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Biliary cancer drug candidate wins orphan drug designation from FDA

By Brian Buntz | June 29, 2021

Rafael Pharmaceuticals logoCranbury, 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
Tagged With: acute myeloid leukemia, biliary cancer, Burkitt’s lymphoma, CPI-613, devimistat, myelodysplastic syndrome, orphan drug status, pancreatic cancer, peripheral T-cell lymphomas, Rafael Pharmaceuticals, soft tissue sarcoma.
 

About The Author

Brian Buntz

As the pharma and biotech editor at WTWH Media, Brian has almost two decades of experience in B2B media, with a focus on healthcare and technology. While he has long maintained a keen interest in AI, more recently Brian has made making data analysis a central focus, and is exploring tools ranging from NLP and clustering to predictive analytics.

Throughout his 18-year tenure, Brian has covered an array of life science topics, including clinical trials, medical devices, and drug discovery and development. Prior to WTWH, he held the title of content director at Informa, where he focused on topics such as connected devices, cybersecurity, AI and Industry 4.0. A dedicated decade at UBM saw Brian providing in-depth coverage of the medical device sector. Engage with Brian on LinkedIn or drop him an email at bbuntz@wtwhmedia.com.

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