The National Institute for Health and Care Excellence (NICE) has recommended idebenone (Raxone, Chiesi) as the first licensed treatment for Leber’s hereditary optic neuropathy (LHON), offering new hope to patients in England living with this rare, inherited eye disease.
LHON is a mitochondrial disorder caused by gene mutations that impair energy production in retinal ganglion cells. It typically begins with painless central vision blurring in one eye, quickly progressing to both eyes and often leading to severe, permanent vision loss within months. The condition primarily affects young men and teenagers, with devastating consequences for independence, education, and employment.
Idebenone, a synthetic analogue of coenzyme Q10, works by restoring mitochondrial energy production, allowing dysfunctional retinal cells to recover function. Clinical trials have shown the therapy can both improve vision and slow deterioration in patients with LHON.
NICE estimates that around 250 patients in England could be eligible for the treatment, which involves taking two tablets three times daily. Until now, management options were limited to nutritional supplements, lifestyle advice, and counselling, with no approved therapies addressing the underlying disease mechanism.
“This treatment could improve people's eyesight and help their quality of life by allowing them to live independently for longer,” says Helen Knight, Director of Medicines Evaluation at NICE. Professor Patrick Yu-Wai-Man, a Professor of Ophthalmology at the University of Cambridge and NICE committee member, adds that the decision comes as “a great relief to the LHON community in this country, bringing hope to those who have experienced significant visual loss from this mitochondrial genetic disorder.”
