LHON is a rare maternally inherited progressive eye disease that causes irreversible loss of visual acuity, with approximately 80% of patients becoming blind within a year of symptom onset. Early diagnosis is the key to optimising prognosis.
Mutations associated with LHON cause leakage of electrons from retinal ganglion cells, impairing transmission of optical signals and generating reactive oxygen species that damage and destroy the cells.
Idebenone is an antioxidant and has the capacity to reactivate inactive retinal ganglion cells if treatment is initiated early enough, when the cells are still viable.
Post hoc analysis of the RHODOS clinical trial, which enrolled 85 patients with LHON duration of up to 5 years, showed that 30% of patients treated with idebenone experienced clinically relevant recovery of visual acuity after 6 months, compared with 10% of patients receiving placebo.