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Pilocarpine

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Also known as OcucarpineOcusert piloOcusert pilo-20Ocusert pilo-40PilocarpinumPilocarpolPilokarpinSpersacarpineSyncarpine(+)-PilocarpineSID170464845

Summary

Pilocarpine (CHEMBL550) is an approved small-molecule antiglaucoma drug (ATC N07AX01) targeting CHRM1, CHRM2, and CHRM3; indicated across 11 conditions including open-angle glaucoma and ocular hypertension.

At a glance

  • Status: Approved (max clinical phase 4)
  • Modality: Small molecule
  • ATC class: N07AX01 (+2 more)
  • Targets: 5 (CHRM1, CHRM2, CHRM3…)
  • Indications: 11 conditions
  • Clinical trials: 39
  • Chemistry: 208.26 Da · C11H16N2O2

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL550
NamePilocarpine
TypeSmall molecule
Max phase4
FDA approvedyes
PubChem CID5910
ChEBICHEBI:8207
ATCN07AX01, S01EB01, S01EB51
Molecular formulaC11H16N2O2
Molecular weight208.26
InChIKeyQCHFTSOMWOSFHM-WPRPVWTQSA-N

SMILES: CC[C@H]1[C@H](COC1=O)CC2=CN=CN2C

IUPAC name: (3S,4R)-3-ethyl-4-[(3-methylimidazol-4-yl)methyl]oxolan-2-one

ChEBI definition: The (+)-enantiomer of pilocarpine.

Pharmacological roles (ChEBI): antiglaucoma drug.

Also known as: Ocucarpine, Ocusert pilo, Ocusert pilo-20, Ocusert pilo-40, Pilocarpine, Pilocarpinum, Pilocarpol, Pilokarpin, Spersacarpine, Syncarpine, pilocarpine, PILOCARPINE

Parent form; salt/anhydrous children: CHEMBL1200330, CHEMBL1213136

Patent coverage: 10,020 distinct patent families (37,191 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

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
CHRM1M1 receptorPartial agonist5.10.2%P11229
CHRM2M2 receptorPartial agonist4.90%P08172
CHRM3M3 receptorPartial agonist5.10%P20309
CHRM4M4 receptorPartial agonist5.21.6%P08173
CHRM5M5 receptorPartial agonist50%P08912

Broader ChEMBL bioactivity targets: 14 (assay-derived). Sample: Muscarinic acetylcholine receptor M4, Muscarinic acetylcholine receptor, Muscarinic acetylcholine receptor M5, Muscarinic acetylcholine receptor M2, Muscarinic acetylcholine receptor M1, Cannabinoid receptor 1, 5-hydroxytryptamine receptor 2A, Muscarinic acetylcholine receptor DM1, Muscarinic acetylcholine receptor M3, Muscarinic acetylcholine receptor M1.

Bioactivity

ChEMBL activities: 37 potent at pChembl ≥ 5 of 42 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
CHRM17.76EC5017.5nMCHEMBL_ACT_15182618
CHRM17.42AC5038nMCHEMBL_ACT_25209416
P084827.17IC5068nMCHEMBL_ACT_543708
P084827.1IC5080nMCHEMBL_ACT_37760
P084827.08Ki82.9nMCHEMBL_ACT_631188
CHRM56.2EC50631nMCHEMBL_ACT_25093313
CHRM16.09EC50812.8nMCHEMBL_ACT_25093261
CHRM16.08EC50831.8nMCHEMBL_ACT_15213720
P084826.02IC50960nMCHEMBL_ACT_37761
CHRM26EC501000nMCHEMBL_ACT_25093275
P084826Ki1010nMCHEMBL_ACT_631189
P084825.96Ki1110nMCHEMBL_ACT_631190
CYP2A135.85Ki1400nMCHEMBL_ACT_15468126
CHRM35.84EC501445nMCHEMBL_ACT_25093291
CHRM15.82AC501500nMCHEMBL_ACT_25211954
CHRM15.57Kd2692nMCHEMBL_ACT_15213648
CHRM15.57Kd2692nMCHEMBL_ACT_15213748
P163955.55Ki2800nMCHEMBL_ACT_12433408
P163955.53Ki2980nMCHEMBL_ACT_12433387
CYP2A65.52Ki3000nMCHEMBL_ACT_15468118
CYP2A135.52Kd3000nMCHEMBL_ACT_15468153
P084825.51Ki3100nMCHEMBL_ACT_962224
CHRM45.48EC503311nMCHEMBL_ACT_25093297
CYP2A65.44Kd3600nMCHEMBL_ACT_15468144
P084825.43Ki3670nMCHEMBL_ACT_929680
CHRM25.42AC503803nMCHEMBL_ACT_25214828
P084825.35IC504430nMCHEMBL_ACT_1095398
HTR2A5.33AC504719nMCHEMBL_ACT_25173575
CHRM15.18Ki6607nMCHEMBL_ACT_2140351
P126575.16EC506900nMCHEMBL_ACT_178729

