Leber hereditary optic neuropathy is a mitochondrial disorder characterised by bilateral sequential vision loss with limited treatment options. Idebenone, a benzoquinone, is the only drug currently approved for LHON by the European Medicines Agency. Its suggested mechanism of actions includes improving mitochondrial function and reducing retinal ganglion cell death [1]. This infographic summarises the design and results of the LEROS Trial. This trial was a multicentre, non-randomised, phase IV trial evaluating the use of idebenone versus a cohort of natural history comparators gathered from electronic databases. Participants taking idebenone were recruited from centres across Europe and the United States between 2016 and 2021, with the natural history cohort coming from two case report surveys conducted in Europe in 2013 to 2014. The idebenone cohort was significantly more likely to have clinically relevant benefit at all disease stages and at both 12 and 24 months than the natural history comparators. Clinically relevant benefit was defined as +5 letters on ETDRS charts if off-chart and +10 letters if on-chart [2].
References
Lyseng-Williamson KA. Idebenone: a review in Leber’s hereditary optic neuropathy. Drugs. 2016;76:805–13. https://doi.org/10.1007/s40265-016-0574-3.
Yu-Wai-Man P, Carelli V, Newman NJ, Silva MJ, Linden A, Van Stavern G, et al. Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: the LEROS nonrandomized controlled trial. Cell Rep Med. 2024;5:101437 https://doi.org/10.1016/j.xcrm.2024.101437.
Author information
Authors and Affiliations
Contributions
Design and drafting: SSM, CP and ML. Review: ML, FLA, SPM and GB. All authors agreed the final version.
Corresponding author
Ethics declarations
Competing interests
Susan Mollan reports consultancy fees (Invex Therapeutics); advisory board fees (Ocular therapeutix); speaker fees (Teva); Travel (Abbvie and European Alliance of Associations for Rheumatology); Receipt of equipment (Heidelberg Engineering). Susan Mollan is a section editor at Eye Journal. Authors declare no other financial relationships with any organisations that might have an interest in the submitted work; and no other relationships or activities that could appear to have influenced the submitted work.
Additional information
Publisher’s note Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.
Rights and permissions
Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/.
About this article
Cite this article
Sherratt-Mayhew, S., Page, C., Lowe, M. et al. Infographic: therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: the LEROS nonrandomized controlled trial. Eye (2025). https://doi.org/10.1038/s41433-025-03991-z
Received:
Revised:
Accepted:
Published:
Version of record:
DOI: https://doi.org/10.1038/s41433-025-03991-z
