Drug

Pilocarpine

In clinical development

ClassMuscarinic acetylcholine receptor M3 agonistEmerging researchLate-stage developmentSteady momentum

Also known as Adsorbocarpine, Akarpine, E-Pilo-1, E-Pilo-2+21 more

Adsorbocarpine, Akarpine, E-Pilo-1, E-Pilo-2, E-Pilo-4, E-Pilo-6, Isopto Carpine, Isoptocarpine, Ocu-Carpine, P1E1, P2E1, P3E1, P4E1, P6E1, Pilocar, Pilocarpine Nitrate, Pilocarpine, Ophthalmic, Pilostat, Qlosi, Salagen, Vuity, PILOKARPIN, Pilocarpina, 2(3H)-FURANONE, 3-ETHYLDIHYDRO-4-((1-METHYL-1H-IMIDAZOL-5-YL)METHYL)-, (3S-CIS)-, Pilocarpine Hydrochloride.

RxNorm8328 ChEMBLCHEMBL550 ATCN07AX01 ATCS01EB01 ATCS01EB51UNII01MI4Q9DI3

Research papers

Active clinical trials

Protein targets

Latest activity

beta

Recent clinical, regulatory, research and industry developments relating to this drug.

Randomized, Double-Masked, Placebo-Controlled, Multicenter, Phase 3 Study of the Safety and Efficacy of Nyxol (Phentolamine Ophthalmic Solution 0.75%) as a Single Agent and With Adjunctive Low-Dose Pilocarpine Hydrochloride Ophthalmic Solution 0.4% in Subjects With Presbyopia

Clinical trial2026-03-12Results posted · ClinicalTrials.gov

A Prospective Randomized Control Trial of Pilocarpine Use After Combined Cataract/Kahook Dual Blade Surgery

Clinical trial2025-11-10Results expected Q2 2028 · ClinicalTrials.gov

Safety and Efficacy of Nyxol (0.75% Phentolamine Ophthalmic Solution) With Pilocarpine Eye Drops in Subjects With Presbyopia

Clinical trial2025-10-10Results posted · ClinicalTrials.gov

Xerostomia in Patients With a Life-limiting Condition or Frailty: a Double-blind Placebo-controlled Randomized Clinical Trial

Clinical trial2025-02-01Primary completion · ClinicalTrials.gov

Efficacy of Pilocarpine As a Secretagogue Versus Artificial Tears for the Treatment of Dry Eye

Clinical trial2024-12-20Completed · ClinicalTrials.gov

A phosphodiesterase 4 (PDE4) inhibitor, amlexanox, reduces neuroinflammation and neuronal death after pilocarpine-induced seizure.

Research2024-04-16Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics

Anterior Segment Imaging With Ultrahigh-resolution OCT in Patients With Glaucoma and Pseudoexfoliation Syndrome - a Pilot Study

Clinical trial2023-12-01Primary completion · ClinicalTrials.gov

Efficacy of Pilocarpine 1% in Pseudophakia Individuals With Presbyopia

Clinical trial2022-12-30Primary completion · ClinicalTrials.gov

Comparison of Pilocarpine, Brimonidine, Oxymetazoline, Hialuronic Acid, Bromfenac Ophthalmic Compound With Pilocarpine and Brimonidine to Improve Uncorrected Visual Acuity in Healthy Presbyopic Individuals

Clinical trial2021-08-30Primary completion · ClinicalTrials.gov

Phase 2 results from Radiation Therapy Oncology Group Study 0537: a phase 2/3 study comparing acupuncture-like transcutaneous electrical nerve stimulation versus pilocarpine in treating early radiation-induced xerostomia.

Research2012-01-17Cancer

A phase III randomized, double-blind, placebo-controlled study of pilocarpine for vaginal dryness: North Central Cancer Treatment group study N04CA.

Research2011-05-01The journal of supportive oncology

Dentate granule cell neurogenesis is increased by seizures and contributes to aberrant network reorganization in the adult rat hippocampus.

Research1997-05-01The Journal of neuroscience : the official journal of the Society for Neuroscience

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name

Pilocarpine

Aliases & brands

AdsorbocarpineAkarpineE-Pilo-1E-Pilo-2E-Pilo-4E-Pilo-6Isopto CarpineIsoptocarpineOcu-CarpineP1E1P2E1P3E1P4E1P6E1PilocarPilocarpine NitratePilocarpine, OphthalmicPilostatQlosiSalagenVuityPILOKARPINPilocarpina2(3H)-FURANONE, 3-ETHYLDIHYDRO-4-((1-METHYL-1H-IMIDAZOL-5-YL)METHYL)-, (3S-CIS)-Pilocarpine Hydrochloride

RxNorm CUI

8328

ChEMBL ID

CHEMBL550

ATC codes

N07AX01S01EB01S01EB51

UNII

01MI4Q9DI3

Primary mechanism

Muscarinic acetylcholine receptor M3 agonist

Pharmacology & targets

2 targets

Known molecular targets and mechanisms supported by curated pharmacology databases.

