Idebenone is recommended, within its marketing authorisation, as an option for treating visual impairment in Leber's hereditary optic neuropathy (LHON) in people 12 years and over.
Idebenone is recommended only if the company provides it according to the commercial arrangement.
NICE has produced tools and resources to support implementation of this guidance.
Why the committee made this recommendation
Standard care for LHON includes nutritional supplements, genetic counselling and lifestyle management advice. There are no licensed medicines for the underlying causes of LHON, so there is an unmet need for new treatments.
Evidence from a clinical trial directly comparing idebenone with standard care suggests that idebenone may be no better at improving vision. But this is uncertain because the trial was small number of people and of short duration. Longer-term trials show that idebenone improves vision but, in these trials, it was not compared with standard care. The results of an indirect comparison with standard care suggest that there may be a modest improvement in vision in the long term.
The cost-effectiveness estimates for idebenone are within the range that NICE normally considers an acceptable use of NHS resources. So, it is recommended.