The UK medicines regulator (MHRA) approves the first treatment for Friedreich’s ataxia (FA), Omaveloxolone (Skyclarys).
On 23 April 2025, the MHRA approved Omaveloxolone for the treatment of those aged 16 and over with FA in the UK.
In response to the announcement, Ataxia UK’s Chief Executive Sue Millman says,
‘This is a huge milestone for the FA community, and Ataxia UK is delighted that we are now one step closer to Omaveloxolone being made available to adults with FA in the UK. Ataxia UK will continue to do everything we can to ensure that people with FA in the UK access to the drug at the earliest possible opportunity.’
Prof Giunti, Head of the Ataxia UK-accredited Ataxia Centre in London says,
‘We at the London Ataxia Centre UCL/UCLH are very proud to have contributed to this milestone in Friedreich’s ataxia history with our preclinical and clinical data that were crucial to the success of the trial.’
Read the press release from the MHRA here. Read more about Omaveloxolone on our website here.
Disappointing news about NICE evaluation
As we have reported in the past, Omaveloxolone was under evaluation by NICE, the body that oversees reimbursement of medicines in England and Wales. Unfortunately, we have been told today that Biogen has taken the decision to withdraw from this evaluation following discussions with NICE. During their discussions NICE had indicated to Biogen that they would not proceed the evaluation to the committee stage, when the patient and clinical community would have had the opportunity to highlight the unmet need in FA and the potential of the treatment. Read the statement from Biogen to Ataxia UK here. Although this is really disappointing news we understand Biogen are committed to resubmitting to NICE. In Scotland the Scottish Medicine Consortium (SMC) will decide whether it should be made available on the NHS in Scotland using a similar process to that of NICE. Wales and Northern Ireland can also adopt NICE recommendations.
As soon as we have more information on timelines, we will provide an update.
Ataxia UK completely understands the delays in the evaluation of Omaveloxolone by NICE will be profoundly disappointing. We share this disappointment wholeheartedly and recognise how challenging it must be to receive such mixed news—the positive approval from the MHRA alongside these delays.
We want to reassure the community that we understand that Biogen is committed to resubmitting to NICE, and we are unwavering in our own dedication to supporting this process. We are doing everything in our power to advocate for timely progress, and we will provide updates as soon as we have more clarity on the timelines.
To strengthen our connection and communication with the community, we are also planning a webinar and Q&A session soon, where we can discuss concerns, share any updates, and try to provide answers to your questions.
