Stiripentol

Summary

Stiripentol (CHEMBL1983350) is an approved small molecule (ATC N03AX17); indicated across 6 conditions including epilepsy and visual epilepsy.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: N03AX17
• Indications: 6 conditions
• Clinical trials: 11
• Chemistry: 234.29 Da · C14H18O3

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1983350
Name	Stiripentol
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	5311454
ATC	N03AX17
Molecular formula	C14H18O3
Molecular weight	234.29
InChIKey	IBLNKMRFIPWSOY-FNORWQNLSA-N

SMILES: CC(C)(C)C(/C=C/C1=CC2=C(C=C1)OCO2)O

IUPAC name: (E)-1-(1,3-benzodioxol-5-yl)-4,4-dimethylpent-1-en-3-ol

Also known as: BCX 2600, BCX-2600, Diacomit, Estiripentol, Stiripentol, SID124896656, STIRIPENTOL, SID144207171, StiripentolÊ, stiripentol, StiripentolÂ, C0164870

Patent coverage: 788 distinct patent families (2,890 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 2,887 (100%) 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: 8 (assay-derived). Sample: Androgen receptor, Amine oxidase [flavin-containing] A, Progesterone receptor, GABA-A receptor; alpha-1/beta-2/gamma-2, Sodium-dependent noradrenaline transporter, Sodium-dependent dopamine transporter, 3’,5’-cyclic-AMP phosphodiesterase 4A, Vascular endothelial growth factor receptor 2.

Bioactivity

ChEMBL activities: 3 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
AR	5.52	AC50	3010	nM	CHEMBL_ACT_25203252
SLC6A2	5.44	AC50	3655	nM	CHEMBL_ACT_25145894
SLC6A3	5.33	AC50	4647	nM	CHEMBL_ACT_25124853

Target pathways

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

Indications & clinical

Indications

6 indications (4 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
juvenile myoclonic epilepsy	4	MONDO:0009696	MONDO:0009696
primary hyperoxaluria	2	MONDO:0002474	MONDO:0002474
chronic kidney disease	1	MONDO:0005300	EFO:0003884

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

Clinical trials

Total trials: 11.

Phase distribution

Phase	Trials
Not specified	6
PHASE1	2
PHASE4	1
PHASE3	1
PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05419180	PHASE4	COMPLETED	Study of Interest of Stiripentol and Carbamazepine in the Treatment of Patients With Pharmacoresistant Focal Epilepsies
NCT06465472	PHASE3	NOT_YET_RECRUITING	Evaluation of the Efficacy and Safety of Stiripentol in Patients 6 Years and Older With Primary Hyperoxaluria Type 1, 2 or 3
NCT03819647	PHASE2	COMPLETED	Evaluation of the Efficacy of Stiripentol (Diacomit) as Monotherapy for the Treatment of Primary Hyperoxaluria
NCT03866928	PHASE1	COMPLETED	Pharmacokinetic Study of Stiripentol and Its Metabolites After Multiple Dose Oral Administration in Healthy Male Volunteers
NCT05735951	PHASE1	COMPLETED	Pharmacokinetic Parameters of Stiripentol in Renal Impaired Patients and Matching Controls With Normal Renal Function
NCT01533506	Not specified	NO_LONGER_AVAILABLE	Stiripentol in Dravet Syndrome
NCT01835314	Not specified	NO_LONGER_AVAILABLE	Compassionate Use of Stiripentol in Dravet Syndrome
NCT01983722	Not specified	APPROVED_FOR_MARKETING	Treatment Plan to Provide Expanded Access to Stiripentol for Patients With Dravet Syndrome
NCT02239276	Not specified	NO_LONGER_AVAILABLE	Expanded Access Use of Stiripentol in Dravet Syndrome or Sodium Channel Mutation Epileptic Encephalopathies
NCT03196466	Not specified	COMPLETED	Population Pharmacokinetics of Antiepileptic in Pediatrics
NCT06540378	Not specified	COMPLETED	Stiripentol for the Treatment of Refractory Status Epilepticus

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).