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Allopurinol

drug
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Also known as Allopurinol component of duzalloAllopurinolumAlopurinolAloralAluline 100Aluline 300CaplenalCosuricHamarin 100Hamarin 300LedopurLopurinNSC-101655NSC-1390SuspendolTakanaruminUrictoXanthomax-100Xanthomax-300

Summary

Allopurinol (CHEMBL1467) is an approved small-molecule EC 1.17.3.2 (xanthine oxidase) inhibitor (ATC M04AA01) targeting XDH; indicated across 46 conditions including gout and leukemia.

At a glance

  • Status: Approved (max clinical phase 4)
  • Modality: Small molecule
  • ATC class: M04AA01 (+1 more)
  • Targets: 1 (XDH)
  • Indications: 46 conditions
  • Clinical trials: 169
  • Chemistry: 136.11 Da · C5H4N4O

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL1467
NameAllopurinol
TypeSmall molecule
Max phase4
FDA approvedyes
PubChem CID135401907
ChEBICHEBI:40279
ATCM04AA01, M04AA51
Molecular formulaC5H4N4O
Molecular weight136.11
InChIKeyOFCNXPDARWKPPY-UHFFFAOYSA-N

SMILES: C1=NNC2=C1C(=O)NC=N2

IUPAC name: 1,5-dihydropyrazolo[5,4-d]pyrimidin-4-one

ChEBI definition: A bicyclic structure comprising a pyrazole ring fused to a hydroxy-substituted pyrimidine ring.

Pharmacological roles (ChEBI): EC 1.17.3.2 (xanthine oxidase) inhibitor, radical scavenger, gout suppressant.

Other ChEBI roles (chemical / environmental): antimetabolite.

Also known as: Allopurinol, Allopurinol component of duzallo, Allopurinolum, Alopurinol, Aloral, Aluline 100, Aluline 300, Caplenal, Cosuric, Hamarin 100, Hamarin 300, Ledopur

Parent form; salt/anhydrous children: CHEMBL1200477

Patent coverage: 25,695 distinct patent families (85,212 SureChEMBL compound mentions), from 8 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

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

GeneTargetActionpAffinityCancer dependencyUniProt
XDHxanthine dehydrogenaseInhibition5.150%P47989

Broader ChEMBL bioactivity targets: 14 (assay-derived). Sample: Xanthine dehydrogenase/oxidase, Survival motor neuron protein, Prelamin-A/C, RecQ-like DNA helicase BLM, Ras-related protein Rab-9A, Xanthine dehydrogenase/oxidase, Thyrotropin receptor, Sodium-dependent serotonin transporter, Alpha-1A adrenergic receptor, Adenosine receptor A2a.

Bioactivity

ChEMBL activities: 102 potent at pChembl ≥ 5 of 128 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
P804579.27Kd0.54nMCHEMBL_ACT_18252133
XDH8.46IC503.5nMCHEMBL_ACT_28359014
XDH8.46IC503.5nMCHEMBL_ACT_28359074
XDH8.44IC503.6nMCHEMBL_ACT_16807583
XDH7.3IC5050nMCHEMBL_ACT_25597678
XDH7Ki100nMCHEMBL_ACT_15008226
XDH7IC50100nMCHEMBL_ACT_18252135
XDH6.75IC50180nMCHEMBL_ACT_3214644
XDH6.7IC50200nMCHEMBL_ACT_16437882
XDH6.62IC50240nMCHEMBL_ACT_2332853
P804576.6IC50250nMCHEMBL_ACT_18690408
P804576.58IC50260nMCHEMBL_ACT_18252109
P804576.58IC50260nMCHEMBL_ACT_456785
Q005196.52IC50300nMCHEMBL_ACT_25597635
BLM6.4Potency398.1nMCHEMBL_ACT_4741957
BLM6.4Potency398.1nMCHEMBL_ACT_4913797
XDH6.22IC50600nMCHEMBL_ACT_24751949
XDH6.16Ki700nMCHEMBL_ACT_20627770
XDH6.16Ki700nMCHEMBL_ACT_24885798
P804576.14IC50730nMCHEMBL_ACT_5131227
P229856.12IC50753nMCHEMBL_ACT_5131206
XDH6.02IC50950nMCHEMBL_ACT_16639498
P804576Ki1000nMCHEMBL_ACT_14679765
XDH6IC501000nMCHEMBL_ACT_18322300
P804576IC501000nMCHEMBL_ACT_18960957
RAB9A6Potency1000nMCHEMBL_ACT_3860561
SMN16Potency1000nMCHEMBL_ACT_3863375
XDH5.81Ki1550nMCHEMBL_ACT_16430646
P804575.8IC501600nMCHEMBL_ACT_2430368
XDH5.76IC501730nMCHEMBL_ACT_25726935

