Cranbury, N.J.–based Rafael Pharmaceuticals has announced that its lead pipeline drug CPI-613 (devimistat) has won orphan drug status from FDA for biliary cancer.
The orphan designation extends the orphan drug status for devimistat, including pancreatic cancer, acute myeloid leukemia, myelodysplastic syndrome, Burkitt’s lymphoma, peripheral T-cell lymphomas, and soft tissue sarcoma.
The Europe Medicines Agency has granted orphan drug designation to devimistat for pancreatic cancer and acute myeloid leukemia.
“Biliary cancer is often identified as an advanced stage cancer and considered aggressive with only modest response to existing treatment options for patients,” said Dr. Vaibhav Sahai, an associate professor of medical oncology at The University of Michigan Medicine in a statement. “The orphan drug designation for devimistat showcases the importance of discovering these new treatment options,” added Sahai, who was the principal investigator in the Phase 1b/2 clinical trial of devimistat.
Biliary cancer, a rare and aggressive cancer type, affects approximately 12,000 people in the U.S., according to the National Organization for Rare Disorders.
According to Rafael, devimistat is a first-in-class compound targeting enzymes active in cancer cell energy metabolism.
The drug is currently undergoing Phase 3 trials for acute myeloid leukemia and pancreatic cancer.
The European Society of Medical Oncology World Gastrointestinal Congress (ESMO), scheduled for June 30 through July 3, will feature an oral presentation on devimistat. The presentation will focus on a multicenter, randomized phase 1b/2 study of gemcitabine and cisplatin with or without CPI-613. The study investigated those options as first-line therapies for patients with advanced unresectable biliary tract cancer.
Filed Under: clinical trials, Drug Discovery, Oncology