European Medicines Agency’s Committee for Orphan Medicinal Products issues a positive opinion for orphan drug designation of acelarin for the treatment of biliary tract cancer

A global Phase III study (NuTide:121) is comparing acelarin, in combination with cisplatin, to the current standard of care (gemcitabine plus cisplatin) for the first-line treatment of patients with biliary tract cancer.

SPS commentary:

Acelarin is comprised of gemcitabine monophosphate and a protective phosphoramidate moiety, which enables the drug to enter the cancer cell independent of membrane transporters and protects it from both extracellular and intracellular degradation. Once it has entered the cell, the moiety is cleaved off and an activated, phosphorylated form of gemcitabine released. This is designed to overcome resistance to gemcitabine, as its phosphorylation via an activating enzyme (which can be deficient in cancer cells) is no longer required. The activated form of gemcitabine undergoes conversion ultimately to the key anti-cancer triphosphate metabolite, at much higher intracellular levels than that seen with gemcitabine.


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