Biliary tract cancer, which is also called as cholangiocarcinoma, is an extremely rare and hard to treat cancer.
It impacts more than 2,000 people in the UK annually with incidents steadily growing each year.
A first-in-class clinical lead compound, CPI-613 (devimistat) targets enzymes that are involved in cancer cell energy metabolism and are located in the mitochondria of cancer cells.
Devimistat is intended to target the mitochondrial tricarboxylic acid (TCA) cycle, a process that is seen as essential to tumor cell multiplication and survival, selectively in cancer cells.
Cornerstone Pharmaceuticals president and CEO Sanjeev Luther said: “We are on a mission to develop cancer treatments for patients who have significant unmet clinical needs. Biliary tract cancer is considered rare and aggressive, with a large gap in effective treatment options.
“Our goal is that this milestone will provide hope for patients and families in a setting where the current prognosis is devastating.”
This ODD is the fourth to be granted for the drug by the EMA, after the current designations for Burkitt’s lymphoma, pancreatic cancer and acute myeloid leukemia (AML).
The Food and Drug Administration (FDA) has granted orphan drug designation for devimistat in seven indications in the US, including in biliary tract cancer.
Cornerstone principal investigator for Phase II trial for patients with biliary tract cancer in combination with gemcitabine and cisplatin Dr Vaibhav Sahai said: “With strong and continued Phase 2 enrollment rates and additional trial sites opening, the research community remains engaged towards identifying the capabilities devimistat has in biliary cancer.
“Devimistat is a potentially life-saving therapy, bringing hope to patients and loved ones affected by biliary tract cancer.”