Supplement
Idebenone is a lab-made relative of coenzyme Q10 that can pass electrons inside mitochondria, the parts of cells that make energy. People mostly look at it for mitochondrial conditions, and the evidence is moderate for Leber's hereditary optic neuropathy, where trials found visual acuity improved, especially in people whose eyes differed in vision at the start. Results in Friedreich's ataxia were mixed, and most other findings come from lab work.
Sources: PMID 19125241; PMID 35203712
- Updated
- How we grade
- 29 studies cited
- Best evidence
- Grade B
- Conditions studied
- 6
- Outcomes
- 12
- Graded outcomes
- 0165
Evidence by condition
- Strong
- Moderate
- Limited
- Very limited
General
| Grade | Outcome | Effect | Size | Studies | People | Studies list |
|---|---|---|---|---|---|---|
| Visual Acuity in LHON Multiple clinical trials demonstrated improvement in visual acuity in patients with Leber's hereditary optic neuropathy (LHON). Idebenone is approved in Europe (as Raxone) for this indication based on RCT evidence including the RHODOS trial, with benefits most pronounced in patients with discordant visual acuity at baseline. | Improves (the measure goes up) | Moderate effect | 6 studies | 250 people | ||
Studies that measured visual acuity in lhon
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| Mitochondrial Function Multiple in vitro and animal studies demonstrate idebenone modulates mitochondrial respiration, with distinct effects in astrocytes vs. neurons mediated by NQO1 activity (PMID:32350039). A case report showed dramatic improvement in mitochondrial cardiomyopathy (PMID:11286379). Idebenone can bypass complex I deficiencies by accepting electrons, a unique property vs. CoQ10. | Improves (the measure goes up) | Moderate effect | 7 studies | |||
Studies that measured mitochondrial function
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| Neurological Function in Friedreich's Ataxia Multiple clinical trials assessed idebenone for neurological outcomes in Friedreich's ataxia. Results were mixed and inconsistent, with low-dose and high-dose regimens studied. Cochrane reviews (2009, 2012) concluded that evidence for neurological benefit was not robust. | Improves (the measure goes up) | Small effect | 6 studies | 200 people | ||
Studies that measured neurological function in friedreich's ataxia
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| Noise-Induced Hearing Loss In guinea pig models, idebenone showed protective properties against noise-induced hearing loss (PMID:16738476). Coadministration with vitamin E provided enhanced antioxidant protection against acoustic trauma (PMID:18303566). No human studies available. | Changes; see studies | Moderate effect | 4 studies | |||
Studies that measured noise-induced hearing loss | ||||||
| Hereditary Myopathy Symptoms A pilot study evaluated idebenone in patients with hereditary myopathies, providing preliminary human evidence of potential benefit (PMID:35758956). Larger controlled trials are needed to confirm efficacy. | Improves (the measure goes down) | Small effect | 4 studies | 20 people | ||
Studies that measured hereditary myopathy symptoms | ||||||
| Pulmonary Fibrosis Preclinical study demonstrated idebenone has preventative and therapeutic effects on pulmonary fibrosis via preferential suppression of fibroblast activity (PMID:31754474). No human clinical trials for this indication. | Improves (the measure goes down) | Moderate effect | 3 studies | |||
| Grade | Outcome | Effect | Size | Studies | People | Studies list |
|---|---|---|---|---|---|---|
| Cardiac Function in Friedreich's Ataxia Several clinical trials in Friedreich's ataxia patients showed some improvement in cardiac hypertrophy markers. Two Cochrane systematic reviews (2009, 2012) evaluated antioxidant treatments including idebenone for Friedreich's ataxia, noting partial cardiac benefits but inconsistent neurological outcomes. | Improves (the measure goes up) | Small effect | 6 studies | 200 people | ||
Studies that measured cardiac function in friedreich's ataxia
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Antioxidant Status
- Oxidative Stress: improves
Aging
- Skin Aging and UV Protection: changes; see studies
| Grade | Outcome | Effect | Size | Studies | People | Studies list |
|---|---|---|---|---|---|---|
| Skin Aging and UV Protection In vitro and cosmetic studies demonstrate idebenone has potent antioxidant activity relevant to skin protection (PMID:18482011). Advanced delivery systems (solid lipid nanoparticles, nanostructured lipid carriers) improve skin permeation (PMID:22234598, PMID:22659127). Topical idebenone is used commercially in anti-aging formulations. | Changes; see studies | Small effect | 5 studies | |||
Studies that measured skin aging and uv protection
