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Idebenone: A Review in Leber’s Hereditary Optic Neuropathy

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Abstract

Idebenone (Raxone®), a short-chain benzoquinone, is the only disease-specific drug approved to treat visual impairment in adolescents and adults with Leber’s hereditary optic neuropathy (LHON), a rare genetic mitochondrial disease that causes rapid and progressive bilateral vision loss. The mechanism of action of idebenone involves its antioxidant properties and ability to act as a mitochondrial electron carrier. Idebenone overcomes mitochondrial complex I respiratory chain deficiency in patients with LHON by transferring electrons directly to mitochondrial complex III (by-passing complex I), thereby restoring cellular energy (ATP) production and re-activating inactive-but-viable retinal ganglion cells, which ultimately prevents further vision loss and promotes vision recovery. The approval of idebenone in the treatment of LHON was based on the overall data from a randomized clinical trial, a follow-up study and real-world data. Taken together, these studies provide convincing evidence that oral idebenone 900 mg/day for 24 weeks has persistent beneficial effects in preventing further vision impairment and promoting vision recovery in patients with LHON relative to the natural course of the disease. Therefore, idebenone is a valuable agent to treat visual impairment in adolescents and adults with LHON.

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Acknowledgments

During the peer review process, the manufacturer of idebenone was also offered an opportunity to review this article. Changes resulting from comments received were made on the basis of scientific and editorial merit.

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  1. Springer, Private Bag 65901, Mairangi Bay, 0754, Auckland, New Zealand

    Katherine A. Lyseng-Williamson

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  1. Katherine A. Lyseng-Williamson
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Correspondence to Katherine A. Lyseng-Williamson.

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The preparation of this review was not supported by any external funding.

Conflicts of interest

Katherine A. Lyseng-Williamson is a salaried employee of Adis/Springer, is responsible for the article content and declares no relevant conflicts of interest.

Additional information

The manuscript was reviewed by: S. W. Cheng, Department of Paediatrics and Adolescent Medicine, Caritas Medical Centre, Hong Kong; P. G. Griffiths, Ophthalmology Services, St Bernard’s Hospital, Gilbraltar.

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Lyseng-Williamson, K.A. Idebenone: A Review in Leber’s Hereditary Optic Neuropathy. Drugs 76, 805–813 (2016). https://doi.org/10.1007/s40265-016-0574-3

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