NICE recommends treatment for rare genetic eye disorder
Ella Day | August 11, 2025 | News story | Medical Communications | Chiesi, Leber Hereditary Optic Neuropathy, NHS England, National Institute for Health and Care Excellence, Opthalmology, Rare Diseases
The National Institute for Health and Care Excellence (NICE) has issued final draft guidance recommending Cheisi’s idebenone (Raxone) within its marketing authorisation for treating visual impairment in people aged 12 years and over with Leber hereditary optic neuropathy (LHON).
LHON is an inherited mitochondrial disorder that affects around one in 31,000 people in England and can cause rapid, severe and often permanent loss of central vision. NICE estimates that up to 471 people could be eligible for idebenone treatment once final guidance is published.
Previously, the therapy was available on the NHS in Scotland, Wales and Northern Ireland, but not in England. This decision brings provision across the UK into alignment.
Patrick Yu-Wai-Man, professor of ophthalmology at the University of Cambridge, UK, said: “This decision will come as a great relief to the LHON community and brings hope to those who have experienced significant visual loss from this mitochondrial genetic disorder.”
LHON most often affects men aged between 15 and 35 years, with onset in one eye followed by the other within weeks or months. In around a quarter of cases, both eyes are affected almost simultaneously.
Idebenone is the first and only licensed treatment for vision impairment in LHON and is taken orally. Clinical and real-world studies infer that it is generally well tolerated.
David Garzón, senior director, rare diseases at Chiesi UK and Ireland said: “We are proud that NICE has recognised the significant unmet need for people living with this rare and debilitating disease.”
Ella Day
11/8/25
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