Drug for rare disease that causes sudden vision loss available on NHS in England
The new NHS drug that can treat ‘devastating’ sight loss disease
- A new treatment called idebenone, sold as Raxone, is now available on the NHS in England for Leber Hereditary Optic Neuropathy (LHON), a debilitating genetic eye disease.
- LHON is a rare inherited condition that primarily affects the optic nerve, leading to rapid and often severe vision loss and blindness.
- The National Institute for Health and Care Excellence (NICE) approved idebenone, which evidence suggests can improve eyesight and quality of life by restoring energy production in affected eye cells.
- Approximately 250 patients over the age of 12 are expected to be eligible for the treatment, which has shown effectiveness in about half of those treated.
- This approval marks the first time NICE has sanctioned a treatment for any mitochondrial condition, offering significant hope to the estimated 2,000 people in England affected by LHON.