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Raxone, has been approved in final draft guidance as a treatment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.
Helen Knight, Director of Medicines Evaluation at NICE,
said: "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. The evidence shows this
treatment could improve people's eyesight and help their quality
of life by allowing them to live independently for longer."
Professor Patrick Yu-Wai-Man, NICE committee member and Professor
of Ophthalmology at the University of Cambridge,
commented: “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.
Read the final draft guidance for idebenone for treating visual
impairment in Leber's hereditary optic neuropathy in people 12
years and over.
