Clinical experience with Idebenone (Raxone®) in the treatment of patients with Leber's Hereditary Optic Neuropathy (LHON)

Abstract

PurposeLHON is an orphan mitochondrial disorder affecting the retinal ganglion cells leading to permanent blindness from which recovery is rare. An increasing body of evidence indicates that idebenone has therapeutic potential for the treatment of LHON. Data from a randomized placebo‐controlled study (RHODOS), from a number of case reports and retrospective cohort studies demonstrate that patients with established vision loss may benefit from idebenone treatment and recover visual acuity (VA).This study reports VA outcomes for patients with recent onset of vision loss who received idebenone treatment under an ongoing global Expanded Access Program (EAP) where currently 82 patients are enrolled and have been treated for an average of 15 months. The outcomes will be compared with findings of the RHODOS study and a Case Record Survey collecting VA data from untreated patients.MethodsAnalyses are performed to assess recovery from the VA nadir. Clinically relevant recovery was defined as (i) improvement from nadir by at least 10 letters on the ETDRS chart or (ii) improvement from “off‐chart” at nadir to being able to read at least 5 letters on‐chart. Furthermore, the prevention of vision loss for patients with residual vision below 1.0 logMAR (20/200) at start of therapy was analysed.ResultsA high proportion of patients (about 50% at submission of the abstract) treated with idebenone under this global EAP experienced a clinically meaningful recovery of vision. In addition, in patients with residual vision below 1.0 logMAR at start of therapy, loss of VA to above this level could be prevented in a large number of patients (about 60% at submission of the abstract).ConclusionsThe therapeutic potential of idebenone in the treatment of LHON is further demonstrated by the clinical experience in a large cohort of patients under a global EAP.