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Fluorouracil

drug
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Also known as 5 FU5.f.u.AccusiteActikerallAdrucilCaracEfudexEfudixFluoro-uracil rocheFluoroplexFluorouracil component of folfoxFluorouraciloFluorouracilumFluorouricilFluracilNSC-19893PhthoruracilQueroplexRO-29757

Summary

Fluorouracil (CHEMBL185) is an approved small-molecule antineoplastic agent (ATC L01BC02); indicated across 114 conditions including actinic keratosis and neoplasm; with CIViC clinical evidence for 28 variant-indication associations (e.g. DPYD DPYD*13 HOMOZYGOSITY in cancer).

At a glance

  • Status: Approved (max clinical phase 4)
  • Modality: Small molecule
  • ATC class: L01BC02 (+1 more)
  • Indications: 114 conditions
  • Clinical trials: 1,549
  • Precision-oncology evidence (CIViC): 28 variant–indication associations
  • Chemistry: 130.08 Da · C4H3FN2O2

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL185
NameFluorouracil
TypeSmall molecule
Max phase4
FDA approvedyes
PubChem CID3385
ChEBICHEBI:46345
ATCL01BC02, L01BC52
Molecular formulaC4H3FN2O2
Molecular weight130.08
InChIKeyGHASVSINZRGABV-UHFFFAOYSA-N

SMILES: C1=C(C(=O)NC(=O)N1)F

IUPAC name: 5-fluoro-1H-pyrimidine-2,4-dione

ChEBI definition: A nucleobase analogue that is uracil in which the hydrogen at position 5 is replaced by fluorine. It is an antineoplastic agent which acts as an antimetabolite - following conversion to the active deoxynucleotide, it inhibits DNA synthesis (by blocking the conversion of deoxyuridylic acid to thymidylic acid by the cellular enzyme thymidylate synthetase) and so slows tumour growth.

Pharmacological roles (ChEBI): antineoplastic agent, immunosuppressive agent, radiosensitizing agent.

Other ChEBI roles (chemical / environmental): antimetabolite, xenobiotic, environmental contaminant.

Also known as: 5 FU, 5.f.u., Accusite, Actikerall, Adrucil, Carac, Efudex, Efudix, Fluoro-uracil roche, Fluoroplex, Fluorouracil, Fluorouracil component of folfox

Patent coverage: 83,522 distinct patent families (299,469 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 299,360 (100%) of the total. Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Broader ChEMBL bioactivity targets: 14 (assay-derived). Sample: Microtubule-associated protein tau, Survival motor neuron protein, Prelamin-A/C, Thrombopoietin, Endonuclease 4, Peripheral myelin protein 22, Thyrotropin receptor, Aromatase, Uracil phosphoribosyltransferase, Serine/threonine-protein kinase mTOR.

Bioactivity

ChEMBL activities: 20 potent at pChembl ≥ 5 of 27 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
LMNA7.95Potency11.2nMCHEMBL_ACT_3665214
THPO7.9Potency12.6nMCHEMBL_ACT_4809534
THPO7.9Potency12.6nMCHEMBL_ACT_5074241
P076077.7IC5020nMCHEMBL_ACT_6151517
P076077.22IC5060nMCHEMBL_ACT_6151532
LMNA6.9Potency125.9nMCHEMBL_ACT_3627804
LMNA6.8Potency158.5nMCHEMBL_ACT_4402439
P076076.66IC50220nMCHEMBL_ACT_6151512
P076076.22IC50600nMCHEMBL_ACT_6151527
CYP19A16.2IC50630nMCHEMBL_ACT_24663729
SMN16.2Potency631nMCHEMBL_ACT_3864856
TSHR6.1Potency794.3nMCHEMBL_ACT_3939460
TSHR6.1Potency794.3nMCHEMBL_ACT_4711433
LMNA5.9Potency1259nMCHEMBL_ACT_3647585
HTT5.7Potency1995nMCHEMBL_ACT_3748045
PMP225.32Potency4776nMCHEMBL_ACT_4358816
SMN15.3Potency5012nMCHEMBL_ACT_3907880
MAPT5.25Potency5623nMCHEMBL_ACT_4030398
P076075.16IC507000nMCHEMBL_ACT_6151507
P0A6C15.05Potency8912nMCHEMBL_ACT_4084702