Target pathways

Aggregated over 1 target gene(s): XDH.

Top Reactome pathways

3 total, by targets touching each:

PathwayTargetsGenes
Purine catabolism1XDH
Butyrophilin (BTN) family interactions1XDH
Azathioprine ADME1XDH

Dominant GO biological processes

GO termTargets
allantoin metabolic process1
guanine catabolic process1
inosine catabolic process1
deoxyinosine catabolic process1
adenosine catabolic process1
deoxyadenosine catabolic process1
deoxyguanosine catabolic process1
AMP catabolic process1
IMP catabolic process1
lactation1
hypoxanthine catabolic process1
xanthine catabolic process1
iron-sulfur cluster assembly1
regulation of epithelial cell differentiation1
obsolete amide catabolic process1

Indications & clinical

Indications

46 indications (5 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
gout4MONDO:0005393EFO:0004274
leukemia4MONDO:0005059EFO:0000565
lymphoma4MONDO:0005062EFO:0000574
neoplasm4MONDO:0005070MONDO:0004992
cardiovascular disorder3MONDO:0004995EFO:0000319
hypertensive disorder3MONDO:0005044EFO:0000537
acute lymphoblastic leukemia3MONDO:0004967EFO:0000220
ulcerative colitis3MONDO:0005101EFO:0000729
obesity disorder3MONDO:0011122EFO:0001073
heart disorder3MONDO:0005267EFO:0003777
non-Hodgkin lymphoma3MONDO:0018908EFO:0005952
tumor lysis syndrome3MONDO:0043875EFO:1001479
congenital heart disease3MONDO:0005453EFO:0005207
type 2 diabetes mellitus3MONDO:0005148MONDO:0005148
bipolar disorder3MONDO:0004985MONDO:0004985
injury3MONDO:0021178EFO:0000546
brain disorder3MONDO:0005560HP:0001298
metabolic syndrome X2MONDO:0011565EFO:0000195
heart failure2MONDO:0005252EFO:0003144
congestive heart failure2MONDO:0005009EFO:0000373
B-cell chronic lymphocytic leukemia2MONDO:0004948EFO:0000095
myelodysplastic syndrome2MONDO:0018881EFO:0000198
acute myeloid leukemia2MONDO:0018874EFO:0000222
diabetes mellitus2MONDO:0005015EFO:0000400
plasma cell myeloma2MONDO:0009693EFO:0001378
leishmaniasis2MONDO:0011989EFO:0005044
hand-foot syndrome2MONDO:0700048EFO:1001893
chronic kidney disease2MONDO:0005300EFO:0003884
peritoneal neoplasm2MONDO:0006901MONDO:0002087
fallopian tube neoplasm2MONDO:0021092MONDO:0002158
ovarian cancer2MONDO:0008170MONDO:0008170
nephrolithiasis2MONDO:0008171EFO:0004253
essential hypertension2MONDO:0001134MONDO:0001134
cirrhosis of liver2MONDO:0005155EFO:0001422
inflammatory bowel disease1MONDO:0005265EFO:0003767
B-cell acute lymphoblastic leukemia1MONDO:0004947EFO:0000094
small cell lung carcinoma1MONDO:0008433EFO:0000702
osteoarthritis1MONDO:0005178MONDO:0005178
tuberculosis1MONDO:0018076MONDO:0018076
plasma cell neoplasm1MONDO:0004959EFO:0000200
T-cell acute lymphoblastic leukemia1MONDO:0004963EFO:0000209

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

Clinical trials

Total trials: 169.

