NHS Approves Breakthrough Eye Treatment

The NHS has finally approved its first-ever treatment for a rare genetic eye disease that has left hundreds of young men and boys facing permanent blindness.

Story Highlights

  • NICE recommends idebenone (Raxone) as first NHS treatment for Leber hereditary optic neuropathy
  • Up to 471 people in England with the rare genetic eye disease will gain access to licensed therapy
  • Treatment targets underlying mitochondrial dysfunction rather than just managing symptoms
  • NHS England must provide access within three months of final guidance publication in September 2025

Historic NHS Approval for Rare Genetic Blindness

The National Institute for Health and Care Excellence issued final draft guidance recommending idebenone for treating Leber hereditary optic neuropathy in patients aged 12 and older. This rare mitochondrial genetic disorder predominantly strikes young men and boys, causing rapid, painless central vision loss that often leads to permanent blindness. The approval represents the first time a licensed medicine targeting LHON’s underlying cause will be available through England’s healthcare system.

LHON affects approximately one in 31,000 people in England, with mutations in mitochondrial DNA impairing energy production in retinal ganglion cells. Until now, patients faced a devastating diagnosis with no licensed treatments, relying only on supportive care including nutritional supplements and lifestyle management. The disease’s impact extends beyond vision loss, significantly affecting independence, education, and employment prospects for young patients during critical developmental years.

Clinical Evidence Demonstrates Treatment Effectiveness

Multiple clinical trials including RHODOS, RHODOS-OFU, LEROS, and PAROS provided evidence supporting idebenone’s efficacy and safety profile. The synthetic benzoquinone works by restoring mitochondrial function in damaged retinal cells, addressing the disease’s root cause rather than merely treating symptoms. Clinical data shows 30 percent of patients experienced vision improvement compared to only 10 percent receiving placebo treatment, with benefits maintained even after treatment cessation.

Watch: Treating Leber hereditary optic neuropathy

Professor Patrick Yu-Wai-Man from the University of Cambridge and Moorfields Eye Hospital, a leading LHON authority, emphasized the decision’s significance. He stated that England now aligns with the rest of the United Kingdom in providing idebenone access through the NHS, bringing great relief to the LHON community. The treatment demonstrates generally mild side effects similar to placebo, making it well-tolerated among patients in clinical studies.

Rapid Implementation Timeline Promises Quick Access

NHS England must implement patient access within three months of NICE’s final guidance publication, expected in September 2025. This aggressive timeline ensures eligible patients can begin treatment by December 2025, following a comprehensive evaluation process that began with topic selection in January 2023. Chiesi Pharmaceuticals has agreed to a confidential NHS discount through a patient access scheme, facilitating cost-effective implementation across England’s healthcare system.

The approval establishes a crucial precedent for NHS funding of rare disease therapies targeting mitochondrial dysfunction. This breakthrough may stimulate additional research and development in mitochondrial and genetic eye diseases, potentially benefiting patients with similar conditions. The decision demonstrates the NHS’s commitment to rare disease innovation while balancing clinical effectiveness with economic considerations for taxpayers and healthcare sustainability.

Sources:

NICE recommends Raxone in Leber hereditary optic neuropathy – Chiesi UK Press Release
Hundreds with rare inherited eye disease to benefit after NICE recommends treatment – NICE Official Announcement
NICE announces UK approval of idebenone for treatment of Leber hereditary optic neuropathy – Ophthalmology Times Europe
NICE approves idebenone for LHON in the UK – Ophthalmology Times

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