Target pathways

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

Indications & clinical

Indications

114 indications (7 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
actinic keratosis4MONDO:0005173EFO:0002496
neoplasm4MONDO:0005070EFO:0000616
adenocarcinoma4MONDO:0004970EFO:0000228
basal cell carcinoma4MONDO:0020804EFO:0004193
keratosis4MONDO:0006566EFO:1000720
colorectal adenocarcinoma3MONDO:0005008EFO:0000365
exocrine pancreatic carcinoma3MONDO:0005192EFO:0002618
hepatocellular carcinoma3MONDO:0007256EFO:0000182
nasopharyngeal neoplasm3MONDO:0005375EFO:0004252
breast neoplasm3MONDO:0021100EFO:0003869
rectal cancer3MONDO:0006519EFO:1000657
carcinoma of esophagus3MONDO:0019086EFO:0002916
pancreatic neoplasm3MONDO:0021040EFO:0003860
gastric carcinoma3MONDO:0004950EFO:0000178
head and neck squamous cell carcinoma3MONDO:0010150EFO:0000181
carcinoma3MONDO:0004993EFO:0000313
gastric adenocarcinoma3MONDO:0005036EFO:0000503
squamous cell carcinoma3MONDO:0005096EFO:0000707
laryngeal neoplasm3MONDO:0021071EFO:0003817
anus neoplasm3MONDO:0003046EFO:0003835
gastric neoplasm3MONDO:0021085EFO:0003897
colorectal neoplasm3MONDO:0005335EFO:0004142
upper aerodigestive tract neoplasm3MONDO:0005398EFO:0004284
colonic neoplasm3MONDO:0005401EFO:0004288
cholangiocarcinoma3MONDO:0019087EFO:0005221
esophageal squamous cell carcinoma3MONDO:0005580EFO:0005922
head and neck cancer3MONDO:0005627EFO:0006859
chalazion3MONDO:0005844EFO:0007363
hepatoblastoma3MONDO:0018666EFO:1000292
dysplasia of cervix3MONDO:0006736EFO:1000910
inflammatory breast carcinoma3MONDO:0006804EFO:1000984
proliferative vitreoretinopathy3MONDO:0700115EFO:1001129
colorectal carcinoma3MONDO:0024331EFO:1001951
urothelial carcinoma3MONDO:0040679EFO:0008528
intrahepatic cholangiocarcinoma3MONDO:0003210EFO:1001961
urinary bladder neoplasm3MONDO:0004987EFO:0000294
hypopharyngeal carcinoma3MONDO:0005216EFO:0002938
neuroendocrine neoplasm3MONDO:0019496EFO:1001901
colon adenocarcinoma3MONDO:0002271EFO:1001949
neuroendocrine carcinoma3MONDO:0002120MONDO:0002120
kidney cancer3MONDO:0002367MONDO:0002367
rectal neoplasm3MONDO:0002165MONDO:0044937
paranasal sinus neoplasm3MONDO:0005289EFO:0003866
oral cavity cancer3MONDO:0005515EFO:0005570
pharynx cancer3MONDO:0005517EFO:0005577
head and neck neoplasm3MONDO:0005586EFO:0005950
biliary tract neoplasm3MONDO:0005304EFO:0003891
malignant pancreatic neoplasm3MONDO:0009831EFO:1000359
glaucoma3MONDO:0005041MONDO:0005041
undifferentiated carcinoma3MONDO:0005617EFO:0006772
colon carcinoma3MONDO:0002032EFO:1001950
pancreatic ductal adenocarcinoma3MONDO:0005184MONDO:0005184
nasopharyngeal carcinoma3MONDO:0015459MONDO:0015459
intestinal cancer3MONDO:0005814MONDO:0021118
non-small cell lung carcinoma2MONDO:0005233EFO:0003060
peripheral neuropathy2MONDO:0005244EFO:0003100
tumor of uterus2MONDO:0021353EFO:0003859
peritoneal neoplasm2MONDO:0006901EFO:1001100
cutaneous neuroendocrine carcinoma2MONDO:0019210EFO:1001471
digestive system neoplasm2MONDO:0021223EFO:0008549
pancreatic endocrine carcinoma2MONDO:0005893EFO:0007416
thrombotic disease2MONDO:0000831MONDO:0000831
ovarian cancer2MONDO:0008170MONDO:0008170
oral cavity squamous cell carcinoma2MONDO:0004958EFO:0000199
ovarian carcinoma2MONDO:0005140EFO:0001075
skin carcinoma2MONDO:0002656EFO:0009259
bile duct neoplasm2MONDO:0021662MONDO:0003059
vitiligo2MONDO:0008661EFO:0004208
gallbladder carcinoma2MONDO:0003220EFO:1001956
hilar cholangiocarcinoma2MONDO:0003345EFO:1001959
bile duct carcinoma2MONDO:0005496EFO:0005540
gallbladder neoplasm2MONDO:0021253MONDO:0005411
carcinoid tumor2MONDO:0005369MONDO:0024503
ependymoma1MONDO:0016698EFO:1000028
myelodysplastic syndrome1MONDO:0018881EFO:0000198
lymphoma1MONDO:0005062EFO:0000574
renal cell carcinoma1MONDO:0005086EFO:0000681
melanoma1MONDO:0005105EFO:0000756
plasma cell myeloma1MONDO:0009693EFO:0001378
central nervous system cancer1MONDO:0002714EFO:0000326
myeloproliferative neoplasm1MONDO:0020076EFO:0002428
non-Hodgkin lymphoma1MONDO:0018908EFO:0005952
metastatic melanoma1MONDO:0005191EFO:0002617
fallopian tube carcinoma1MONDO:0006206EFO:1000251
gastrointestinal stromal tumor1MONDO:0011719MONDO:0011719
leukemia1MONDO:0005059EFO:0000565
triple-negative breast carcinoma1MONDO:0005494EFO:0005537
plasma cell neoplasm1MONDO:0004959EFO:0000200
oral mucosa leukoplakia1MONDO:0004844MONDO:0004844

