MPs, experts and families affected by the rare neurodegenerative disease Friedreich's Ataxia (FA) called on the government this month to enable interim access to a drug for which UCLH and UCL led the UK clinical trials, and which has been approved by the medicines regulator.
FA progressively affects the nervous system, causing loss of balance and coordination, weakening of the heart and other complications. The condition significantly reduces life expectancy, with an average age at death of around 37 years. Around 1,100 people in the UK are living with FA, while more than 11,000 have a form of ataxia.
Prof Paola Giunti, who led the UK trials of Omaveloxolone (Omav) at UCLH and UCL, joined an event in Parliament highlighting delays and disparities in access to the drug. Omav is the first licensed treatment for Friedreich's Ataxia and the only treatment shown to slow progression of the disease.
The event, hosted by Jonathan Brash MP and Mary Glindon MP, also highlighted the need to reform how medicines for rare diseases are appraised in the UK.
In 2025, the Medicines and Healthcare products Regulatory Agency (MHRA) approved Omav for the treatment of Friedreich's Ataxia in people aged 16 and over. The drug was previously approved by regulators in the United States in 2022 and the European Union in 2024.
Despite MHRA approval, people living with FA in the UK cannot routinely access the treatment through the NHS.
At the event, Ataxia UK proposed an interim access pathway for Omav while discussions continue on routine NHS access to the drug.
Prof Giunti said: "FA is a progressive and debilitating condition. Delays in treatment have serious consequences. It is vital that interim access is provided for patients to slow further progression. A new early access pathway for rare diseases is now necessary to close the gap of inequalities with other countries."
Families shared their experiences directly with parliamentarians, who were invited to sign an open letter to the Secretary of State for Health and Social Care calling for interim access to Omav.
Read more on the UCL news website.