Muscarinic acetylcholine receptor M3

CHRM3 · P20309

AGONISTMuscarinic acetylcholine receptor M3 agonist

Muscarinic acetylcholine receptor M1

CHRM1 · P11229

AGONISTMuscarinic acetylcholine receptor M1 agonist

Clinical trials

31 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme

CLINICALTRIALS.GOV · LIVE REGISTRY

registered trials across all phases

LATEST COMPLETION 2024

Active studies

Recruiting

Late-stage (III+)

Completed

Discontinued

PHASE DISTRIBUTIONn = 31

Early Phase 11Phase 14Phase 21Phase 2 / 31Phase 310Phase 42Phase N / A12

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

Effect of Pilocarpine in Patients With Xerostomia

Unknown statusPH IVUniversity of Sao Paulon=802017

Xerostomy Treatment in Patients With Sjogren's Syndrome in Chile :A Double Blind Control Trial Comparing Orally Pilocarpine Drops and Artificial Saliva

CompletedPH IVPontificia Universidad Catolica de Chilen=762008

A Prospective Randomized Control Trial of Pilocarpine Use After Combined Cataract/Kahook Dual Blade Surgery

RecruitingPH IIIMontefiore Medical Centern=1422028

Xerostomia in Patients With a Life-limiting Condition or Frailty: a Double-blind Placebo-controlled Randomized Clinical Trial

Unknown statusPH IIIMaastricht University Medical Centern=1202025

CompletedPH IIIOcuphire Pharma, Inc.n=3332023

Recent completions

Trials that read out recently, adding to the completed evidence base.

Efficacy of Pilocarpine As a Secretagogue Versus Artificial Tears for the Treatment of Dry Eye

CompletedN/AAl-Azhar Universityn=402024

CompletedPH IIIOcuphire Pharma, Inc.n=3332023

Safety and Efficacy of Nyxol (0.75% Phentolamine Ophthalmic Solution) With Pilocarpine Eye Drops in Subjects With Presbyopia

CompletedPH IIOcuphire Pharma, Inc.n=1502021

A Phase 3 Study of the Safety and Efficacy of 1% and 2% Pilocarpine Ophthalmic Solutions Administered With the Optejet® Microdose Dispenser for Temporary Improvement of Near Vision in Adults With Presbyopia

CompletedPH IIIEyenovia Inc.n=842021

Local Application of Pilocarpine for Relieving Dry Mouth Complaints: A Randomized Controlled Pilot Trial

CompletedPH IMaastricht University Medical Centern=142020

The Diurnal and Nocturnal Effect of Pilocarpine on Intraocular Pressure and Ocular Perfusion Pressure

CompletedN/AUniversity of Colorado, Denvern=272018

Research activity

4 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Publications over time

19972024

Most influential

Dentate granule cell neurogenesis is increased by seizures and contributes to aberrant network reorganization in the adult rat hippocampus.

The Journal of neuroscience : the official journal of the Society for Neuroscience · 1997 · 1,402 cites

Cancer · 2012 · 34 cites

A phosphodiesterase 4 (PDE4) inhibitor, amlexanox, reduces neuroinflammation and neuronal death after pilocarpine-induced seizure.

Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2024 · 14 cites

A phase III randomized, double-blind, placebo-controlled study of pilocarpine for vaginal dryness: North Central Cancer Treatment group study N04CA.

The journal of supportive oncology · 2011 · 9 cites

Related drugs

2 matches

Drugs sharing diseases, protein targets and literature with this one. Ranked by graph evidence (shared targets + diseases weighted above co-mentions).

Bromodeoxyuridine

Studied across 3 of the same disease areas as Pilocarpine in the shared literature.

3 shared diseases1 shared paper

Amlexanox

Studied across 2 of the same disease areas as Pilocarpine in the shared literature.

2 shared diseases1 shared paper

References & data sources

RxNorm (U.S. National Library of Medicine) — drug identity
ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
Europe PMC — research literature
ClinicalTrials.gov — clinical trials