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

Clinical trials

Total trials: 1,549.

Phase distribution

PhaseTrials
PHASE2689
PHASE3392
PHASE1198
PHASE1/PHASE2128
Not specified57
PHASE2/PHASE342
PHASE430
EARLY_PHASE113

Top trials by phase / activity

NCTPhaseStatusTitle
NCT06499415PHASE4RECRUITINGCombination Therapy of 5-Fluorouracil and CALcipotriol Versus 5-Fluorouracil in the Treatment of Actinic Keratosis
NCT07014540PHASE4NOT_YET_RECRUITINGA Irinotecan Liposome Trial of in Advanced Neuroendocrine Carcinoma
NCT07094893PHASE4NOT_YET_RECRUITINGAnti-EGFR Agents in Patients With Right-sided Advanced Colorectal Cancer With Wild-type RAS and AREG/EREG High Status
NCT07158164PHASE4RECRUITINGDPYD Pharmacogenomics and Fluoropyrimidine (FP) Dose-Adjustment
NCT07352566PHASE4NOT_YET_RECRUITINGUtilization of a Microdevice for Psoriasis and Atopic Dermatitis
NCT07397520PHASE4RECRUITINGThe Application of Intralesional 5-fluorouracil for the Management of Hypertrophic Facial Scars in the Periocular Region Resulting From Trauma and Surgical Procedures in Individuals Aged Over 18 Years
NCT00138060PHASE4COMPLETEDToxicity/Benefit Ratio Optimization of Chemotherapy in Colorectal Cancer (CRC) Patients by Determination of Individual Genotypic Determinants
NCT00346489PHASE4COMPLETEDOutcomes of Intraoperative 5-Fluorouracil Versus Mitomycin C
NCT00559676PHASE4COMPLETEDStudy of Biomarkers in Patients Undergoing Chemotherapy for Metastatic Colorectal Cancer
NCT00607685PHASE4COMPLETED5FU vs 5FU With Viscoelastic Formulation for the Prevention of Scarring Post-trabeculectomy
NCT00696488PHASE4COMPLETEDMeasuring Adherence To Topical 5-Fluorouracil in a Clinic Population
NCT00847912PHASE4COMPLETEDCSP #562 - The VA Keratinocyte Carcinoma Chemoprevention Trial
NCT01023633PHASE4UNKNOWNOPTIMOX1 in Chinese mCRC Patients
NCT01059110PHASE4TERMINATEDComparison of Five Treatments in Patients With Plantar Warts
NCT01849380PHASE4UNKNOWNNeoadjuvant ECS Versus ECF in Local Advanced Breast Cancer
NCT01972503PHASE4UNKNOWNIntraoperative Intraportal Chemotherapy Combined With Adjuvant Chemotherapy for Stage II and III Colorectal Cancer
NCT02015650PHASE4TERMINATEDCetuximab Compared to Mitomycin-C and 5-Fluorouracil for Locally Advanced Squamous Cell Carcinomas of the Head and Neck
NCT02155439PHASE4COMPLETEDTreatment of Keloidscars With Intralesional Triamcinolone and 5-fluorouracil Injections-prospective, Randomized, Controlled Trial - Pilot Study
NCT02281682PHASE4UNKNOWNIM Versus 5-FU Versus IMI Versus MAL-PDT in Treatment of Actinic Keratosis
NCT02366819PHASE4SUSPENDEDGenetic Analysis-Guided Irinotecan Hydrochloride Dosing of mFOLFIRINOX in Treating Patients With Locally Advanced Gastroesophageal or Stomach Cancer
NCT02484677PHASE4COMPLETEDPharmacokinetics and Pharmacogenetics-based Adaptive Dosing of 5-fu (5-Fluorouracile) in Head & Neck Cancer Patient Undergoing Docetaxel, Cisplatin, 5-Fluorouracile (=TPF) Therapy
