Idebenone and its uses

Idebenone known and sold under the trade names Catena, Raxone, Sovrima, and others) is a drug originally developed by Takeda Pharmaceutical Company to treat Alzheimer's disease and other cognitive deficits.This has had limited success Santhera Pharmaceuticals has begun studying it for the treatment of neuromuscular diseases.In 2010, early clinical trials for the treatment of Friedreich's ataxia and Duchenne muscular dystrophy were completed.As of December 2013, the drug was not approved for these indications in North America or Europe.It was approved by the European Medicines Agency (EMA) for the treatment of Leber's hereditary optic neuropathy (LHON) and was designated an orphan drug in 2007.Chemically, idebenone is a quinone organic compound.It is also marketed commercially as a synthetic analog of coenzyme Q10 (CoQ10).

Uses 

Indications that are or were approved in some territories.Idebenone improves learning and memory in mice.In humans, assessments of surrogate endpoints such as electroretinograms, auditory evoked potentials,and visual analogue scales have also demonstrated positive nootropic effects but larger studies with hard endpoints are lacking.Research on idebenone as a potential treatment for Alzheimer's disease has been inconsistent, but there may be a slight trend toward benefit.In May 1998, approval for this indication was withdrawn in Japan due to lack of proven efficacy.In some European countries, the drug may be used to treat individual patients in special cases.

Friedreich's ataxia (Sovrima):

Preliminary testing in humans has been done and idebenone has been found to be a safe drug for the treatment of Friedreich's ataxia (FA), with positive effects on cardiac hypertrophy and neurological function.The latter was only significantly improved in younger patients. In a different experiment, tested on eight patients for one year, idebenone reduced the rate at which heart function deteriorated but did not prevent the progression of ataxia.The drug was approved for FA in Canada in 2008, subject to conditions including demonstration of efficacy in further clinical trials.However, on February 27, 2013, Health Canada announced that idebenone would be voluntarily recalled by its Canadian manufacturer, Santhera Pharmaceuticals, on April 30, 2013, because the drug was not in further clinical trials showed curative effect.In 2008, the European Medicines Agency (EMA) refused marketing authorization for this indication.As of 2013, the drug is not approved for use in FA in Europe[14] and the US as there are no approved treatments.

Leber's hereditary optic neuropathy (Raxone):

Leber hereditary optic neuropathy (LHON) is a mitochondrial hereditary (mother of all offspring) degeneration of retinal ganglion cells (RGCs) and their axons, resulting in acute or subacute loss of central vision; this primarily affects young adults male.Santhera completed the Phase III clinical trial for this indication in Europe and achieved positive results and submitted a marketing application for the drug to the European regulatory agency in July 2011.It was approved by the EMA for this indication and designated as an orphan drug in 2007.

Indications being explored:

Duchenne muscular dystrophy (Catena).After experiments in mice and preliminary studies in humans, idebenone entered Phase II clinical trials in 2005 and Phase III clinical trials in 2009.

Other neuromuscular disorders:

Phase I and II clinical trials for the treatment of MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) were conducted.A phase I/II trial in primary progressive multiple sclerosis concluded that idebenone does not inhibit disability progression.As of 2022, a Phase III clinical trial for the treatment of Parkinson's disease is underway.

Lifestyle:

Idebenone is said to have similar properties to CoQ10 in terms of antioxidant properties and has therefore been used for anti-aging based on the free radical theory. Clinical evidence for this use is lacking.It has been used topically to treat wrinkles.