Benzinga | Jun 29, 2021 11:02AM EDT

Rafael Holdings Says FDA Grants Rafael Pharmaceuticals Orphan Drug Designation For CPI-613 For Treatment Of Biliary Cancer

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(r) (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."

"Biliary cancer affects a small subset of cancer patients, but the unique complexities of the disease create an incredible need to investigate and identify effective treatments," said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. "Therapies developed for hard-to-treat cancers need to consider the unique and complex intricacies of each disease. Having effective treatment options available to patients significantly improves the outlook for patients diagnosed with a rare cancer."

Devimistat has been granted orphan drug status by the FDA for the treatment of pancreatic cancer, acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), Burkitt's lymphoma, peripheral T-cell lymphomas, and soft tissue sarcoma, and now, biliary cancer. With this, devimistat reaches 7 designations in total, making it one of the few compounds to have achieved this milestone. These designations support a future for therapies that focus on cancer metabolism.