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.