Submitted by lac59 on Thu, 07/08/2025 - 15:27
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.
Professor Yu-Wai-Man, who runs specialist clinics for patients with inherited optic neuropathies in Cambridge and London and receives referrals from across the whole of the UK said: “LHON causes devastating visual loss and it is a life-changing diagnosis for the affected individual and their family. England is now in line with the rest of the United Kingdom with Idebenone now available through the NHS. This will come 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.”
Katie Waller, Head of Patient Programmes at The Lily Foundation, a major contributor to submission process and the UK’s leading charity dedicated to fighting mitochondrial disease said: “This is a huge win for the mito community, and we’re proud to have been a key stakeholder throughout the NICE submission process. While it isn’t a cure, Idebenone offers real potential to preserve or improve vision, giving people the chance to regain independence, confidence and a better quality of life. Plus the fact that there is now an NHS-approved treatment for mitochondrial disease brings hope for future drug development.”
The approval follows a wave of positive developments in mitochondrial medicine, including the recently announced early successes of mitochondrial donation IVF. It also reflects the power of patient advocacy, thanks in large part to the tireless campaigning by the LHON Society, who ensured patient voices were central throughout the NICE evaluation process.
Mitochondrial disease is caused by faults in the mitochondria – tiny structures within cells that produce over 90% of the body’s energy. Every year, around one baby in 5000 is born with mitochondrial DNA mutations that can cause the disease. Despite years of research, there is currently no cure.
LHON is one of the most common types of mitochondrial disease, primarily affecting the optic nerve and retina. Initial symptoms include blurring of the central vision and loss of colour vision, and although not usually painful, the disease leads to severe vision loss and eventual blindness.
Idebenone is a pill taken three times a day that works by supporting mitochondrial function in the retina and optic nerve. It’s thought to help improve energy production in the cells of the optic nerve and retina, potentially slowing or reducing the damage to vision.
To learn more about the LEROS study – view publication
This article was first published on the NIHR Cambridge BRC website here.
