Comprehensive Monograph: Uridine Triacetate (DB09144)

Section 1: Executive Summary

[Uridine triacetate is a pyrimidine analog with a unique dual-indication profile, functioning as both an orphan drug for a rare genetic metabolic disorder and a critical emergency antidote in oncology. Identified by DrugBank ID DB09144 and CAS Number 4105-38-8, it is a small molecule chemically engineered as a highly bioavailable oral prodrug of the naturally occurring nucleoside, uridine.][1][ This structural modification significantly enhances its therapeutic utility by enabling the achievement of systemic uridine concentrations that are 4- to 6-fold higher than what is possible with equimolar doses of uridine itself.][1]

[The drug's clinical applications are governed by two distinct pharmacological mechanisms. For the treatment of hereditary orotic aciduria, a rare condition caused by a deficiency in uridine monophosphate synthase, uridine triacetate acts as a replacement therapy. Marketed as Xuriden®, it bypasses the genetic defect by providing an exogenous source of uridine, thereby restoring the pyrimidine nucleotide pool and reducing the toxic accumulation of orotic acid through feedback inhibition.][1][ In its second role, marketed as Vistogard®, it serves as a life-saving antidote for overdose or severe, early-onset toxicity resulting from the chemotherapeutic agents fluorouracil or capecitabine. In this context, it functions as a competitive antagonist, where the supplemental uridine outcompetes the toxic fluorouracil metabolite, fluorouridine triphosphate (FUTP), for incorporation into the RNA of healthy cells, thus preventing cellular damage and death.][1]