Phase distribution

PhaseTrials
PHASE245
PHASE433
PHASE332
Not specified21
PHASE118
PHASE2/PHASE310
PHASE1/PHASE27
EARLY_PHASE13

Top trials by phase / activity

NCTPhaseStatusTitle
NCT04875702PHASE4RECRUITINGTreat-to-Target Serum Urate Versus Treat-to-Avoid Symptoms in Gout
NCT07369622PHASE4NOT_YET_RECRUITINGHLA-B*58:01-Guided Therapy for Gout: Effectiveness, Safety, and Cost-Effectiveness
NCT00477789PHASE4COMPLETEDEffects of Allopurinol on Diastolic Function in Chronic Heart Failure Patients
NCT00512057PHASE4COMPLETEDAPEX Study: Effects of Allopurinol on Coronary and Peripheral Endothelial Function in Patients With Cardiac Syndrome X
NCT00560079PHASE4COMPLETEDEfficacy of Allopurinol and Dypiridamole in Acute Mania
NCT00643123PHASE4COMPLETEDAllopurinol Add-on Treatment for Refractory Mania
NCT00688480PHASE4COMPLETEDDo Xanthine Oxidase Inhibitors Reduce Both Left Ventricular Hypertrophy and Endothelial Dysfunction in Cardiovascular Patients With Renal Dysfunction?
NCT00732251PHASE4TERMINATEDAllopurinol Maintenance Study for Bipolar Disorder
NCT00864825PHASE4COMPLETEDAllopurinol in Schizophrenia: A Randomized Trial Administering Allopurinol vs Placebo as add-on Antipsychotics in Patients With Schizophrenia
NCT00919243PHASE4COMPLETEDPrednisone for Heart Failure Patients With Hyperuricemia
NCT00997542PHASE4COMPLETEDAllopurinol in Chronic Heart Failure
NCT01147705PHASE4COMPLETEDAllopurinol as a Possible Oxygen Sparing Agent During Exercise in Peripheral Arterial Disease
NCT01310673PHASE4COMPLETEDInitiation of Allopurinol at First Medical Contact for Acute Attacks of Gout
NCT01391325PHASE4COMPLETEDAllopurinol Outcome Study
NCT01451645PHASE4COMPLETEDEfficacy and Safety of Colchicine for the Prevention of Gout Flares During the Initiation of Allopurinol
NCT01457820PHASE4TERMINATEDAllopurinol in Acute Coronary Syndrome
NCT01550107PHASE4COMPLETEDAllopurinol in Functional Impairment (ALFIE) Trial: ‘Improving Muscle Strength’
NCT01951404PHASE4COMPLETEDDoes ALlopurinol Regress lefT Ventricular Hypertrophy in End Stage REnal Disease: The ALTERED Study
NCT01988402PHASE4COMPLETEDDoes Allopurinol Prolong a Treated, Acute Gout Flare?
NCT02122718PHASE4COMPLETEDXILO-FIST, the Effect of Allopurinol on the Brain Heart and Blood Pressure After Stroke
NCT02237339PHASE4COMPLETEDDoes Allopurinol Reduce Thickening of the Left Ventricle of the Heart in Patient With Treated Hypertension?
NCT02477488PHASE4COMPLETEDOptimal Administration of Allopurinol in Dialysis Patients
NCT02500641PHASE4COMPLETEDIntensive Urate Lowering Therapy of Febuxostat Compared to Allopurinol on Cardiovascular Risk in Patients With Gout
NCT02600780PHASE4COMPLETEDZurig (Febuxostat) 40mg Efficacy and Safety Trial
NCT02752633PHASE4COMPLETEDEffect of Allopurinol and Febuxostat on Urinary 2,8-Dihydroxyadenine Excretion
NCT02797028PHASE4UNKNOWNInvestigation of the Atherogenic Effects of Uric Acid-modified in Endothelial Dysfunction and the the Preventional Mechanism by Anthocyanidines
NCT02829177PHASE4COMPLETEDMicroalbuminuria and Allopurinol in Type 1 Diabetes
NCT02956278PHASE4COMPLETEDThe Effects of BCRP Q141K on Allopurinol Pharmacokinetics and Dynamics
NCT03195153PHASE4UNKNOWNEffect of Allopurinol on Mono and Co-administration With Statins on Platelets Reactivity on Diabetic Patiets Treated With Aspirin and Insulin
NCT03700645PHASE4UNKNOWNAllopurinol in Diabetes Mellitus and Multivessel Coronary Artery Disease
NCT03745729PHASE4COMPLETEDAllopurinol in Acute Coronary Syndrome
NCT05474560PHASE4COMPLETEDFebuxostat Versus Allopurinol on Hepatic Steatosis in MAFLD Patients
NCT05540184PHASE4UNKNOWNAllopurinol and Trimetazidine as a Preventive of Acute Kidney Injury in PCI Patients
NCT03150693PHASE3RECRUITINGInotuzumab Ozogamicin and Frontline Chemotherapy in Treating Young Adults With Newly Diagnosed B Acute Lymphoblastic Leukemia
NCT04217421PHASE3RECRUITINGCerebrum and Cardiac Protection With Allopurinol in Neonates With Critical Congenital Heart Disease Requiring Cardiac Surgery With Cardiopulmonary Bypass
NCT05943821PHASE3RECRUITINGThe Effect of Allopurinol on the Risk of Cardiovascular Events in Patients with Cardiovascular Risk
NCT07089875PHASE3RECRUITINGA Study of Dotinurad Versus Allopurinol in Participants With Gout
NCT07089888PHASE3RECRUITINGA Study of Dotinurad Versus Allopurinol in Tophaceous Gout
NCT07574060PHASE2/PHASE3NOT_YET_RECRUITINGAllopurinol in Depression
NCT00000658PHASE3COMPLETEDA Phase III Randomized Trial of Low-Dose Versus Standard-Dose mBACOD Chemotherapy With rGM-CSF for Treatment of AIDS-Associated Non-Hodgkin’s Lymphoma

