First ever mitochondrial disease treatment approve… - The Lily Foundation
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First ever mitochondrial disease treatment approved!

Research

7 August 2025

We’re celebrating another huge step forward for the mito community. For the first time ever, a treatment for one form of mitochondrial disease has been approved for use on the NHS in England. Idebenone is now available to help people living with LHON, marking a historic breakthrough and a huge leap forward in care and hope!

A bottle of white pills on its side with some spilling out onto a pink surface

We’re delighted to share the news that the National Institute for Health and Care Excellence (NICE) has just announced the approval of idebenone for the treatment of Leber’s hereditary optic neuropathy (LHON) in people aged 12 and over. The drug’s been available for limited use by patients in Scotland and Wales for several years, but until now wasn’t licenced in England.

Following the recent positive news about the early successes of mitochondrial donation, this is another landmark breakthrough for those affected by rare mitochondrial diseases. It brings an end to years of geographical inequality and means patients in England living with LHON disease will now have access to a drug that can improve quality of life and help preserve vision – a life-changing difference for those affected.

This success didn’t happen overnight. A big shoutout to our friends at the LHON Society who campaigned tirelessly to make this happen, placing the patient voice at the centre of all their interactions with NICE. We also want to acknowledge the patients who were involved, giving powerful personal testimonies about what access to the drug has meant to them. This is a true example of real-world evidence from those with lived experience helping to achieve a landmark decision.

Lily’s Head of Patient Programmes, Katie, said: “This is a huge win for the mito community and we’re proud to have been a key stakeholder throughout the process. While it isn’t a cure, this treatment offers real potential for patients to preserve or improve vision, giving the chance to regain independence, confidence and a better quality of life. And the fact that there is now a treatment available for mitochondrial disease brings hope for future drug development.”

What is Leber’s hereditary optic neuropathy?

LHON is one of the most common types of mitochondrial disease and is caused by mutations in mitochondrial DNA. Predominantly impacting young adult males, it generally affects the optic nerve – that’s the large nerve that leaves the back of each eye to carry visual information to the brain.

Patients with LHON disease usually first notice problems with their vision in their 20s or 30s, with initial symptoms being blurring of the central vision and loss of colour vision. Often this starts in just one eye but generally affects the other eye within weeks or months. Although not usually painful, it leads to severe vision loss and eventual blindness.

How does idebenone work?

Idebenone (also known by its brand name, Raxone) is a pill that works by supporting mitochondrial function in the retina and optic nerve. A recommended dose of two tablets three times a day is thought to help improve energy production in the cells of the optic nerve and retina, potentially slowing or reducing damage to vision.

Clinical studies have shown that idebenone can help stabilise vision in some patients and, in certain cases, may even lead to improvement when treatment is provided within five years of the onset of vision loss. It should be stressed that not everyone who is treated will benefit, and responses vary from person to person.

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. 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.”

In line with NICE’s recommendations, LHON patients in England who are 12 years and over are encouraged to speak with the healthcare professional responsible for their care to understand whether idebenone is the right treatment for them.

A win for the whole mito community

This is more than just a treatment approval – it’s a milestone. The first ever therapy for a mitochondrial disease now available across the whole of Great Britain, it shows that progress is possible, and that collaboration, persistence and patient power are key to driving genuine change.

At The Lily Foundation, we’ll keep on fighting until there’s an approved treatment for every type of mitochondrial disease. In the meantime, we’re here to advocate for patients and ensure they all have access to the care and support they deserve.

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