NCT02530801PHASE4UNKNOWNStrategies for Management of Recurrent Pterygium
NCT02557503PHASE4UNKNOWNHepatic Arterial Infusion of Oxaliplatin and Fluorouracil Treatment of Advanced Primary Liver Cancer After TACE
NCT02582970PHASE4COMPLETEDA Study of Bevacizumab (Avastin) in Combination With Chemotherapy in Participants With Metastatic Cancer of the Colon or Rectum
NCT03037736PHASE4WITHDRAWNOutpatient Performed Pterygium Surgery Study
NCT03904381PHASE4UNKNOWNEfficacy and Safety of XEN® Gel Stent and Post-operative Management in Patients With Open Angle Glaucoma
NCT04269369PHASE4UNKNOWNImplementation of Pre-emptive Geno- and Phenotyping in 5-Fluorouracil- or Capecitabine-treated Patients
NCT04875026PHASE4COMPLETEDFrequency and Intensity of Local Reactions in Patients Treated With 4% 5-FU vs 4% 5-FU Associated With an Emollient Cream: a Randomised, Controlled Clinical Trial
NCT05008887PHASE4UNKNOWNFractional CO2 Laser-assisted Cutaneous Delivery of Methotrexate Versus 5-fluorouracil in Stable Non-segmental Vitiligo
NCT05444673PHASE4UNKNOWNA Prospective, Single-arm Clinical Study of Pabrolizumab in Combination With Cisplatin + 5-FU for the Neoadjuvant Treatment of Unresectable, Advanced Squamous Carcinoma of the Temporal Bone Single-arm Clinical Study
NCT00217737PHASE3ACTIVE_NOT_RECRUITINGOxaliplatin, Leucovorin, and Fluorouracil With or Without Bevacizumab in Treating Patients Who Have Undergone Surgery for Stage II Colon Cancer
NCT00433420PHASE3ACTIVE_NOT_RECRUITINGCombination Chemotherapy With or Without Fluorouracil and/or Pegfilgrastim in Treating Women With Node-Positive Breast Cancer
NCT00588770PHASE3ACTIVE_NOT_RECRUITINGChemotherapy With or Without Bevacizumab in Treating Patients With Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma
NCT00980460PHASE3ACTIVE_NOT_RECRUITINGRisk-Based Therapy in Treating Younger Patients With Newly Diagnosed Liver Cancer
NCT01954992PHASE3RECRUITINGGlufosfamide Versus 5-FU in Second Line Metastatic Pancreatic Cancer
NCT02025023PHASE3RECRUITINGLocal 5-Fluorouracil Injection for the Treatment of Chalazia: A Prospective, Comparative Study
NCT02135042PHASE2/PHASE3ACTIVE_NOT_RECRUITINGIndividualized Treatment Treating Patients With Stage II-IVB Nasopharyngeal Cancer Based on EBV DNA
NCT02509286PHASE3ACTIVE_NOT_RECRUITINGPerioperative Chemotherapy Compared To Neoadjuvant Chemoradiation in Patients With Adenocarcinoma of the Esophagus
NCT02535221PHASE3RECRUITINGNeoadjuvant Endocrine Therapy Versus Chemotherapy in Premenopausal Patients With ER+ & HER2- Breast Cancer
NCT02912559PHASE3ACTIVE_NOT_RECRUITINGCombination Chemotherapy With or Without Atezolizumab in Treating Patients With Stage III Colon Cancer and Deficient DNA Mismatch Repair

Clinical evidence (CIViC)

Variant × indication × effect (28 predictive associations from 28 curated evidence items):