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

14 molecules share ≥1 primary target. Top 14 by shared-target count:

MoleculeSourceStatusShared targets
FEBUXOSTATChEMBL + PubChemPhase 4 (approved)XDH
INDOMETHACINChEMBLPhase 4 (approved)XDH
THIOGUANINEChEMBLPhase 4 (approved)XDH
ADENINEChEMBLPhase 3XDH
QUERCETINChEMBLPhase 3XDH
RUTINChEMBLPhase 3XDH
BAICALEINChEMBLPhase 2XDH
BROPIRIMINEChEMBLPhase 2XDH
FISETINChEMBLPhase 2XDH
GENISTEINChEMBLPhase 2XDH
ISOQUERCETINChEMBLPhase 2XDH
LUTEOLINChEMBLPhase 2XDH
OXYPURINOLChEMBLPhase 2XDH
TOPIROXOSTATChEMBLPhase 2XDH

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 30

This page pulls from 30 datasets indexed by BioBTree:

chebichembl_activitychembl_moleculechembl_targetcivic_evidenceclingen_dosageclinical_trialsdepmapefoensemblentrezgogoparentgtopdbgtopdb_interactiongtopdb_ligandhgncmeshmondomsigdbpatent_compoundpharmgkbpharmgkb_guidelinepubchempubchem_activityreactomereactomeparentrefseqtranscriptuniprot