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Idebenone

Generic Name
Idebenone
Brand Names
Raxone
Drug Type
Small Molecule
Chemical Formula
C19H30O5
CAS Number
58186-27-9
Unique Ingredient Identifier
HB6PN45W4J

Overview

Idebenone is a synthetic analogue of ubiquinone (also known as Coenzyme Q10), a vital cell antioxidant and essential component of the Electron Transport Chain (ETC). It has been proposed that by interacting with the ETC, idebenone increases ATP production required for mitochondrial function, reduces free radicals, inhibits lipid peroxidation, and consequently protects the lipid membrane and mitochondria from oxidative damage . More specifically, idebenone is thought to transfer electrons directly to complex III of the mitochondrial ETC, thereby circumventing complex I and restoring cellular energy (ATP) generation . Due to its ability to reduce oxidative damage and improve ATP production, idebenone was originally investigated for its potential use in Alzheimer's Disease and other cognitivie disorders . Lack of improvement in cognitive function halted its production for these conditions, however it continues to be investigated for use in other conditions associated with mitochondrial damage. Idebenone is currently only indicated for use by the European Medicines Agency (EMA) for the treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON). LHON is a mitochondrially inherited degeneration of retinal ganglion cells, resulting in acute central vision loss. Due to its biochemical mode of action, it's thought that idebenone may re-activate viable-but-inactive retinal ganglion cells (RGCs) in LHON patients . It is not currently approved for use by either the Food and Drug Administration (USA) or Health Canada.

Indication

Idebenone is indicated for use by the European Medicines Agency (EMA) for the treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON). It is not currently approved for use by either the Food and Drug Administration (USA) or Health Canada .

Associated Conditions

  • Alzheimer's Disease (AD)
  • Leber’s hereditary optic neuropathy

Research Report

Published: Aug 6, 2025

Idebenone (DB09081): A Comprehensive Monograph on its Pharmacology, Clinical Trajectory, and Therapeutic Potential

Executive Summary

Idebenone (CAS: 58186-27-9; DrugBank ID: DB09081) is a synthetic small molecule, structurally analogous to the endogenous antioxidant Coenzyme Q10 (CoQ10). Initially developed for Alzheimer's disease with limited success, its therapeutic journey is a compelling case study in pharmaceutical repurposing, driven by a mechanistic rationale centered on mitochondrial support and antioxidant activity. This report provides a comprehensive, evidence-based analysis of Idebenone, covering its physicochemical properties, multifaceted pharmacology, complex clinical development history, divergent global regulatory status, and future therapeutic potential.

Pharmacologically, Idebenone possesses a dual mechanism. It functions as a mitochondrial electron carrier, capable of bypassing a defective Complex I in the electron transport chain to restore cellular ATP production. This specific action is the cornerstone of its efficacy in its sole approved indication: the treatment of visual impairment in adolescent and adult patients with Leber's Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease characterized by Complex I deficiency. Concurrently, Idebenone is a potent antioxidant that inhibits lipid peroxidation and scavenges free radicals. Emerging research also points to a novel anti-inflammatory role through the inhibition of the NLRP3 inflammasome.

Continue reading the full research report

Clinical Trials

Title
Posted
Study ID
Phase
Status
Sponsor
2023/08/14
Phase 4
Recruiting
Xiangya Hospital of Central South University
2023/07/05
N/A
Completed
2022/06/09
Phase 4
UNKNOWN
Beijing Tiantan Hospital
2020/12/16
Phase 1
Active, not recruiting
2020/09/01
Phase 2
Recruiting
2020/05/08
N/A
NO_LONGER_AVAILABLE
2019/11/05
Phase 2
Withdrawn
Second Affiliated Hospital, School of Medicine, Zhejiang University
2019/08/28
Phase 1
Recruiting
Second Affiliated Hospital, School of Medicine, Zhejiang University
2018/07/27
Phase 3
Terminated
2018/02/15
N/A
NO_LONGER_AVAILABLE

FDA Drug Approvals

Approved Product
Manufacturer
NDC Code
Route
Strength
Effective Date
No FDA approvals found for this drug.

HSA Drug Approvals

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No HSA approvals found for this drug.

NMPA Drug Approvals

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Dosage Form
Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

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Registration No.
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Strength
Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product
ARTG ID
Sponsor
Registration Type
Status
Registration Date
No TGA approvals found for this drug.

Health Canada Drug Approvals

Approved Product
Company
DIN
Dosage Form
Strength
Market Date
CATENA
santhera pharmaceuticals (switzerland) limited
02314150
Tablet - Oral
150 MG
10/15/2008

CIMA AEMPS Drug Approvals

Approved Product
Company
Registration Number
Pharmaceutical Form
Prescription Type
Status
RAXONE 150 MG COMPRIMIDOS RECUBIERTOS CON PELICULA
1151020001
COMPRIMIDO RECUBIERTO CON PELÍCULA
Diagnóstico Hospitalario
Not Commercialized

Philippines FDA Drug Approvals

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Company
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Strength
Approval Date
No Philippines FDA approvals found for this drug.

Saudi SFDA Drug Approvals

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No Saudi SFDA approvals found for this drug.

Malaysia NPRA Drug Approvals

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Company
Registration Number
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Approval Date
No Malaysia NPRA approvals found for this drug.

UK EMC Drug Information

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MA Holder
MA Number
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Active Ingredient
Authorization Date
No UK EMC drug information found for this drug.

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