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
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.
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. |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
---|---|---|---|
Authorised | 9/8/2015 | ||
Authorised | 9/8/2015 | ||
Authorised | 9/8/2015 | ||
Authorised | 9/8/2015 | ||
Authorised | 9/8/2015 | ||
Authorised | 9/8/2015 | ||
Authorised | 9/8/2015 |
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
---|---|---|---|---|---|
No HSA approvals found for this drug. |
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
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No NMPA approvals found for this drug. |
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
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No PPB approvals found for this drug. |
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
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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
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
---|---|---|---|---|---|
No Philippines FDA approvals found for this drug. |
Saudi SFDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
---|---|---|---|---|---|
No Saudi SFDA approvals found for this drug. |
Malaysia NPRA Drug Approvals
Approved Product | Company | Registration Number | Dosage Form | Strength | Approval Date |
---|---|---|---|---|---|
No Malaysia NPRA approvals found for this drug. |
UK EMC Drug Information
Medicine Name | MA Holder | MA Number | Pharmaceutical Form | Active Ingredient | Authorization Date |
---|---|---|---|---|---|
No UK EMC drug information found for this drug. |
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