LHON Eye Disease Treatment Recommended by NICE
Hundreds of people in England living with inherited vision loss could benefit from a newly recommended eye disease treatment. NICE has approved idebenone (Raxone) for use on the NHS to treat Leber’s hereditary optic neuropathy.
Introduction
NICE has released final draft guidance recommending the first LHON eye disease treatment, idebenone (Raxone), for use on the NHS. This is the first time an eye disease treatment has been approved for NHS use in managing this rare condition.
A New Treatment Option for LHON
Leber’s hereditary optic neuropathy is a rare mitochondrial condition that causes sudden, painless loss of central vision. It most commonly affects young males and can lead to permanent blindness. Until now, there have been no approved treatments available on the NHS.
Raxone will be available to patients aged 12 and over who have LHON caused by specific mitochondrial DNA mutations.
How the LHON Eye Disease Treatment Works
The condition is caused by a mutation that limits energy production in the cells of the eye. Idebenone works by helping restore energy production, which may allow damaged cells to function again. Clinical trials show the drug can slow vision loss and, in some cases, improve sight.
Who Will Benefit from LHON Eye Disease Treatment
NICE’s recommendation means hundreds of eligible patients across England will now have access to the first eye disease treatment for LHON. This decision brings hope for people living with inherited vision loss and may reduce long-term visual disability.
Conclusion
NICE’s final draft guidance recommends idebenone (Raxone) as the first NHS-funded eye disease treatment for Leber’s hereditary optic neuropathy. This milestone offers patients an opportunity to manage a previously untreatable condition and improve their quality of life.
Citation:
National Institute for Health and Care Excellence. (2025, 7 August). Hundreds with rare inherited eye disease to benefit after NICE recommends treatment. NICE
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