FDA Grants Rafael Pharmaceuticals Orphan Drug Designation for CPI-613® (Devimistat) for Treatment of Biliary Cancer Company reaches important milestone to advance potential novel therapy for this subset of gastrointestinal cancers

Rafael Pharmaceuticals, Inc. (“Rafael” or the “Company”), a leader in the growing field of cancer metabolism-based therapeutics, announced today that the U.S. Food and Drug Administration (FDA) granted orphan drug designation for CPI-613^® (devimistat) for the treatment of biliary cancer.

Biliary cancer, which includes gallbladder cancer and bile duct cancer (which is also known as cholangiocarcinoma), is classified as a rare disease, affecting just 12,000 people in the United States each year, respectively. Biliary cancer appears when cancer cells form in the bile ducts or gallbladder, an essential aspect of the gastrointestinal system.

“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, M.S., Associate Professor of Medical Oncology at The University of Michigan Medicine and the principal investigator on the Phase 1b/2 clinical trial of devimistat in combination with gemcitabine and cisplatin for patients with biliary cancer, announced last year. “The orphan drug designation for devimistat showcases the importance of discovering these new treatment options.”

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