The UK National Institute for Health and Care Excellence (NICE) has issued final draft guidance recommending Chiesi’s Raxone (idebenone) for treating visual impairment in people aged 12 years and over with Leber Hereditary Optic Neuropathy (LHON).

The rare inherited mitochondrial disorder causes sudden, severe vision loss and can lead to permanent blindness. LHON affects around 1 in 31,000 people in England, with 20–25 new cases diagnosed annually.

The condition typically affects young men between 15 and 35 years old, who are four to five times more likely to develop symptoms than women. In roughly a quarter of cases, vision loss occurs in both eyes within a short period, resulting in long-term disability.

Clinical Data

NICE’s recommendation was primarily based on the results of the RHODOS trial (NCT00747487), which randomly assigned 85 patients aged 14 or older to receive 900 mg/day of Raxone or a placebo for 24 weeks.

At the 6-month mark, Raxone showed meaningful clinical improvements in visual acuity, particularly in patients with differing vision between their eyes at baseline. Visual recovery was observed in 30% of treated patients compared to 10% in the placebo group, with some improvements evident as early as 1 month.

A follow-up observational study, RHODOS-OFU (NCT01421381), indicated that these improvements were largely sustained even after discontinuing Raxone.

Raxone is an oral therapy and the first licensed treatment for visual impairment associated with LHON. Before its approval, management options were limited to best supportive care. In trials and real-world use, Raxone was generally well tolerated, with the most common side effects being mild to moderate diarrhea, nasopharyngitis, cough, and back pain.

Expanded Access Across England

Announced on 7 August, the decision provides patients in England with their first NHS-funded treatment for LHON. It also brings England in line with Scotland, where Raxone has been available since 2017, and Wales and Northern Ireland, where it was introduced in 2021. NICE estimates that up to 471 people in England may be eligible for treatment.

According to patient advocacy groups, LHON affects not only vision but also independence, education, employment, and mental health. Many individuals require assistive technologies, caregiver support, and social services. While idebenone is not a cure, clinical studies suggest it can improve visual acuity in some patients, potentially increasing their ability to live independently.
Clinical experts note that the availability of Raxone represents an important addition to the limited treatment options for LHON. Advocacy groups, including The Lily Foundation and the LHON Society, were engaged in the NICE submission process and described the recommendation as a step towards improving the quality of life for patients.

David Garzón, Senior Director, Rare Diseases at Chiesi UK and Ireland, said: “We are proud that NICE has recognized the significant unmet need for people living with this rare and debilitating disease.”

“It has been a long journey – it is very challenging to assess rare disease therapies via the NICE standard technology appraisal route and, while we understand the uncertainties raised within the process, we must collectively acknowledge that this is the reality of rare diseases and find a way to bring innovation to patients faster.”