Gabapentin

Summary

Gabapentin (CHEMBL940) is an approved small-molecule anticonvulsant (ATC N02BF01) targeting KCNQ3 and KCNQ5; indicated across 67 conditions including epilepsy and visual epilepsy.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: N02BF01
• Targets: 2 (KCNQ3, KCNQ5)
• Indications: 67 conditions
• Clinical trials: 357
• Chemistry: 171.24 Da · C9H17NO2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL940
Name	Gabapentin
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	3446
ChEBI	CHEBI:42797
ATC	N02BF01
Molecular formula	C9H17NO2
Molecular weight	171.24
InChIKey	UGJMXCAKCUNAIE-UHFFFAOYSA-N

SMILES: C1CCC(CC1)(CC(=O)O)CN

IUPAC name: 2-[1-(aminomethyl)cyclohexyl]acetic acid

ChEBI definition: A γ-amino acid that is cyclohexane substituted at position 1 by aminomethyl and carboxymethyl groups. Used for treatment of neuropathic pain and restless legs syndrome.

Pharmacological roles (ChEBI): anticonvulsant, calcium channel blocker.

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

Also known as: CI-945, DM-1796, Gabapentin, Gabapentina, Gabapentine, GOE 3450, GOE-3450, Gralise, Neurontin, NSC-742194, NSC-759254, gabapentin

Patent coverage: 11,893 distinct patent families (46,522 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).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
KCNQ3	Kv7.3	Activation	8.28	0.1%	O43525
KCNQ5	Kv7.5	Activation	8.72	1.2%	Q9NR82

Broader ChEMBL bioactivity targets: 11 (assay-derived). Sample: Ubiquitin carboxyl-terminal hydrolase 2, Survival motor neuron protein, Prelamin-A/C, RecQ-like DNA helicase BLM, Voltage-dependent calcium channel subunit alpha-2/delta-1, Thyrotropin receptor, Muscarinic acetylcholine receptor M1, Cytochrome P450 1A2, Voltage-dependent calcium channel subunit alpha-2/delta-1, Aldehyde dehydrogenase 1A1.

Bioactivity

ChEMBL activities: 6 potent at pChembl ≥ 5 of 13 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
CACNA2D1	7.57	IC50	27	nM	CHEMBL_ACT_331188
O08532	6.92	Ki	120	nM	CHEMBL_ACT_1685905
O08532	6.92	Ki	120.2	nM	CHEMBL_ACT_1685906
SMN1	5.85	Potency	1412	nM	CHEMBL_ACT_3903609
ALDH1A1	5.2	Potency	6310	nM	CHEMBL_ACT_4143272
P54290	5.05	IC50	8900	nM	CHEMBL_ACT_1710449

Target pathways

Aggregated over 2 target gene(s): KCNQ3, KCNQ5.

Top Reactome pathways

8 total, by targets touching each:

Pathway	Targets	Genes
Neuronal System	2	KCNQ3, KCNQ5
Potassium Channels	2	KCNQ3, KCNQ5
Voltage gated Potassium channels	2	KCNQ3, KCNQ5
Developmental Biology	1	KCNQ3
L1CAM interactions	1	KCNQ3
Axon guidance	1	KCNQ3
Interaction between L1 and Ankyrins	1	KCNQ3
Nervous system development	1	KCNQ3

Dominant GO biological processes

GO term	Targets
potassium ion transmembrane transport	2
monoatomic ion transport	2
potassium ion transport	2
monoatomic ion transmembrane transport	2
transmembrane transport	2
protein targeting	1
protein import into nucleus	1
exocytosis	1
endocytosis	1
chemical synaptic transmission	1
sensory perception of sound	1
gene expression	1
response to auditory stimulus	1
neuron remodeling	1
neuronal action potential	1

Indications & clinical

Indications

67 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).

