Hundreds with rare inherited eye disease to benefit after NICE recommends treatment
NICE recommends idebenone for Leber's hereditary optic neuropathy following clinical evidence of vision improvement.
Hundreds of adults and teenagers in England with a rare, inherited eye disease will benefit from a new treatment after NICE today recommended idebenone for visual impairment caused by Leber's hereditary optic neuropathy (LHON).
The drug, the first treatment for LHON recommended by NICE for use on the NHS in England, is made by Chiesi and marketed as Raxone, has been approved in final draft guidance as a treatment option for people aged 12 and over with LHON.
LHON is caused by a gene mutation that prevents cells in the eye from producing the energy needed to function normally. The condition typically begins with painless blurring of central vision in one or both eyes, often progressing rapidly to blindness in both eyes within months. It primarily affects young men and boys, with devastating consequences for independence, education and employment prospects.
Idebenone works by restoring cells' ability to produce energy, allowing inactive eye cells to function again and potentially improving eyesight. Clinical evidence from longer-term trials demonstrates the treatment can improve vision and reduce the deterioration of people’s eyesight.
An estimated 250 people in England could be eligible for the treatment, which involves taking two tablets three times daily. Currently, standard care consists only of nutritional supplements, counselling and lifestyle management, with no licensed medicines available to address the underlying causes of LHON.
This is a debilitating condition that has a hugely significant impact on people, robbing them of their independence and quality of life. The sudden change in sight can make daily activities such as reading, travelling and recognising faces very difficult or impossible.
Helen continued: "The evidence shows this treatment could improve people's eyesight and help their quality of life by allowing them to live independently for longer."
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.
The treatment will be available through a confidential commercial arrangement with a patient access scheme that provides the NHS with a discount. NHS England will provide the treatment within three months of NICE publishing final guidance.