The first ever treatment for a mitochondrial condition has been approved for use on the NHS in England in a major breakthrough in the fight against the disease. The National Institute for Health and Care Excellence (NICE) has announced the approval of Idebenone (Raxone) for the treatment of Leber’s Hereditary Optic Neuropathy (LHON) a rare inherited disease causing sudden vision loss, in patients aged 12 and over. This advancement marks a pivotal moment for the mitochondrial community, offering new hope for individuals affected by this rare, vision-threatening condition.
Although Idebenone has been available for limited use in Scotland and Wales, this is the first time patients in England will have access to the therapy through the NHS, ending years of geographical inequality and potentially improving quality of life for those affected.
Patrick Yu-Wai-Man, Professor of Ophthalmology and Honorary Consultant Neuro-ophthalmologist at the University of Cambridge, Moorfields Eye Hospital and the UCL Institute of Ophthalmology, was the principal investigator for the LEROS study. The study, which was supported by the National Institute for Health and Care Research (NIHR) was crucial in convincing NICE that idebenone should be made available to patients with visual loss from LHON.