Indication	Trial phase	MONDO	EFO
epilepsy	4	MONDO:0005027	EFO:0000474
visual epilepsy	4	MONDO:0001386	HP:0001250
restless legs syndrome	4	MONDO:0005391	EFO:0004270
neuralgia	4	MONDO:0021667	EFO:0009430
insomnia	3	MONDO:0013600	EFO:0004698
scoliosis	3	MONDO:0005392	EFO:0004273
carpal tunnel syndrome	3	MONDO:0007275	EFO:0004143
fibromyalgia	3	MONDO:0005546	EFO:0005687
focal epilepsy	3	MONDO:0005384	EFO:0004263
cirrhosis of liver	3	MONDO:0005155	EFO:0001422
opiate dependence	3	MONDO:0005530	EFO:0005611
diabetic neuropathy	3	MONDO:0006626	EFO:1000783
pelvic organ prolapse	3	MONDO:0000082	EFO:0004710
anxiety	3	MONDO:0011918	EFO:0005230
oropharynx cancer	3	MONDO:0004608	EFO:1001931
dementia	3	MONDO:0001627	HP:0000726
head and neck cancer	3	MONDO:0005627	EFO:0006859
dyspepsia	3	MONDO:0002268	EFO:0008533
spermatogenic failure	3	MONDO:0004983	EFO:0000279
intervertebral disk degenerative disorder	3	MONDO:0011385	HP:0008419
herpes zoster	3	MONDO:0005609	EFO:0006510
delirium	3	MONDO:0045057	EFO:0009267
depressive disorder	3	MONDO:0002050	MONDO:0002050
lung disorder	3	MONDO:0005275	EFO:0003818
alcohol abuse	3	MONDO:0002046	MONDO:0007079
brain injury	3	MONDO:0043510	MONDO:0043510
interstitial cystitis	3	MONDO:0018301	EFO:0008507
alcohol withdrawal	2	MONDO:0005433	EFO:0004777
nicotine dependence	2	MONDO:0008575	EFO:0003768
drug dependence	2	MONDO:0005303	EFO:0003890
reproductive system disorder	2	MONDO:0005039	EFO:0000512
hyperemesis gravidarum	2	MONDO:0006791	EFO:1000971
mucositis	2	MONDO:0020579	EFO:1001898
injury	2	MONDO:0021178	EFO:0000546
cannabis dependence	2	MONDO:0005689	EFO:0007191
rotator cuff syndrome	2	MONDO:0007028	EFO:1001250
breast neoplasm	2	MONDO:0021100	MONDO:0007254
HIV infectious disease	2	MONDO:0005109	EFO:0000764
sleep disorder	2	MONDO:0100081	EFO:0008568
multiple sclerosis	2	MONDO:0005301	MONDO:0005301
sickle cell disease	2	MONDO:0011382	MONDO:0011382
neoplasm	2	MONDO:0005070	MONDO:0004992
peripheral neuropathy	2	MONDO:0005244	EFO:0003100
psychotic disorder	1	MONDO:0005485	EFO:0005407
glioblastoma	1	MONDO:0018177	EFO:0000519
autism spectrum disorder	0	MONDO:0005258	EFO:0003756
alopecia	0	MONDO:0004907	EFO:1002028

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

Clinical trials

Total trials: 357.

Phase distribution

Phase	Trials
PHASE4	113
Not specified	83
PHASE3	66
PHASE2	45
PHASE2/PHASE3	18
EARLY_PHASE1	13
PHASE1	12
PHASE1/PHASE2	7