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Alzheimer’s Disease
- Cognitive Function in Dementia: improves
| Grade | Outcome | Effect | Size | Studies | People | Studies list |
|---|---|---|---|---|---|---|
| Cognitive Function in Dementia Limited human studies in dementia patients showed modest cognitive benefits. Preclinical studies demonstrated idebenone protects against amyloid beta toxicity in cortical neurons (PMID:32925607). Nootropic use in cognitively healthy individuals is not supported by human evidence. | Improves (the measure goes up) | Small effect | 4 studies | 100 people | ||
Studies that measured cognitive function in dementia | ||||||
Autoimmune Disease
- Autoimmune Inflammation in Lupus: improves
| Grade | Outcome | Effect | Size | Studies | People | Studies list |
|---|---|---|---|---|---|---|
| Autoimmune Inflammation in Lupus In a murine lupus model, idebenone improved mitochondrial metabolism and reduced inflammation (PMID:31566908). A review of CoQ10 analogs discussed immunomodulatory potential in systemic autoimmune diseases (PMID:33924642). No human clinical trial data for autoimmune indications. | Improves (the measure goes down) | Moderate effect | 3 studies | |||
Cataracts
- Cataract Risk: improves
| Grade | Outcome | Effect | Size | Studies | People | Studies list |
|---|---|---|---|---|---|---|
| Cataract Risk In a rat model of galactose-induced cataract, idebenone (alongside esculetin) ameliorated cataract formation (PMID:32301145). No human studies for cataract prevention. | Improves (the measure goes down) | Small effect | 3 studies | |||
Key findings
- Visual Acuity in LHONImproves (the measure goes up)
- Mitochondrial FunctionImproves (the measure goes up)
- Cardiac Function in Friedreich's AtaxiaImproves (the measure goes up)
Safety notes in the studies
- Preclinical studies demonstrated idebenone protects against amyloid beta toxicity in cortical neurons (PMID:32925607).
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- Grade B
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Studies cited
29 studies from PubMed
- Cytoprotective Effect of Idebenone through Modulation of the Intrinsic Mitochondrial Pathway of Apoptosis in Human Retinal Pigment Epithelial Cells Exposed to Oxidative Stress Induced by Hydrogen Peroxide.
- A pilot study of idebenone in the treatment of patients with hereditary myopathies.
- Leber hereditary optic neuropathy: new and emerging therapies.
- Developments in the Treatment of Leber Hereditary Optic Neuropathy.
- Therapeutic Potential and Immunomodulatory Role of Coenzyme Q(10) and Its Analogues in Systemic Autoimmune Diseases.
- Coenzyme Q10 effects in neurological diseases.
- Esculetin and idebenone ameliorate galactose-induced cataract in a rat model.
- Idebenone Has Distinct Effects on Mitochondrial Respiration in Cortical Astrocytes Compared to Cortical Neurons Due to Differential NQO1 Activity.
- Idebenone protects mitochondrial function against amyloid beta toxicity in primary cultured cortical neurons.
- Idebenone has preventative and therapeutic effects on pulmonary fibrosis via preferential suppression of fibroblast activity.
- Mitochondrial biogenesis and neural differentiation of human iPSC is modulated by idebenone in a developmental stage-dependent manner.
- Both idebenone and idebenol are localized near the lipid-water interface of the membrane and increase its fluidity.
- Pharmacological effect of a new idebenone formulation in a model of carrageenan-induced inflammatory pain.
- Coenzyme Q10 as a therapy for mitochondrial disease.
- Review of clinical trials for mitochondrial disorders: 1997-2012.
- Antioxidants and other pharmacological treatments for Friedreich ataxia.
- Nanostructured lipid carriers improve skin permeation and chemical stability of idebenone.
- Idebenone-loaded solid lipid nanoparticles for drug delivery to the skin: in vitro evaluation.
- Idebenone and resveratrol extend lifespan and improve motor function of HtrA2 knockout mice.
- Pharmacokinetic evaluation of idebenone.
- Pharmacokinetics and metabolism of idebenone in healthy male subjects.
- Low-dose idebenone treatment in Friedreich's ataxia with and without cardiac hypertrophy.
- Antioxidants and other pharmacological treatments for Friedreich ataxia.
- Antioxidant protection against acoustic trauma by coadministration of idebenone and vitamin E.
- Idebenone in Friedreich's ataxia.
- Determination of the antioxidative capacity of an antioxidant complex and idebenone: an in vitro rapid and sensitive method.
- Protective properties of idebenone in noise-induced hearing loss in the guinea pig.
- Dramatic improvement in mitochondrial cardiomyopathy following treatment with idebenone.
- Antioxidant properties of 2,3-dimethoxy-5-methyl-6-(10-hydroxydecyl)-1,4-benzoquinone (idebenone).