NICE Approves Idebenone for LHON in England

The United Kingdom’s National Institute for Health and Care Excellence (NICE) has given the green light for idebenone (Raxone, Chiesi) as a treatment option for patients aged 12 years and older with Leber hereditary optic neuropathy (LHON). This decision comes in the form of final draft guidance, with NICE expected to publish its final guidance in September. The National Health Service (NHS) will make the therapy available within three months following the publication date.

Understanding LHON and Its Impact

LHON is a rare inherited mitochondrial disease that often begins with painless blurring of central vision, either in one or both eyes. The condition progresses rapidly, and many patients, often children or young adults in their 20s and early 30s, lose their vision completely within months.

According to NICE, young men and boys are more frequently diagnosed with LHON and face a higher risk of severe vision loss. Approximately 250 people in England could be eligible for treatment with idebenone.

Clinical Evidence Supporting Idebenone

Long-term clinical trials have shown that idebenone can have reparative or regenerative effects on cells in the retina and optic nerve. By reactivating these cells, the therapy can improve or restore vision and slow or prevent further vision deterioration in LHON patients.

Idebenone, taken orally in tablet form, is the first licensed pharmaceutical to directly address the underlying causes of LHON.

Expert Perspectives on the Approval

Professor Patrick Yu-Wai-Man, a NICE committee member and Professor of Ophthalmology at the University of Cambridge, welcomed the decision:

“LHON causes devastating visual loss, and it is a life-changing diagnosis for the affected individual and their family. This new medicine 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.”

Helen Knight, Director of Medicines Evaluation at NICE, described LHON as a “debilitating” condition that robs patients of independence:

“The sudden change in sight can make daily activities such as reading, travelling, and recognizing faces very difficult or impossible. The evidence shows this treatment could improve people's eyesight and help their quality of life by allowing them to live independently for longer.”