Idebenone Reference

Reference summary

Idebenone is a short-chain synthetic analog of coenzyme Q10, designed to remain reduced under conditions of complex I dysfunction and to transfer electrons directly to complex III. The EU LHON approval is anchored in the RHODOS trial (Klopstock 2011, Brain, PMID 21788663) and supportive expanded-access data, which showed visual-acuity recovery in a subset of discordant LHON patients. The US Friedreich ataxia program (IONIA, MICONOS) did not establish efficacy. Smaller mitochondrial-disease cohorts, mitochondrial encephalomyopathies, and Duchenne muscular dystrophy (DELOS trial, Buyse 2015, Lancet, PMID 25907158) suggest possible benefit on respiratory function endpoints but did not produce a US approval. Cognitive-impairment literature from the 1990s in mild-to-moderate Alzheimer disease produced mixed results and is not the basis for modern marketing.

Regulatory and posture

Categories

Nootropic, Metabolic

Aliases

Catena, Raxone, CV-2619, Sovrima, Hydroxydecyl ubiquinone

Evidence posture

human - EU-approved for Leber's hereditary optic neuropathy at 900 mg per day. Not FDA-approved in the US. The Friedreich ataxia development program did not advance. US 'supplement-dose' idebenone (45-150 mg per day in cosmetic and supplement products) is far below trial doses and is not supported by the LHON evidence base.

Regulatory status

No FDA-approved idebenone drug label. Approved in the EU and several other markets as Raxone (Santhera) for Leber's hereditary optic neuropathy (LHON) since 2015. Marketed in Canada as Catena. In the US, idebenone is sold as a dietary supplement and as a cosmetic ingredient; no FDA-approved drug label exists, and the US Friedreich ataxia development program (Sovrima) did not reach FDA approval after the IONIA trial failed to meet its primary endpoint.

Content review status

research reference