Target pathways

Aggregated over 5 target gene(s): CHRM1, CHRM2, CHRM3, CHRM4, CHRM5.

Top Reactome pathways

17 total, by targets touching each:

PathwayTargetsGenes
Signal Transduction5CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
Signaling by GPCR5CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
Class A/1 (Rhodopsin-like receptors)5CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
Amine ligand-binding receptors5CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
GPCR downstream signalling5CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
Muscarinic acetylcholine receptors5CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
GPCR ligand binding5CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
G alpha (q) signalling events3CHRM1, CHRM3, CHRM5
G alpha (i) signalling events2CHRM2, CHRM4
Metabolism1CHRM3
Integration of energy metabolism1CHRM3
Membrane Trafficking1CHRM2
Acetylcholine regulates insulin secretion1CHRM3
Regulation of insulin secretion1CHRM3
Vesicle-mediated transport1CHRM2
Cargo recognition for clathrin-mediated endocytosis1CHRM2
Clathrin-mediated endocytosis1CHRM2

Dominant GO biological processes

GO termTargets
signal transduction5
G protein-coupled receptor signaling pathway5
G protein-coupled receptor signaling pathway, coupled to cyclic nucleotide second messenger5
adenylate cyclase-inhibiting G protein-coupled acetylcholine receptor signaling pathway5
G protein-coupled acetylcholine receptor signaling pathway5
chemical synaptic transmission5
phospholipase C-activating G protein-coupled acetylcholine receptor signaling pathway3
nervous system development3
acetylcholine receptor signaling pathway3
regulation of locomotion2
saliva secretion2
regulation of smooth muscle contraction2
phospholipase C-activating G protein-coupled receptor signaling pathway1
neuromuscular synaptic transmission1
positive regulation of monoatomic ion transport1

Indications & clinical

Indications

11 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
open-angle glaucoma4MONDO:0005338EFO:0004190
ocular hypertension4MONDO:0006875EFO:1001069
Sjogren syndrome4MONDO:0010030EFO:0000699
angle-closure glaucoma4MONDO:0001744MONDO:0001744
glaucoma4MONDO:0005041MONDO:0005041
presbyopia3MONDO:0001330MONDO:0001330
dry eye syndrome2MONDO:0006733EFO:1000906
myopia0MONDO:0001384HP:0000545

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

Clinical trials

Total trials: 39.

Phase distribution

PhaseTrials
Not specified17
PHASE38
PHASE45
PHASE14
EARLY_PHASE13
PHASE2/PHASE31
PHASE21

