Historical note and terminology
Coenzyme Q10 (ubiquinone), a vitamin, was discovered in 1957 by Frederick Crane at Purdue University in Indiana. Four years later, Peter Mitchell of the University of Edinburgh figured out how coenzyme Q10 produces energy at the cellular level and won the Nobel Prize for chemistry for this discovery in 1978. By the mid-1960s, Japanese researchers recognized that coenzyme Q10 concentrated in the myocardium. In the early 1980s, the first study of coenzyme Q10 was conducted in the treatment of cardiomyopathy. It is currently under investigation for the treatment of neurodegenerative disorders. Although not yet approved by the FDA, the product is available from health food stores. Idebenone, a synthetic analog of coenzyme Q10, has been tested in phase III clinical trials in the treatment of neurodegenerative diseases but has not been approved for use in any of these in the United States. In 2008, Health Canada approved Catena (a proprietary preparation of idebenone) for use in the symptomatic management of patients with Friedreich ataxia based on the promising nature of the clinical evidence. Market authorization was issued with a condition for need of a confirmatory study.
In 2013, the European Medicines Agency (EMA) denied an application originating in Switzerland for the approval of Raxone, a synthetic CoQ10 derivative for Leber hereditary optic neuropathy, because too few patients had been investigated during clinical development; however, there were no safety concerns. Later, this indication was approved by the EMA. In 2007, the FDA granted orphan drug designation to idebenone for the treatment of Duchenne muscular dystrophy, and phase 3 clinical trials were conducted, but approval for this indication was rejected in 2017. An application by the manufacturer for extension of CoQ10 use in patients with Duchenne muscular dystrophy was rejected by the EMA and confirmed after a review in January 2018.