Cornerstone Pharmaceuticals, Inc. a company focused on rare cancer therapeutics and formerly known as Rafael Pharmaceuticals, announced that the European Medicines Agency (EMA) has granted orphan drug designation to CPI-613 (devimistat) for the treatment of advanced unresectable biliary tract cancer.
Also known as cholangiocarcinoma, biliary tract cancer is an extremely rare and hard to treat cancer, affecting just over 2,000 people in the UK annually with incidents steadily increasing every year.
“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,” said Sanjeev Luther, President and CEO of Cornerstone Pharmaceuticals. “Our goal is that this milestone will provide hope for patients and families in a setting where the current prognosis is devastating.”
This orphan drug designation is the fourth granted for devimistat by the EMA, following existing 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 United States, including in biliary tract cancer.
“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,” said Dr. Vaibhav Sahai, MBBS, M.S., principal investigator with Cornerstone’s Phase 2 trial for patients with biliary tract cancer in combination with gemcitabine and cisplatin. “Devimistat is a potentially life-saving therapy, bringing hope to patients and loved ones affected by biliary tract cancer.”