VariantIndicationEffectTherapyLevelCIViC
DPYD DPYD*13 HOMOZYGOSITYCancerAdverse ResponseCapecitabine + Fluorouracil + TegafurCIViC AEID1801
DPYD DPYD*2A HOMOZYGOSITYCancerAdverse ResponseTegafur + Capecitabine + FluorouracilCIViC AEID1800
DPYD RS67376798 HOMOZYGOSITYCancerAdverse ResponseTegafur + Fluorouracil + CapecitabineCIViC AEID1803
AURKA EXPRESSIONEsophagus Squamous Cell CarcinomaSensitivity/ResponseRadiation Therapy + Fluorouracil + CisplatinCIViC BEID1653
CDKN1B EXPRESSIONHead And Neck Squamous Cell CarcinomaSensitivity/ResponseCisplatin + FluorouracilCIViC BEID889
EGFR AmplificationHead And Neck Squamous Cell CarcinomaSensitivity/ResponseFluorouracil + Platinum Compound + CetuximabCIViC BEID691
HSPA5 EXPRESSIONColorectal CancerSensitivity/ResponseFluorouracilCIViC BEID914
HSPH1 T17 DELETIONColorectal CancerSensitivity/ResponseFluorouracil + OxaliplatinCIViC BEID1164
MTHFR A222VRectum CancerSensitivity/ResponseFluorouracilCIViC BEID1757
MTHFR A222VStomach CancerSensitivity/ResponseFluorouracilCIViC BEID669
VEGFA Decreased Peri-therapeutic ExpressionColorectal CancerSensitivity/ResponseLevoleucovorin + Irinotecan + Bevacizumab + FluorouracilCIViC BEID818
ALCAM EXPRESSIONColorectal CancerResistanceFluorouracilCIViC BEID853
CDKN1A EXPRESSIONColorectal CancerResistanceFluorouracilCIViC BEID852
DKK1 NUCLEAR EXPRESSIONColorectal CancerResistanceLevoleucovorin + Oxaliplatin + Fluorouracil + IrinotecanCIViC BEID824
GNAS c.393T>CEsophageal CancerResistanceCisplatin + FluorouracilCIViC BEID2895
SMAD4 DeletionColorectal CancerResistanceFluorouracilCIViC BEID1644
TYMS 3TRP/3TRP genotypeColorectal CancerResistanceFluorouracil + IrinotecanCIViC BEID678
DPYD EXON 11-19 DELETIONHead And Neck Squamous Cell CarcinomaSensitivity/ResponseLeucovorin + FluorouracilCIViC CEID7787
ERBB2 L755SColorectal AdenocarcinomaSensitivity/ResponseTrastuzumab + Leucovorin + FluorouracilCIViC CEID4955
PIK3CA E81KColorectal CancerResistanceCetuximab + Fluorouracil + OxaliplatinCIViC CEID4464
PIK3CA R93WColorectal CancerResistanceOxaliplatin + Cetuximab + FluorouracilCIViC CEID4467
ATRX DeletionCancerSensitivity/ResponseCisplatin + FluorouracilCIViC DEID9239
HSPH1 NUCLEAR EXPRESSIONGastric AdenocarcinomaSensitivity/ResponseFluorouracil + CisplatinCIViC DEID1163
NPTXR OverexpressionStomach CarcinomaSensitivity/ResponseCisplatin + FluorouracilCIViC DEID12774
ERCC6 OverexpressionColorectal CancerResistanceFluorouracilCIViC DEID12121
LGR5 LGR5FLColorectal CancerResistanceFluorouracilCIViC DEID9623
RB1 OverexpressionPancreatic AdenocarcinomaResistanceDoxorubicin + Fluorouracil + Gemcitabine + MitomycinCIViC DEID1324
YAP1 OverexpressionEsophageal CancerResistanceDocetaxel + FluorouracilCIViC DEID1982

Pharmacology

Pharmacogenomics

PharmGKB dosing guidelines (5) — CPIC / DPWG genotype-guided dosing for this drug (drug × pharmacogene):

GuidelineSourceGene(s)DosingRecommendation
Annotation of DPWG Guideline for fluorouracil and DPYDDPWGDPYDyesyes
Annotation of CPIC Guideline for fluorouracil and DPYDCPICDPYDyesyes
Annotation of RNPGx Guideline for capecitabine, fluorouracil and DPYDRNPGxDPYDyesyes
Annotation of SEFF/SEOM Guideline for capecitabine, fluorouracil, tegaSEFF/SEOMDPYDyesyes
Annotation of AIOM Guideline for capecitabine, fluorouracil, tegafur aAIOMDPYDyesyes

PharmGKB also curates 197 clinical and 1,046 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

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

About this page

Generated deterministically by Sugi Atlas from primary biomedical databases via BioBTree. Every record on this page traces to a primary source.

Generated
Atlas version
v1.1.2
BioBTree
v2.0.2

Sources 17

This page pulls from 17 datasets indexed by BioBTree:

chebichembl_activitychembl_moleculechembl_targetcivic_evidenceclinical_trialsefogtopdb_interactiongtopdb_ligandhgncmeshmondopatent_compoundpharmgkbpharmgkb_guidelinepubchemuniprot