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Idebenone: A Review in Leber's Hereditary Optic Neuropathy.
Drugs 2016; 76(7):805-13D

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.

Authors+Show Affiliations

Springer, Private Bag 65901, Mairangi Bay, 0754, Auckland, New Zealand. demail@springer.com.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

27071925

Citation

Lyseng-Williamson, Katherine A.. "Idebenone: a Review in Leber's Hereditary Optic Neuropathy." Drugs, vol. 76, no. 7, 2016, pp. 805-13.
Lyseng-Williamson KA. Idebenone: A Review in Leber's Hereditary Optic Neuropathy. Drugs. 2016;76(7):805-13.
Lyseng-Williamson, K. A. (2016). Idebenone: A Review in Leber's Hereditary Optic Neuropathy. Drugs, 76(7), pp. 805-13. doi:10.1007/s40265-016-0574-3.
Lyseng-Williamson KA. Idebenone: a Review in Leber's Hereditary Optic Neuropathy. Drugs. 2016;76(7):805-13. PubMed PMID: 27071925.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Idebenone: A Review in Leber's Hereditary Optic Neuropathy. A1 - Lyseng-Williamson,Katherine A, PY - 2016/4/14/entrez PY - 2016/4/14/pubmed PY - 2017/3/9/medline SP - 805 EP - 13 JF - Drugs JO - Drugs VL - 76 IS - 7 N2 - 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. SN - 1179-1950 UR - https://www.unboundmedicine.com/medline/citation/27071925/Idebenone:_A_Review_in_Leber's_Hereditary_Optic_Neuropathy_ L2 - https://dx.doi.org/10.1007/s40265-016-0574-3 DB - PRIME DP - Unbound Medicine ER -