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# Orphan Drug Status for NuCana’s Acelarin
- URL: https://www.fdaweb.com/orphan-drug-status-for-nucanas-acelarin/
- Published: 2019-06-17T12:00:00.000Z
- Updated: 2026-09-15T01:33:01.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5144351

FDA has granted NuCana an orphan drug designation for its investigational drug, Acelarin (NUC-1031), and its use in treating biliary tract cancer. The company describes the new chemical entity as its “ProTide transformation of gemcitabine... Our Phase 1b study of Acelarin combined with cisplatin showed an approximate doubling of the response rate expected with the standard of care, gemcitabine plus cisplatin, with several patients achieving significant reductions in their tumor volume as well as further tumor shrinkage over time.” A Phase 3 trial is being planned for later this year.