NICE approves idebenone for treatment of Leber’s hereditary optic neuropathy

The National Institute for Health and Care Excellence has approved idebenone (marketed by Chiesi as Raxone) for the treatment of Leber’s hereditary optic neuropathy (LHON) in patients aged 12 and over.

Although idebenone has been available for limited use in Scotland and Wales, this is the first time that the therapy will be available to patients on the NHS in England.

Katie Waller, head of patient programmes at The Lily Project, described the approval as “a huge win for the mito community.”

“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,” she said.

Professor Patrick Yu-Wai-Man, NICE committee member and Professor of Ophthalmology at the University of Cambridge, highlighted that England is now in line with the rest of the UK when it comes to the availability of idebenoneon 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,” he said.