This is a huge win for the mito community, and [the The Lily Foundation are] proud to have been a key stakeholder throughout the NICE submission process.
Katie Waller, Head of Patient Programmes at The Lily Foundation
While it isn’t a cure, Idebenone offers real potential to preserve or improve vision, giving people the chance to regain independence, confidence and a better quality of life. Plus the fact that there is now an NHS-approved treatment for mitochondrial disease brings hope for future drug development.
Mitochondrial disease occurs when the mitochondria, small structures inside our cells responsible for generating more than 90% of the body’s energy, do not function properly. LHON is one of the most common types of mitochondrial disease, primarily affecting the optic nerve and retina. Early symptoms include blurred central vision and loss of colour vision. While typically painless, the disease progresses to severe vision loss and eventual blindness.
Idebenone is an oral medication, taken three times a day, that works by supporting mitochondrial function in the retina and optic nerve. Potentially slowing or reducing damage to vision by helping to improve energy production in the cells of both the retina and optic nerve.
According to The Lily Foundation, every year, around one baby in 5000 is born with mitochondrial DNA mutations that can cause the disease. There is currently no cure. However, progress has been made in mitochondrial donation IVF, which prevents the transfer of the disease to offspring.