Uridine Triacetate

drug
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Also known as PN-401PN401RG-2133RG2133Triacetate d'uridineTriacetato de uridinaVistogardVistonuridineXuriden2',3',5'-TRI-O-ACETYLURIDINEuridine-triacetateUridine triacetateÊ2';3';5'-Tri-O-acetyluridineUridine triacetateÂ

Summary

Uridine Triacetate (CHEMBL2107381) is an approved small-molecule prodrug (ATC A16AX13); indicated across 5 conditions including exocrine pancreatic carcinoma and gastric neoplasm.

At a glance

  • Status: Approved (max clinical phase 4)
  • Modality: Small molecule
  • ATC class: A16AX13
  • Indications: 5 conditions
  • Clinical trials: 3
  • Chemistry: 370.31 Da · C15H18N2O9

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL2107381
NameUridine Triacetate
TypeSmall molecule
Max phase4
FDA approvedyes
PubChem CID20058
ChEBICHEBI:90914
ATCA16AX13
Molecular formulaC15H18N2O9
Molecular weight370.31
InChIKeyAUFUWRKPQLGTGF-FMKGYKFTSA-N

SMILES: CC(=O)OC[C@@H]1[C@H]([C@H]([C@@H](O1)N2C=CC(=O)NC2=O)OC(=O)C)OC(=O)C

IUPAC name: [(2R,3R,4R,5R)-3,4-diacetyloxy-5-(2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methyl acetate

ChEBI definition: An acetate ester that is uracil in which the three hydroxy hydrogens are replaced by acetate group. A prodrug for uridine, it is used for the treatment of hereditary orotic aciduria and for management of fluorouracil toxicity.

Pharmacological roles (ChEBI): prodrug, neuroprotective agent, orphan drug.

Also known as: PN-401, PN401, RG-2133, RG2133, Triacetate d’uridine, Triacetato de uridina, Uridine triacetate, Vistogard, Vistonuridine, Xuriden, URIDINE TRIACETATE, 2’,3’,5’-TRI-O-ACETYLURIDINE

Patent coverage: 520 distinct patent families (1,465 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

5 indications (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

IndicationTrial phaseMONDOEFO
exocrine pancreatic carcinoma3MONDO:0005192EFO:0002618
gastric neoplasm2MONDO:0021085MONDO:0001056
neoplasm1MONDO:0005070EFO:0000616
inborn mitochondrial metabolism disorder1MONDO:0004069MONDO:0044970

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 3.

Phase distribution

PhaseTrials
PHASE31
PHASE11
Not specified1

Top trials by phase / activity

NCTPhaseStatusTitle
NCT02110147PHASE3COMPLETEDOpen-Label Study of Uridine Triacetate in Pediatric Patients With Hereditary Orotic Aciduria
NCT00060515PHASE1TERMINATEDRG2133 (2’,3’,5’-Tri-O-Acetyluridine) in Mitochondrial Disease
NCT01432301Not specifiedAPPROVED_FOR_MARKETINGUridine Triacetate as Antidote for Patients at Excess Risk of 5-FU Toxicity Due to Overdosage or Impaired Elimination

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).