Baclofen

Summary

Baclofen (CHEMBL701) is an approved small-molecule central nervous system depressant (ATC M03BX01); indicated across 24 conditions including cerebral palsy and spinal cord disorder.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: M03BX01
• Indications: 24 conditions
• Clinical trials: 85
• Chemistry: 213.66 Da · C10H12ClNO2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL701
Name	Baclofen
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	2284
ChEBI	CHEBI:2972
ATC	M03BX01
Molecular formula	C10H12ClNO2
Molecular weight	213.66
InChIKey	KPYSYYIEGFHWSV-UHFFFAOYSA-N

SMILES: C1=CC(=CC=C1C(CC(=O)O)CN)Cl

IUPAC name: 4-amino-3-(4-chlorophenyl)butanoic acid

ChEBI definition: A monocarboxylic acid that is butanoic acid substituted by an amino group at position 4 and a 4-chlorophenyl group at position 3. It acts as a central nervous system depressant, GABA agonist and muscle relaxant.

Pharmacological roles (ChEBI): muscle relaxant, central nervous system depressant, GABA agonist.

Also known as: (rs)-baclofen, BA-34,647, BA-34647, Baclofen, Baclofene, Baclofeno, Baclospas-10, Fleqsuvy, Gablofen, Kemstro, Lioresal, Lioresal intrath

Parent form; salt/anhydrous children: CHEMBL1897150

Patent coverage: 6,468 distinct patent families (25,446 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
	GABAB receptor	Full agonist	6.2

Broader ChEMBL bioactivity targets: 11 (assay-derived). Sample: Prelamin-A/C, RecQ-like DNA helicase BLM, 4’-phosphopantetheinyl transferase ffp, GABA-A receptor; anion channel, Gamma-aminobutyric acid type B receptor subunit 1, GABA-B receptor, GABA B receptor, D(3) dopamine receptor, Adenosine receptor A3, Nuclear factor NF-kappa-B p105 subunit.

Bioactivity

ChEMBL activities: 22 potent at pChembl ≥ 5 of 24 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
O09028	7.52	IC50	30	nM	CHEMBL_ACT_299698
O88871	7.52	IC50	30	nM	CHEMBL_ACT_402602
CYP3A4	7.5	Potency	31.6	nM	CHEMBL_ACT_5006027
CYP3A4	7.5	Potency	31.6	nM	CHEMBL_ACT_5074491
GABBR2	7.46	IC50	35	nM	CHEMBL_ACT_87546
GABBR2	7.3	IC50	50	nM	CHEMBL_ACT_1611113
O88871	7.22	IC50	60	nM	CHEMBL_ACT_299699
O88871	6.97	IC50	107	nM	CHEMBL_ACT_87548
O88871	6.75	IC50	180	nM	CHEMBL_ACT_1088241
O88871	6.7	IC50	200	nM	CHEMBL_ACT_1110499
BLM	6.7	Potency	199.5	nM	CHEMBL_ACT_4750005
BLM	6.7	Potency	199.5	nM	CHEMBL_ACT_4937912
O88871	6.7	IC50	200	nM	CHEMBL_ACT_587401
O88871	6.48	IC50	330	nM	CHEMBL_ACT_1088240
O88871	6.38	IC50	420	nM	CHEMBL_ACT_776391
GABBR2	5.77	EC50	1700	nM	CHEMBL_ACT_12648763
GABBR1	5.62	AC50	2384	nM	CHEMBL_ACT_25137574
ADORA3	5.38	AC50	4200	nM	CHEMBL_ACT_25134412
NFKB1	5.25	Potency	5623	nM	CHEMBL_ACT_3671894
NFKB1	5.25	Potency	5623	nM	CHEMBL_ACT_4585176
GABBR2	5.22	Ki	6000	nM	CHEMBL_ACT_15669086
LMNA	5.2	Potency	6310	nM	CHEMBL_ACT_3663315

Target pathways

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

Indications & clinical

Indications

24 indications (8 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
cerebral palsy	4	MONDO:0006497	EFO:1000632
spinal cord disorder	4	MONDO:0002545	EFO:0009488
spinal cord injury	4	MONDO:0043797	EFO:1001919
brain injury	4	MONDO:0043510	MONDO:0043510
multiple sclerosis	4	MONDO:0005301	MONDO:0005301
alcohol withdrawal	3	MONDO:0005433	EFO:0004777
gastroesophageal reflux disease	3	MONDO:0007186	EFO:0003948
alcohol abuse	3	MONDO:0002046	MONDO:0007079
Charcot-Marie-Tooth disease	3	MONDO:0015626	MONDO:0015626
cocaine dependence	2	MONDO:0005186	EFO:0002610
nicotine dependence	2	MONDO:0008575	EFO:0003768
hepatocellular carcinoma	2	MONDO:0007256	EFO:0000182
reproductive system disorder	2	MONDO:0005039	EFO:0000512
drug dependence	2	MONDO:0005303	EFO:0003890
cannabis dependence	2	MONDO:0005689	EFO:0007191
fragile X syndrome	2	MONDO:0010383	MONDO:0010383
autism	1	MONDO:0005260	EFO:0003758
obesity disorder	1	MONDO:0011122	EFO:0001073
alcoholic liver cirrhosis	1	MONDO:0006644	EFO:1000802
ataxia telangiectasia	0	MONDO:0008840	MONDO:0008840

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

Clinical trials

Total trials: 85.

