NICE Approves First NHS Treatment for Leber Hereditary Optic Neuropathy (LHON)

The UK-based National Institute for Health and Care Excellence (NICE) has approved idebenone (Raxone; Chiesi Pharmaceuticals) for the treatment of Leber Hereditary Optic Neuropathy (LHON) in patients aged 12 years and older. This marks the first time NICE has endorsed a therapy for any mitochondrial condition, according to Moorfields Eye Hospital NHS Foundation Trust.

LHON is a rare mitochondrial disorder that causes the rapid, painless loss of central vision in both eyes—often progressing to legal blindness within weeks. It is one of the most common mitochondrial diseases, affecting the optic nerve cells that transmit visual information to the brain. The condition disproportionately affects men between 15 and 35 years old, with an estimated 2,500 individuals affected in the UK.

Idebenone is an oral antioxidant therapy taken three times a day. By supporting mitochondrial function, it boosts cellular energy production in the optic nerve, helping to reduce damage and preserve vision. Clinical evidence shows that approximately half of patients experience measurable improvements in central vision—making it easier to recognise faces, navigate daily activities, and maintain independence, according to Moorfields Eye Hospital.

While idebenone has already been available in Wales and Scotland, NICE’s decision ensures that eligible patients in England can now access the treatment without restriction on the NHS. The NICE approval is supported by clinical studies led by NIHR Moorfields Biomedical Research Centre and long-term genetic research funded in part by Moorfields Eye Charity. Over the past decade, Professor Yu-Wai-Man and colleagues have advanced understanding of the genetic and molecular basis of inherited optic neuropathies, paving the way for this breakthrough therapy.