Top trials by phase / activity

NCTPhaseStatusTitle
NCT07113210PHASE4RECRUITINGQuality of Life in Presbyopic Patients Who Are Treated With Qlosi
NCT00438048PHASE4COMPLETEDA Comparison of Orally Pilocarpine and Artificial Saliva in Xerostomy Treatment
NCT00762645PHASE4COMPLETEDTravoprost 0.004% Versus Pilocarpine 1% in Patients With Chronic Angle Closure Glaucoma (CACG)
NCT00803803PHASE4COMPLETEDDose, Effects and Characteristics of Pilocarpine
NCT02982577PHASE4UNKNOWNEffect of Pilocarpine in Patients With Xerostomia
NCT03933631PHASE3RECRUITINGPilocarpine Use After Kahook Goniotomy
NCT06451666PHASE3NOT_YET_RECRUITINGSafety and Efficacy Study of Pilocarpine HCl Ophthalmic Solution in Participants With Presbyopia
NCT00656513PHASE2/PHASE3COMPLETEDAcupuncture-Like Transcutaneous Electrical Nerve Stimulation (ALTENS) or Pilocarpine in Treating Early Dry Mouth in Patients Undergoing Radiation Therapy for Head and Neck Cancer
NCT03804268PHASE3COMPLETEDEfficacy Study of Pilocarpine HCl Ophthalmic Solution (AGN-190584) in Participants With Presbyopia
NCT03857542PHASE3COMPLETEDA Phase 3 Efficacy Study of Pilocarpine HCl Ophthalmic Solution (AGN-190584) in Participants With Presbyopia
NCT04005079PHASE3WITHDRAWNPilocarpine After Combined Cataract/Trabectome Surgery
NCT04657172PHASE3COMPLETEDSafety & Efficacy of Pilocarpine Eye Solutions for Temporary Improvement of Near Vision in Presbyopic Adults
NCT05506137PHASE3UNKNOWNXerostomia in Patients With a Life-limiting Condition or Frailty
NCT05578001PHASE3UNKNOWNEfficacy of Pilocarpine 1% in Presbyopia
NCT04675151PHASE2COMPLETEDSafety and Efficacy of Nyxol With Pilocarpine Eye Drops in Subjects With Presbyopia
NCT01036035PHASE1COMPLETEDEffects of Novel Combinations of Tolterodine/Pilocarpine on Pharmacokinetics (PK) and Pharmacodynamics (PD) of Tolterodine in Healthy Subjects
NCT02420782PHASE1COMPLETEDFirst-in-human: Single Ascending Dose, Food Effect, Drug-drug Interaction, Multiple Ascending Dose, Proof of Pharmacology
NCT02447315PHASE1COMPLETEDA Study to Investigate the Effect of Oral Doses of Pilocarpine on Salivary Secretion and Static Pupillometry in Healthy Subjects
NCT05006911PHASE1UNKNOWNPilocarpine, Brimonidine, Oxymetazoline (PBO) Compound to Control Presbyopia Symptoms
NCT03825081EARLY_PHASE1UNKNOWNPilocarpine and Brimonidine in Patients With Monofocal Lenses
NCT05564832EARLY_PHASE1UNKNOWNPharmacological Treatment of Presbyopia
NCT06783686EARLY_PHASE1COMPLETEDEvaluation of NV701 (Pilocarpine 1.25%) Compared With Vuity (Pilocarpine 1.25%) on Pupil Size in Subjects With Presbyopia
NCT07012512Not specifiedNOT_YET_RECRUITINGAdjusted Study Protocol: Within-Subject Paired-Eye Design Comparing Pilocarpine 0.1%, Phentolamine 0.75%, and Combination Therapy for Cyclopentolate Reversal in Children
NCT00601211Not specifiedCOMPLETEDBioequivalency Study of 5 mg Piolocarpine Tablets Under Fed Conditions
NCT00601874Not specifiedCOMPLETEDBioequivalency Study of 5 mg Pilocarpine Tablets Under Fasting Conditions
NCT00602524Not specifiedCOMPLETEDBioequivalency Study of 7.5 mg Pilocarpine Tablets Under Fasting Conditions
NCT00618059Not specifiedCOMPLETEDBioequivalency Study of 7.5 mg Pilocarpine Under Fed Conditions
NCT01627626Not specifiedCOMPLETEDEffect of 0.1% Pilocarpine Mouthwash on Xerostomia
NCT01690052Not specifiedCOMPLETEDEfficacy of Cevimeline Versus Pilocarpine in the Secretion of Saliva
NCT02613013Not specifiedUNKNOWNLaser Peripheral Iridotomy Plus Laser Peripheral Iridoplasty for Primary Angle Closure
NCT02754570Not specifiedCOMPLETEDThe Diurnal and Nocturnal Effect of Pilocarpine on Intraocular Pressure and Ocular Perfusion Pressure
NCT02865473Not specifiedUNKNOWNAnterior Segment Imaging With Ultrahigh-resolution OCT in Patients With Glaucoma and PEX - a Pilot Study
NCT02935894Not specifiedCOMPLETEDInvestigating the Stability, Variability and Mechanism of Incorporation of Lipid Mediators Into Eccrine Sweat
NCT03460964Not specifiedTERMINATEDMeasuring Sweat Glucose of Patients With Diabetes - The ENGAGE Study
NCT04085094Not specifiedCOMPLETEDGender Differences in Renal Functioning and Disease
NCT04732195Not specifiedCOMPLETEDPilocarpine Microneedles for Sweat Induction (PMN-SI)
NCT05209555Not specifiedCOMPLETEDPreoperative BAMCOG and Cortisol and Postoperative DeltaScan in AVR Patients (Aortic Valve Replacement)
NCT06354842Not specifiedCOMPLETEDMeasurement of Sweat Sodium Concentration in Patients With Chronic Kidney Disease
NCT06752278Not specifiedCOMPLETEDEfficacy of Pilocarpine As a Secretagogue Versus Artificial Tears for the Treatment of Dry Eye

