treatment options.”

The FDA previously granted Orphan Drug designations to devimistat for the treatment of metastatic pancreatic cancer, acute myeloid leukemia, myelodysplastic syndrome, Burkitt lymphoma, peripheral T-cell lymphomas, and soft tissue sarcoma.


FDA grants Rafael Pharmaceuticals Orphan Drug designation for CPI-613® (devimistat) for treatment of biliary cancer. [press release]. Cranbury, NJ: Rafael Pharmaceuticals, Inc.; June 29, 2021. 

This article originally appeared on MPR