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03625011	PHASE4	RECRUITING	Gabapentin Premedication to Reduce Postoperative Pain for Pediatric Tonsillectomy
NCT04240626	PHASE4	ACTIVE_NOT_RECRUITING	Multimodal Analgesia Effect on Post Surgical Patient
NCT04860141	PHASE4	RECRUITING	Efficacy of Preoperative Administration of Gabapentin in 3rd Molar Dental Extraction.
NCT05076110	PHASE4	RECRUITING	Nonopioid Pain Control Regimen After Arthroscopic Hip Procedures
NCT05302999	PHASE4	RECRUITING	Feasibility of Early Gabapentin as an Intervention for Neurorecovery
NCT05690282	PHASE4	ACTIVE_NOT_RECRUITING	Comparing Perioperative Outcomes in Pain Control
NCT06379425	PHASE4	RECRUITING	Impact of Preoperative Opioid-free Multimodal Analgesia on Time to Trial of Void in Ambulatory Urogynecologic Surgeries
NCT06997016	PHASE4	ENROLLING_BY_INVITATION	Enhanced Recovery After Surgery (ERAS) in Live Donor Kidney Transplant Recipients
NCT07158476	PHASE4	RECRUITING	Efficacy of Methylprednisolone for Pain Control After ACL Repair
NCT00153283	PHASE4	COMPLETED	Study of Efficacy of Gabapentin in Therapy of Bronchial Asthma
NCT00169013	PHASE4	COMPLETED	Gabapentin in Phantom and Stump Pain
NCT00189072	PHASE4	TERMINATED	Effects of Gabapentin in the Treatment of Neuropathic Pain on Driving Performance and Cognition
NCT00209872	PHASE4	UNKNOWN	Optimal Multimodal Analgesia in Abdominal Hysterectomy
NCT00209885	PHASE4	UNKNOWN	Optimal Multimodal Analgesia in Laparoscopic Cholecystectomy
NCT00236223	PHASE4	TERMINATED	The Effect of Gabapentin, Ketamine and Dexamethasone on Pain and Opioid Requirements After Hip Surgery
NCT00385671	PHASE4	COMPLETED	An Open-Label Comparison of Duloxetine to Other Alternatives for the Management of Diabetic Peripheral Neuropathic Pain
NCT00537940	PHASE4	COMPLETED	Comparative Study Of Pregabalin And Gabapentin As Adjunctive Therapy In Subjects With Partial Seizures
NCT00584779	PHASE4	TERMINATED	An Open Label Pharmacokinetic Study Of Gabapentin In Japanese Subjects With Renal Impairment Including Hemodialysis
NCT00619983	PHASE4	TERMINATED	Three Way Interaction Between Gabapentin, Duloxetine, and Donepezil in Patients With Diabetic Neuropathy
NCT00634543	PHASE4	COMPLETED	A Study to Compare Safety and Efficacy of Tramadol Hydrochloride/Acetaminophen With Gabapentin in Participants With Diabetic Neuropathy
NCT00644748	PHASE4	COMPLETED	A Study on the Efficacy and Safety of Gabapentin in the Treatment of Patients With Painful Diabetic Neuropathy
NCT00666796	PHASE4	COMPLETED	A Study to Assess the Pharmacodynamic Interaction Between Gabapentin and Ethanol in Healthy Subjects
NCT00785772	PHASE4	TERMINATED	A Post-Marketing Clinical Pharmacokinetics Study Of Gabapentin In Japanese Epileptic Subjects With Renal Impairment
NCT00799149	PHASE4	COMPLETED	Etoricoxib Versus Gabapentin for Knee Arthroscopy
NCT00800527	PHASE4	COMPLETED	Efficacy of Gabapentin in the Treatment of Chest Pain and Paresthesia in Patients With Sternotomy
NCT00855738	PHASE4	COMPLETED	A Prospective, Observational Study On The Effectiveness Of New Antiepileptic Drugs As First Bitherapy In The Daily Clinical Practice
NCT00885586	PHASE4	COMPLETED	Acupuncture in Herpes Zoster Neuralgia (ACUZoster)
NCT00904202	PHASE4	COMPLETED	A Study Of Lidocaine Patch 5% Alone, Gabapentin Alone, And Lidocaine Patch 5% And Gabapentin In Combination For The Relief Of Pain In Patients With Diverse Peripheral Neuropathic Pain Conditions
NCT00982800	PHASE4	COMPLETED	Does Postoperative Gabapentin Reduce Pain, Opioid Consumption and Anxiety and Have a Positive Effect on Health Related Quality of Life After Radical Prostatectomy?
NCT01022736	PHASE4	COMPLETED	Plasma Gabapentin Concentration During and Following Cardiac Bypass
NCT01112878	PHASE4	WITHDRAWN	Oral Clonidine & Gabapentin: Improving Recovery and Pain Management After Outpatient With Major Orthopedic Surgery
NCT01127100	PHASE4	COMPLETED	Gabapentin Versus Transdermal Fentanyl Matrix for Chronic Neuropathic Pain
NCT01177514	PHASE4	COMPLETED	The Role of Gabapentin in Postoperative Pain and Epithelialization After Photorefractive Keratectomy
NCT01364298	PHASE4	COMPLETED	Efficacy and Safety of Gabapentin/B-complex Versus Pregabalin in Diabetic Peripheral Neuropathy Pain Management
NCT01507363	PHASE4	COMPLETED	Analgesic Effect of Gabapentin in Total Knee Arthroplasty (TKA)
NCT01573052	PHASE4	COMPLETED	Gabapentin vs Chlordiazepoxide for Ambulatory Alcohol Withdrawal
NCT01678911	PHASE4	TERMINATED	Efficacy of Gralise® for Chronic Pelvic Pain
NCT01680549	PHASE4	COMPLETED	Pain Control With Total Knee Replacement
NCT01868425	PHASE4	COMPLETED	ACL Repair and Multimodal Analgesia
NCT01966172	PHASE4	COMPLETED	Multimodal Analgesic Treatment Versus Traditional Morphine Analgesia After Cardiac Surgery

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 3 clinical and 10 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

Related molecules

Related molecules

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.

3 molecules share ≥1 primary target. Top 3 by shared-target count:

Molecule	Source	Status	Shared targets
EZOGABINE	ChEMBL	Phase 4 (approved)	KCNQ3, KCNQ5
FLINDOKALNER	ChEMBL	Phase 3	KCNQ3, KCNQ5
FLUPIRTINE	ChEMBL	Phase 4 (approved)	KCNQ3