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 0 clinical and 1 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

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.

576 molecules share ≥1 primary target. Top 60 by shared-target count:

MoleculeSourceStatusShared targets
ACLIDINIUM BROMIDEChEMBL + PubChemPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
CLOZAPINEChEMBL + PubChemPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
GENTIAN VIOLETChEMBL + PubChemPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
MavorixaforChEMBL + PubChemPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
OLANZAPINEChEMBL + PubChemPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
ACETYLCHOLINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
AMIODARONEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
AMITRIPTYLINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
AMOXAPINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
ASTEMIZOLEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
ATROPINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
BENZTROPINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
BETHANECHOLChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
CARBACHOLChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
CARBAMOYLCHOLINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
CHLOROQUINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
CHLORPROMAZINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
CINNARIZINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
CLEMASTINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
CLIDINIUMChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
CLOMIPRAMINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
CYCLIZINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
CYPROHEPTADINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
DARIFENACINChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
DESLORATADINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
DEXCHLORPHENIRAMINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
DICYCLOMINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
DIMENHYDRINATEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
DIPHENHYDRAMINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
DIPHENIDOLChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
DOXEPINChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
EBASTINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
GLYCOPYRRONIUM BROMIDEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
HALOPERIDOLChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
HOMATROPINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
HYDROXYCHLOROQUINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
IMIPRAMINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
IPRATROPIUMChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
KETOTIFENChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
LOXAPINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
MAPROTILINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
MEPAZINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
METHSCOPOLAMINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
METHSCOPOLAMINE BROMIDEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
MIANSERINChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
NORTRIPTYLINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
ORPHENADRINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
OXYBUTYNINChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
PAROXETINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
PERHEXILINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
PIRENZEPINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
PROCHLORPERAZINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
PROMAZINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
PROMETHAZINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
QUETIAPINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
QUINACRINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
RISPERIDONEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
SCOPOLAMINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
SERTRALINEChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5
SOLIFENACINChEMBLPhase 4 (approved)CHRM1, CHRM2, CHRM3, CHRM4, CHRM5

About this page

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

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Sources 30

This page pulls from 30 datasets indexed by BioBTree:

chebichembl_activitychembl_moleculechembl_targetcivic_evidenceclingen_dosageclinical_trialsdepmapefoensemblentrezgogoparentgtopdbgtopdb_interactiongtopdb_ligandhgncmeshmondomsigdbpatent_compoundpharmgkbpharmgkb_guidelinepubchempubchem_activityreactomereactomeparentrefseqtranscriptuniprot