Phase distribution

Phase	Trials
PHASE2	19
PHASE4	18
Not specified	17
PHASE3	11
PHASE2/PHASE3	8
PHASE1	5
EARLY_PHASE1	4
PHASE1/PHASE2	3

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00752934	PHASE4	TERMINATED	Does Oral Baclofen Improve Care and Comfort in Spastic Children in Nursing Homes?
NCT01002105	PHASE4	COMPLETED	Baclofen as Add-On to Standard Treatment of Alcohol- Dependent Patients
NCT01003249	PHASE4	TERMINATED	Dysfunctional Voiding and Lower Urinary Tract Symptoms With Baclofen
NCT01008280	PHASE4	COMPLETED	Baclofen to Reduce Alcohol Use in Veterans With HCV
NCT01032239	PHASE4	COMPLETED	SISTERS: Spasticity In Stroke Study - Randomized Study
NCT01386255	PHASE4	WITHDRAWN	Placebo Controlled Study of Baclofen for GERD in Children With Cerebral Palsy
NCT01955655	PHASE4	COMPLETED	Recurrent Crying Spells in Cerebral Palsy
NCT02462317	PHASE4	COMPLETED	Study of the Effects on Motor Recovery of Early Post-stroke Spasticity Treatment
NCT02529514	PHASE4	SUSPENDED	Usage of Baclofen for Sleep Improvement After Cardiac Surgery
NCT02596763	PHASE4	TERMINATED	Phone-based Safety Monitoring of Baclofen Prescriptions for Alcohol Use Disorder
NCT03068897	PHASE4	COMPLETED	Ibuprofen Plus Metaxolone, Tizanidine, or Baclofen for Low Back Pain
NCT03113396	PHASE4	COMPLETED	Baclofen for Rumination
NCT03293017	PHASE4	UNKNOWN	Baclofen in Managing Acute Alcohol Withdrawal
NCT03720717	PHASE4	TERMINATED	Baclofen as a Perioperative Analgesic Adjuvant
NCT03778281	PHASE4	UNKNOWN	Treatment of Chemotherapy-related Hiccups With Baclofen
NCT03860662	PHASE4	COMPLETED	The Effect of Oral Baclofen and Botulinum Toxin Treatments in Hemiplegic Spasticity on the Nociceptive Flexor Reflex
NCT04523935	PHASE4	COMPLETED	Excessive Crying in Children With Cerebral Palsy and Communication Deficits
NCT05161351	PHASE4	COMPLETED	Evaluating the Safety of Acute Baclofen in Methadone-maintained Individuals With Opiate Dependence.
NCT05921604	PHASE3	ACTIVE_NOT_RECRUITING	Baclofen Versus Gabapentin in Preventing Postoperative Pain After Laparoscopic Sleeve Gastrectomy
NCT05935553	PHASE2/PHASE3	RECRUITING	Baclofen for Improving Benzodiazepine Titration in Benzodiazepine Dependence
NCT07063576	PHASE2/PHASE3	RECRUITING	Effect of Baclofen in Management of Patients With Gastroesophageal Reflux Disease Symptoms
NCT00139789	PHASE3	COMPLETED	A Multicenter, Open-label Randomized Crossover Trial to Assess Subject Preference for Kemstro™ Compared to Conventional Baclofen Tablets in Subjects With Stable Multiple Sclerosis
NCT00367068	PHASE3	COMPLETED	Dutch National ITB Study in Children With Cerebral Palsy
NCT00516503	PHASE3	COMPLETED	Baclofen-Amitriptyline Hydrochloride-Ketamine Gel in Treating Peripheral Neuropathy Caused by Chemotherapy in Patients With Cancer
NCT00877734	PHASE2/PHASE3	COMPLETED	Efficacy and Tolerability of Baclofen for Alcohol Dependence
NCT01265550	PHASE3	COMPLETED	A Randomized Trial of Medical and Surgical Treatments for Patients With GERD Symptoms That Are Refractory to Proton Pump Inhibitors
NCT01266655	PHASE2/PHASE3	COMPLETED	Baclofen for the Treatment of Alcohol Dependence
NCT01604330	PHASE2/PHASE3	COMPLETED	Baclofen for the Treatment of Alcohol Drinkers
NCT01711125	PHASE3	COMPLETED	Baclofen in the Treatment of Alcohol Dependence With or Without Alcoholic Liver Disease
NCT01738282	PHASE3	COMPLETED	Efficacy and Safety of Baclofen for Maintenance of Abstinence in Alcohol Dependent Patients
NCT01743651	PHASE3	COMPLETED	Efficacy Study of Arbaclofen to Treat Spasticity in Multiple Sclerosis
NCT02052440	PHASE3	TERMINATED	Preventing Alcohol Withdrawal Syndrome With Oral Baclofen
NCT02099006	PHASE2/PHASE3	COMPLETED	Novel Topical Therapies for the Treatment of Genital Pain
NCT02723383	PHASE3	COMPLETED	Baclofen to Prevent Agitation in Alcohol Addicted Patients in ICU
NCT04251819	PHASE2/PHASE3	TERMINATED	Buprenorphine Plus Baclofen to Increase Analgesia in Healthy Volunteers
NCT05516953	PHASE2/PHASE3	COMPLETED	Baclofen in Preventing Postoperative Nausea and Vomiting After Bariatric Surgery
NCT05975684	PHASE3	COMPLETED	Baclofen for Children With Rumination Syndrome
NCT05418049	PHASE2	ACTIVE_NOT_RECRUITING	Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug Challenge
NCT00000303	PHASE2	COMPLETED	Rapid Evaluation of Baclofen for Treatment of Cocaine Abuse/Dependence - 6
NCT00082485	PHASE2	COMPLETED	Baclofen for the Treatment of Cocaine Dependence - 1

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

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