Idebenone

Summary

Idebenone (CHEMBL252556) is an approved small-molecule ferroptosis inhibitor (ATC N06BX13); indicated across 8 conditions including attention deficit-hyperactivity disorder and leber hereditary optic neuropathy.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: N06BX13
• Indications: 8 conditions
• Clinical trials: 27
• Chemistry: 338.4 Da · C19H30O5

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL252556
Name	Idebenone
Type	Small molecule
Max phase	4
FDA approved	no
PubChem CID	3686
ChEBI	CHEBI:31687
ATC	N06BX13
Molecular formula	C19H30O5
Molecular weight	338.4
InChIKey	JGPMMRGNQUBGND-UHFFFAOYSA-N

SMILES: CC1=C(C(=O)C(=C(C1=O)OC)OC)CCCCCCCCCCO

IUPAC name: 2-(10-hydroxydecyl)-5,6-dimethoxy-3-methylcyclohexa-2,5-diene-1,4-dione

ChEBI definition: A member of the class of 1,4-benzoquinones which is substituted by methoxy groups at positions 2 and 3, by a methyl group at positions 5, and by a 10-hydroxydecyl group at positions 6. Initially developed for the treatment of Alzheimer’s disease, benefits were modest; it was subsequently found to be of benefit for the symptomatic treatment of Friedreich’s ataxia.

Pharmacological roles (ChEBI): antioxidant, ferroptosis inhibitor.

Also known as: Hydroxydecyl ubiquinone, Idebenona, Idebenone, NSC-759228, Oristar hdu, Raxone, Sovrima, idebenone, SID26719872, SID29216196, IDEBENONE, SID144205417

Patent coverage: 3,188 distinct patent families (8,581 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: 20 (assay-derived). Sample: Tyrosyl-DNA phosphodiesterase 1, Microtubule-associated protein tau, Nuclear receptor ROR-gamma, Fructose-bisphosphate aldolase, Prelamin-A/C, Ferritin light chain, Vasopressin V2 receptor, 5-hydroxytryptamine receptor 2B, Amine oxidase [flavin-containing] A, Equilibrative nucleoside transporter 1.

Bioactivity

ChEMBL activities: 8 potent at pChembl ≥ 5 of 23 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
CYP2C19	6.1	IC50	800	nM	CHEMBL_ACT_7670992
CYP2D6	5.52	IC50	3000	nM	CHEMBL_ACT_7670996
A8B2U2	5.35	Potency	4456	nM	CHEMBL_ACT_4589147
CYP2C9	5.3	IC50	5000	nM	CHEMBL_ACT_7670994
PDE3A	5.19	AC50	6400	nM	CHEMBL_ACT_25191348
CYP3A4	5.16	IC50	7000	nM	CHEMBL_ACT_7671000
MAOA	5.07	AC50	8500	nM	CHEMBL_ACT_25160323
HTR2B	5.05	AC50	8990	nM	CHEMBL_ACT_25164270

Target pathways

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

Indications & clinical

Indications

8 indications (2 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
attention deficit-hyperactivity disorder	4	MONDO:0007743	EFO:0003888
Leber hereditary optic neuropathy	4	MONDO:0010788	MONDO:0010788
Duchenne muscular dystrophy	3	MONDO:0010679	MONDO:0010679
Friedreich ataxia	3	MONDO:0100339	MONDO:0100339
MELAS syndrome	2	MONDO:0010789	Orphanet:550
primary progressive multiple sclerosis	1	MONDO:0000451	EFO:0008520
metabolic dysfunction-associated steatotic liver disease	1	MONDO:0013209	EFO:0003095
Huntington disease	1	MONDO:0007739	MONDO:0007739

Clinical trials

Total trials: 27.

Phase distribution

Phase	Trials
PHASE3	8
PHASE2	6
PHASE4	3
PHASE1/PHASE2	3
PHASE1	3
Not specified	3
PHASE2/PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02774005	PHASE4	COMPLETED	Study to Assess the Efficacy and Safety of Raxone in LHON Patients
NCT03727295	PHASE4	UNKNOWN	Idebenone Treatment of Early Parkinson’s Diseasesymptoms
NCT05411978	PHASE4	UNKNOWN	Safety and Efficacy of Oral Idebenone for Preventive Treatment of Migraine in Adult Migraine Patients
NCT04151472	PHASE3	RECRUITING	Idebenone for the Preventive Treatment of Migraine
NCT00537680	PHASE3	COMPLETED	Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich’s Ataxia
NCT00697073	PHASE3	COMPLETED	Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich’s Ataxia Patients
NCT00905268	PHASE3	COMPLETED	A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich’s Ataxia (FRDA) Patients
NCT00993967	PHASE3	COMPLETED	Long-Term Safety and Tolerability of Idebenone in Friedreich’s Ataxia Patients (MICONOS Extension)
NCT01027884	PHASE3	COMPLETED	Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)
NCT01303406	PHASE3	COMPLETED	Patient Reported Outcomes in Friedreich’s Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI)
NCT01495715	PHASE3	WITHDRAWN	Study With Idebenone in Patients With Chronic Vision Loss Due to Leber’s Hereditary Optic Neuropathy (LHON)
NCT04152655	PHASE2/PHASE3	WITHDRAWN	A Study of Efficacy and Safety of Idebenone Vs. Placebo in Prodromal Parkinson Disease
NCT04534023	PHASE2	ACTIVE_NOT_RECRUITING	A Clinical Study of the Efficacy of Idebenone in the Treatment of iRBD Into Synucleinopathies
NCT00229632	PHASE2	COMPLETED	Idebenone to Treat Friedreich’s Ataxia
NCT00654784	PHASE2	COMPLETED	Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy
NCT00747487	PHASE2	COMPLETED	Study to Assess Efficacy,Safety and Tolerability of Idebenone in the Treatment of Leber’s Hereditary Optic Neuropathy
NCT00758225	PHASE2	COMPLETED	Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)
NCT00887562	PHASE2	COMPLETED	Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes
NCT00950248	PHASE1/PHASE2	COMPLETED	Clinical Trial of Idebenone in Primary Progressive Multiple Sclerosis (IPPoMS)
NCT01854359	PHASE1/PHASE2	COMPLETED	Idebenone for Primary Progressive Multiple Sclerosis
NCT04669158	PHASE1/PHASE2	COMPLETED	Study of Oral Idebenone to Treat Non-Alcoholic Steatohepatitis
NCT00015808	PHASE1	COMPLETED	Safety Study of Idebenone to Treat Friedreich’s Ataxia
NCT00078481	PHASE1	COMPLETED	Phase 1 Trial of Idebenone to Treat Patients With Friedreich’s Ataxia
NCT04071639	PHASE1	UNKNOWN	Symptomatic Therapy for Patients With Huntington’s Disease
NCT02771379	Not specified	COMPLETED	Post Authorisation Safety Study With Raxone in LHON Patients
NCT03433807	Not specified	NO_LONGER_AVAILABLE	Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)
NCT05931029	Not specified	COMPLETED	Study of the Therapeutic Effects of Naohuan Dan and Idebenone in Treating Mild Cognitive Impairment With Kidney Deficiency and Phlegm Stasis

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

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