Fomepizole

Summary

Fomepizole (CHEMBL1308) is an approved small-molecule protective agent (ATC V03AB34); indicated across 1 condition including age-related macular degeneration.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: V03AB34
• Indications: 1 condition
• Clinical trials: 4
• Chemistry: 82.1 Da · C4H6N2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1308
Name	Fomepizole
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	3406
ChEBI	CHEBI:5141
ATC	V03AB34
Molecular formula	C4H6N2
Molecular weight	82.1
InChIKey	RIKMMFOAQPJVMX-UHFFFAOYSA-N

SMILES: CC1=CNN=C1

IUPAC name: 4-methyl-1H-pyrazole

ChEBI definition: A member of the class of pyrazoles that is 1H-pyrazole substituted by a methyl group at position 4.

Pharmacological roles (ChEBI): antidote, protective agent, EC 1.1.1.1 (alcohol dehydrogenase) inhibitor.

Also known as: Antizol, Fomepizol, Fomepizole, NSC-760365, SID90341211, FOMEPIZOLE, SID11111426, SID50111124, fomepizole

Parent form; salt/anhydrous children: CHEMBL1256285

Patent coverage: 2,734 distinct patent families (7,861 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

Broader ChEMBL bioactivity targets: 2 (assay-derived). Sample: Alcohol dehydrogenase, Voltage-gated inwardly rectifying potassium channel KCNH2.

Bioactivity

ChEMBL activities: 2 potent at pChembl ≥ 5 of 3 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
P00327	7.89	Kd	13	nM	CHEMBL_ACT_14705982
P00327	6.85	Ki	141.2	nM	CHEMBL_ACT_419785

Target pathways

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

Indications & clinical

Indications

1 indication (0 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
age-related macular degeneration	1	MONDO:0005150	EFO:0001365

Clinical trials

Total trials: 4.

Phase distribution

Phase	Trials
PHASE2	1
PHASE1	1
EARLY_PHASE1	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00661141	PHASE2	COMPLETED	Phase IIa Study of Fomepizole for Acetaldehyde Toxicity After Ethanol Exposure in Subjects With Altered Ethanol Metabolism
NCT00346853	PHASE1	COMPLETED	Phase 1 Pilot Study of 4-MP to Treat Stargardt Macular Dystrophy
NCT03878693	EARLY_PHASE1	COMPLETED	4-methylpyrazole and Acetaminophen Metabolism
NCT02415712	Not specified	COMPLETED	Specified Drug-use Survey of Fomepizole Intravenous Infusion (All-case Surveillance)

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).