England poised to roll out drug for rare eye disease LHON

Hundreds of adults and teenagers in England with rare eye disease Leber's hereditary optic neuropathy (LHON) will soon have access to a targeted treatment, after Chiesi's Raxone was cleared for NHS use.

Reimbursement authority NICE has recommended use of Raxone (idebenone) in final draft guidance published today, which means NHS trusts in England should start to provide the treatment to eligible patients within the next three months.

The guidance ends a disparity in access to Raxone in the UK, as the drug has been available to LHON patients in Scotland, Wales, and Northern Ireland for several years.

NICE has estimated that 250 people in England could be eligible for the treatment, which involves taking two tablets three times daily and has been backed for use in people aged 12 and over with LHON, which is more common in males between the ages of 15 and 35 and can lead to sudden and severe loss of central vision in both eyes.

"The effects of LHON reach deeply into the lives of patients, their families, caregivers, as well as individuals who carry the LHON genes. Patients often experience loss of independence, with everyday tasks often becoming exceptionally challenging or even unmanageable," said a spokesperson for the LHON Society.

They added that the society is "delighted that idebenone has been recommended for use," noting that this is "a critical step towards full access to idebenone for patients."

Standard care for LHON includes nutritional supplements, genetic counselling, and lifestyle management advice, such as avoiding smoking and alcohol, according to the guidance.

LHON is caused by mutations in the genes coding for mitochondrial DNA (mtDNA) that prevent cells in the eye from producing the energy needed to function normally, and Raxone – the first medicine authorised for use in treating the disease – works by restoring that ability.

Clinical evidence from the RHODOS, LEROS, and PAROS trials has shown that the treatment can improve vision and reduce the deterioration of people's eyesight.

"England is now in line with the rest of the United Kingdom with idebenone now available through the NHS," commented Professor Patrick Yu-Wai-Man, a consultant neuro-ophthalmologist at the University of Cambridge, Moorfields Eye Hospital, and the UCL Institute of Ophthalmology.

"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 added.

Another possible treatment for LHON could be on the horizon, namely GenSight Biologics' gene therapy Lumevoq (lenadogene nolparvovec), although, a European application for approval for that was withdrawn in 2023 and the company is still exploring how it can make it available to patients. The therapy was granted a promising innovative medicine (PIM) designation from the UK medicines regulator, the MHRA, in 2021.