Vigabatrin

Summary

Vigabatrin (CHEMBL89598) is an approved small-molecule anticonvulsant (ATC N03AG04) targeting SLC32A1 and ABAT; indicated across 12 conditions including epilepsy and complex partial epilepsy.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: N03AG04
• Targets: 2 (SLC32A1, ABAT)
• Indications: 12 conditions
• Clinical trials: 21
• Chemistry: 129.16 Da · C6H11NO2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL89598
Name	Vigabatrin
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	5665
ChEBI	CHEBI:63638
ATC	N03AG04
Molecular formula	C6H11NO2
Molecular weight	129.16
InChIKey	PJDFLNIOAUIZSL-UHFFFAOYSA-N

SMILES: C=CC(CCC(=O)O)N

IUPAC name: 4-aminohex-5-enoic acid

ChEBI definition: A γ-amino acid having a γ-vinyl GABA structure. It is an irreversible inhibitor of γ-aminobutyric 664 acid transaminase

Pharmacological roles (ChEBI): anticonvulsant, EC 2.6.1.19 (4-aminobutyrate—2-oxoglutarate transaminase) inhibitor.

Also known as: CPP-109, Gamma vinyl gaba, Gamma-vinyl gaba, Kigabeq, MDL 71,754, MDL-71754, RMI-71754, Sabril, Vigabatrin, Vigabatrina, Vigabatrine, Vigafyde

Parent form; salt/anhydrous children: CHEMBL4303352

Patent coverage: 2,533 distinct patent families (10,141 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
SLC32A1	Vesicular inhibitory amino acid transporter	Inhibition	2.1	0.2%	Q9H598
ABAT	4-aminobutyrate aminotransferase	Irreversible inhibition	3.07	0%	P80404

Broader ChEMBL bioactivity targets: 7 (assay-derived). Sample: RecQ-like DNA helicase BLM, Endonuclease 4, Sodium-dependent noradrenaline transporter, Sodium-dependent serotonin transporter, Sodium-dependent dopamine transporter, 4-aminobutyrate aminotransferase, mitochondrial, 4-aminobutyrate aminotransferase, mitochondrial.

Bioactivity

ChEMBL activities: 1 potent at pChembl ≥ 5 of 8 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
P0A6C1	6.65	Potency	223.9	nM	CHEMBL_ACT_4069648

Target pathways

Aggregated over 2 target gene(s): SLC32A1, ABAT.

Top Reactome pathways

4 total, by targets touching each:

Pathway	Targets	Genes
R-HSA-425393	1	SLC32A1
SLC-mediated transport of neurotransmitters	1	SLC32A1
GABA synthesis, release, reuptake and degradation	1	SLC32A1
Degradation of GABA	1	ABAT

Dominant GO biological processes

GO term	Targets
beta-alanine transport	1
monoatomic ion transport	1
neurotransmitter secretion	1
gamma-aminobutyric acid transport	1
glycine transport	1
hippocampus development	1
gamma-aminobutyric acid import	1
neurotransmitter loading into synaptic vesicle	1
amino acid transmembrane transport	1
neurotransmitter transport	1
proton transmembrane transport	1
response to hypoxia	1
copulation	1
locomotory behavior	1
response to xenobiotic stimulus	1

Indications & clinical

Indications

12 indications (6 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
complex partial epilepsy	4	MONDO:0006710	EFO:1000877
focal epilepsy	4	MONDO:0005384	EFO:0004263
cocaine dependence	2	MONDO:0005186	EFO:0002610
methamphetamine dependence	2	MONDO:0005419	EFO:0004701
obesity disorder	2	MONDO:0011122	EFO:0001073
tuberous sclerosis	2	MONDO:0001734	MONDO:0001734
alcohol abuse	2	MONDO:0002046	MONDO:0007079
Tourette syndrome	1	MONDO:0007661	EFO:0004895

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

Clinical trials

Total trials: 21.

Phase distribution

Phase	Trials
PHASE2	8
PHASE4	3
PHASE3	3
Not specified	3
PHASE2/PHASE3	2
PHASE1/PHASE2	1
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT01266291	PHASE4	TERMINATED	Sabril for Complex Partial Seizures in Adult Tolerability Study (TS) Patients
NCT01278173	PHASE4	COMPLETED	A Study of the Structure and Function of the Retina in Adult Patients With Refractory Complex Partial Seizures Treated With Vigabatrin (Sabril®)
NCT01413711	PHASE4	WITHDRAWN	An Open-Label, Single and Multiple Oral Dose Pharmacokinetic Study of Vigabatrin in Infants With Infantile Spasms
NCT01281202	PHASE2/PHASE3	COMPLETED	Vigabatrin for the Treatment of Cocaine Dependency
NCT02299115	PHASE3	WITHDRAWN	Prednisolone Versus Vigabatrin in the First-line Treatment of Infantile Spasms
NCT03347526	PHASE3	SUSPENDED	A Novel Approach to Infantile Spasms
NCT03421496	PHASE3	TERMINATED	A Study to Assess Cannabidiol Oral Solution With Vigabatrin as Initial Therapy in Participants With Infantile Spasms
NCT04987463	PHASE2/PHASE3	UNKNOWN	Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants
NCT00373581	PHASE2	TERMINATED	Effects of Vigabatrin on Cocaine Self-Administration
NCT00527683	PHASE2	COMPLETED	Double Blind Study of Vigabatrin for the Treatment of Cocaine Dependence
NCT00611130	PHASE2	COMPLETED	Vigabatrin for Treatment of Cocaine Dependence
NCT00730522	PHASE2	TERMINATED	Safety and Efficacy Study of Vigabatrin to Treat Methamphetamine Dependence
NCT01335867	PHASE2	TERMINATED	Vigabatrin for Cocaine and Alcohol Dependence
NCT01585207	PHASE1/PHASE2	COMPLETED	Proof-of-Concept Safety Study of CPP-109 (Vigabatrin) for Treatment Refractory Tourette’s Disorder
NCT02849457	PHASE2	COMPLETED	Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex
NCT04062890	PHASE2	WITHDRAWN	Inhibiting GABA Transaminase to Relieve Obesity Induced Hyperinsulinemia and Insulin Resistance
NCT04321395	PHASE2	COMPLETED	Vigabatrin and Insulin Sensitivity
NCT00626834	PHASE1	COMPLETED	Vigabatrin Ph 1 Cocaine Interaction Study
NCT04302116	Not specified	RECRUITING	Vigabatrin With High Dose Prednisolone Combination Therapy vs Vigabatrin Alone for Infantile Spasm
NCT03003143	Not specified	WITHDRAWN	Effect of Vigabatrin in Refractory Autoimmune Encephalitis Patients
NCT03196466	Not specified	COMPLETED	Population Pharmacokinetics of Antiepileptic in Pediatrics

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

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.

2 molecules share ≥1 primary target. Top 2 by shared-target count:

Molecule	Source	Status	Shared targets
.gamma.-aminobutyric acid	PubChem	Approved	ABAT
Valproic Acid	PubChem	Approved	ABAT