An Addenbrooke’s consultant who has dedicated over two decades researching an incurable hereditary eye condition has welcomed approval of a treatment by a health body.
Professor of ophthalmology and honorary consultant neuro-ophthalmologist, Patrick Yu-Wai-Man, says the drug will bring hope to patients with Leber Hereditary Optic Neuropathy (LHON).
LHON is a rare mitochondrial genetic disease that leads to rapidly worsening vision. The disease primarily affects the cells that make up the optic nerve, which carries visual information from the eyes to the brain, and there is currently no cure.
Early symptoms include blurred central vision and loss of colour vision. The typically painless disease progresses to severe vision loss and then legal blindness, within a few weeks. It disproportionately affects men and those aged between 15 and 35.
Now, the National Institute for Health and Care Excellence (NICE), an authoritative health body that assesses the clinical and cost-effectiveness of new treatments and technologies for the NHS, has approved Idebenone, also called Raxone, in those aged 12 and above.
Idebenone is an oral medicine taken at a dose of 300 milligrams three times a day. This drug works by supporting mitochondrial function in the cells of the optic nerve promoting their survival. The treatment is effective in about half of patients who take it.
Professor Yu-Wai-Man, also an honorary consultant neuro-ophthalmologist at Moorfields Eye Hospital, said:
LHON causes devastating visual loss, and it is a life-changing diagnosis for the affected individual and their family.
England is now in line with the rest of the United Kingdom with Idebenone now available through the NHS. 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.
Professor Patriuck Yu-Wai-Man
Helen Knight, director of medicines evaluation at NICE, said:
Leber Hereditary Optic Neuropathy is a debilitating condition that has a hugely significant impact on people, robbing them of their independence and quality of life.
The evidence shows Idebenone could improve people's eyesight and help their quality of life. We will continue to get the best care to people, fast, ensuring value for the taxpayer.
Helen Knight
Professor Yu-Wai-Man’s research over the past 25 years to understand the genetic and molecular basis of inherited optic neuropathies, including LHON, was supported by Moorfields Eye Charity and NIHR Cambridge and Moorfields Biomedical Research Centres.