Idebenone treatment in Leber's hereditary optic neuropathy: rationale and efficacy

Abstract

Introduction: Leber's hereditary optic neuropathy (LHON) is a rare mitochondrial retinopathy, caused by mutations in subunits of complex I of the respiratory chain. It is characterized by dysfunction and eventually loss of retinal ganglion cells which results in blindness. Clinical evidence suggests that idebenone, a short-chain quinone, can prevent vision loss and even restore vision in affected LHON patients.

Areas covered: This review covers the rationale for the use of idebenone in LHON based on its mode(s) of action, its pharmacodynamics, pharmacokinetics and its clinical efficacy.

Expert opinion: Idebenone was demonstrated as safe and efficacious in several open-label case reports, retrospective cohort studies and in a double-blind, placebo-controlled clinical study. Despite some remaining questions regarding the optimal treatment regimen, idebenone is currently the only therapeutic option for LHON patients and, thus, will become the benchmark treatment for other therapies to follow.

Keywords::

• complex I
• idebenone
• LHON
• mitochondria
• NQO1
• retina
• retinopathy

Declaration of interest

N Gueven currently acts as scientific consultant to Santhera Pharmaceuticals, which is developing idebenone for LHON. D Faldu has no conflicts of interest to declare.