Ethosuximide

Summary

Ethosuximide (CHEMBL696) is an approved small-molecule anticonvulsant (ATC N03AD51); indicated across 8 conditions including epilepsy and irritable bowel syndrome.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: N03AD51 (+1 more)
• Indications: 8 conditions
• Clinical trials: 9
• Chemistry: 141.17 Da · C7H11NO2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL696
Name	Ethosuximide
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	3291
ChEBI	CHEBI:4887
ATC	N03AD51, N03AD01
Molecular formula	C7H11NO2
Molecular weight	141.17
InChIKey	HAPOVYFOVVWLRS-UHFFFAOYSA-N

SMILES: CCC1(CC(=O)NC1=O)C

IUPAC name: 3-ethyl-3-methylpyrrolidine-2,5-dione

ChEBI definition: A dicarboximide that is pyrrolidine-2,5-dione in which the hydrogens at position 3 are substituted by one methyl and one ethyl group. An antiepileptic, it is used in the treatment of absence seizures and may be used for myoclonic seizures, but is ineffective against tonic-clonic seizures.

Pharmacological roles (ChEBI): anticonvulsant, geroprotector, T-type calcium channel blocker.

Also known as: CI-366, CN-10,395, CN-10395, Emeside, Ethosuximide, Ethosuximidum, Etosuximida, NSC-64013, PM-671, Pyknolepsinum, Suxinutin, Zarontin

Patent coverage: 4,441 distinct patent families (15,120 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 14,790 (98%) of the total. Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Broader ChEMBL bioactivity targets: 2 (assay-derived). Sample: Nuclear receptor ROR-gamma, Neuropeptide S receptor.

Bioactivity

ChEMBL activities: 1 potent at pChembl ≥ 5 of 2 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
P51450	5.55	Potency	2818	nM	CHEMBL_ACT_5008154

Target pathways

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

Indications & clinical

Indications

8 indications (1 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
irritable bowel syndrome	3	MONDO:0005052	EFO:0000555
visual epilepsy	3	MONDO:0001386	HP:0001250
complex regional pain syndrome	2	MONDO:0019369	EFO:1001998
neuralgia	2	MONDO:0021667	EFO:0005762
migraine disorder	1	MONDO:0005277	MONDO:0005277
depressive disorder	0	MONDO:0002050	MONDO:0002050

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

Clinical trials

Total trials: 9.

Phase distribution

Phase	Trials
PHASE2	4
PHASE3	2
PHASE1/PHASE2	1
EARLY_PHASE1	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04217733	PHASE3	RECRUITING	Ethosuximide and Pentoxifylline in the Treatment of Abdominal Pain Related to Irritable Bowel Syndrome
NCT00088452	PHASE3	COMPLETED	Childhood Absence Epilepsy Rx PK-PD-Pharmacogenetics Study
NCT00689585	PHASE2	TERMINATED	Safety and Efficacy Study of Ethosuximide for the Treatment of Complex Regional Pain Syndrome (CRPS)
NCT01122381	PHASE1/PHASE2	TERMINATED	Comparison of a Drug and Placebo in the Prevention of Migraine Headaches
NCT01278004	PHASE2	COMPLETED	Chemotherapy Induced Painful Peripheral Neuropathy Ethosuximide (The CIN-E Study)
NCT02973542	PHASE2	COMPLETED	Ethosuximide to Treat IBS
NCT04431778	PHASE2	UNKNOWN	Evaluation of the Efficacy and Tolerance of Low Doses of Ethosuximide in the Treatment of Peripheral Neuropathic Pain
NCT03887624	EARLY_PHASE1	COMPLETED	Investigate the Clinical Responses of Ethosuximide in Patients With Treatment-Resistant Depression.
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, but PharmGKB curates 1 clinical and 3 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

Related molecules

Related molecules

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