NuCana Receives Orphan Drug Designation from the U.S. Food and Drug Administration for Acelarin® for the Treatment of Biliary Tract Cancer
NuCana plc (NASDAQ: NCNA) announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for the Company’s investigational drug, Acelarin® (NUC-1031), for the treatment of biliary tract cancer. Acelarin is a new chemical entity and is NuCana’s ProTide transformation of gemcitabine.
“We are pleased to have received orphan drug designation from the FDA for Acelarin in biliary tract cancer,” said Hugh Griffith, NuCana’s Founder and Chief Executive Officer. “There is a high unmet need for patients suffering from this cancer type. Our Phase Ib 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. We believe Acelarin represents a potential significant advance in biliary tract cancer and we remain on track to open our global Phase III study in combination with cisplatin as a front-line treatment for patients with advanced biliary tract cancer in 2019.”
Orphan drug designation is granted by the FDA to drugs that are defined as those intended for the treatment, prevention or diagnosis of rare diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug designation provides certain benefits and incentives that may include tax credits towards the cost of clinical trials and prescription drug user fee waivers.
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