RAXONE (idebenone), ophthalmic medicinal product

Insufficient clinical benefit in Leber's hereditary optic neuropathy because of the lack of methodologically acceptable clinical data demonstrating the efficacy of the drug, despite a significant therapeutic need

• RAXONE has marketing authorisation in the treatment of visual impairment in adolescent and adult patients with Leber’s hereditary optic neuropathy (LHON).
• There is no demonstration of sufficient methodological quality of the efficacy of idebenone on visual acuity. 
• The tendency to spontaneous improvement of visual acuity is observed in the placebo group in the RHODOS study.
• The data that suggest efficacy of idebenone in the treatment of patients with LHON are the results of certain post-hoc analyses of efficacy data from the RHODOS study as well as data from the provision of the product.
• In the absence of demonstration of its efficacy, this medicinal product has no role in the therapeutic strategy.