On Friday 23rd May, Ataxia UK hosted a webinar and Q&A on access to omaveloxolone for adults with Friedreich’s ataxia (FA) in the UK. This webinar was presented in partnership with Realise Advocacy, who have expertise in drug appraisals.
This webinar covered the approval of SKYCLARYS (omaveloxolone) in the UK in April 2025, as well as the next steps following the disappointing news that Biogen has withdrawn from the NICE evaluation process of omaveloxolone. NICE is the body that evaluates the cost-effectiveness of and oversees reimbursement for medicines in England. Wales and Northern Ireland can also adopt NICE recommendations.
Realise Advocacy shared information about the Health Technology Assessment (HTA) for medicines in England (by NICE) and Scotland (by SMC), The standard HTA process in England, which is used in rare diseases such as FA, as well as treatments for more common diseases, usually takes around 18 months. In Scotland, the process is shorter.Biogen plan to submit to the Scottish Medicines Consortium (SMC) in June 2025. Ataxia UK will provide evidence as part of the submission by Biogen to the SMC. In Scotland there is also a process for patients to gain early access to the drug, as soon as the company submit to the SMC.
The NICE evaluation of omaveloxolone has been halted following evidence submission (which includes evidence submitted by Ataxia UK). This is because Biogen has withdrawn from the NICE process, as NICE were not prepared to take the submission to the next stage (ie: the Evaluation Committee meeting.
Dr Julie Greenfield, Head of Research at Ataxia UK, then went on to explain the work that Ataxia UK has done so far to support the NICE process for omaveloxolone. This includes supporting patient involvement throughout the process, submitting evidence for the scoping process, attending the NICE scoping meeting in 2024, and gathering and submitting evidence to support the NICE evaluation. This evidence included not only the impacts of FA on those affected and the need for a medicine to improve quality of life, but also collecting evidence from people taking the drug outside of the trial, mostly from other countries, including in France.
Julie summarised the next steps from Ataxia UK, including engaging with NICE to see what is needed to resume the NICE process and to have a chance to discuss the submission in the NICE Evaluation Committee meeting. Ataxia UK is also engaging with the FA community, the UK Ataxia Centre clinicians and neurologists, as well as Genetic Alliance UK, and other rare disease charities with experience of situations like this to see what can be learnt from them. Ataxia UK is also continuing to encourage Biogen to resubmit to NICE as soon as possible. We are also exploring if there are any potential routes for early access to omaveloxolone in the UK.
One of the key next steps Ataxia UK has taken to advocate for the FA community in this difficult situation is that we have written an open letter to the UK Secretary of State for Health and Social Care, Rt Hon West Streeting, asking for interim access to omaveloxolone for patients, and calling for changes to the NICE evaluation process which we feel are not appropriate for conditions like FA that are rare, but not ultra rare (for which there is a different evaluation process called Highly Specialised Technology Appraisal – HTA).
Sign the open letter here. We are also encouraging people to forward this letter to their MPs.
You can view the slides from the webinar here.
If you are interested in learning more about the UK drug approval process for omaveloxolone, watch our earlier webinars with Realise Advocacy here and here.
Following the Q&A portion of the webinar, Ataxia UK has put together a list of answers to frequently asked questions. Read the answers to these FAQs below:
FAQs – access to omaveloxolone for adults with FA in the UK:
1. What are the reasons behind Biogen’s withdrawal? Is it cost?
Biogen withdrew their submission because during their discussions NICE had indicated to Biogen that they would not proceed to the committee stage. Read the statement from Biogen to Ataxia UK here. .
Ataxia UK asked NICE for an explanation for not taking the submission to the Committee stage, meaning this was a termination. They told us the reason is confidential. However, they did explain why NICE would terminate an appraisal. The reasons are listed below:
- When NICE is not satisfied that the evidence submission is adequate for the committee to make a decision;
- If an evidence submission has not been received or withdrawn;
- When the company makes a submission that has no plausible potential to be cost-effective (that is, a significantly high ICER) and are unwilling to offer a patient access scheme or if commercial discussions with NHS England are not possible.
2. Did Biogen try to have omaveloxolone reviewed through the Highly Specialised Technology Appraisal route?
Yes. Ataxia UK attended the scoping meeting with Biogen and NICE, and this route was discussed. This route is for conditions rarer than 300 people in England. The number of adults with FA in England is more than this, so unfortunately NICE did not agree to use this evaluation route. Ataxia UK has written an open letter to the UK Secretary of State for Health and Social Care requesting interim access to omaveloxolone for adults with FA, and encouraging NICE to change the evaluation process for conditions that are rare but not ultra-rare. Sign the letter here. We are also encouraging people to forward this letter to their MPs.
3. Does Ataxia UK know when Biogen will resubmit to NICE?
Biogen have told Ataxia UK they are committed to re-submitting to NICE. They are exploring how to revise the omaveloxolone data package supplied as evidence through longer-term trial data and real-world evidence from other countries. They told us they will re-submit when they are confident that NICE would progress the submission to a committee meeting.
4. If Biogen does resubmit to NICE, will that process take another 18 months?
The NICE evaluation process normally takes 18 months. We do not know the exact timings for this case.
5. When will the drug be available in the UK?
Once a license has been given by the MHRA, the drug can be prescribed privately if there is a clinician willing to prescribe the drug. Once the drug is approved by NICE for reimbursement on the NHS, then the drug needs to be made available within 90 days of that approval.
6. Has Biogen submitted to the SMC in Scotland?
Biogen plans to submit to the SMC in June. We will provide an update as soon as it is submitted. From the point of the submission, there is a process for early access to the drug. This is called the Peer Approved Clinical System (PACS) Tier Two process. This allows clinicians to request the use of licensed medicines on a case-by-case basis, even if those medicines have not been routinely recommended by the Scottish Medicines Consortium (SMC). Ataxia UK is exploring this with clinicians and will try to support any people in Scotland who wish to pursue.
7. Where can I find case studies from people with FA who have taken omaveloxolone?
Data was presented at the International Congress for Ataxia Research (ICAR) in 2024 from clinicians in the US and France and the French patient organisation AFAF. Some information is in the public domain. The experiences of 68 people taking omaveloxolone in France was collected as part of a survey, and the results are available online here (currently in French only).(currently in French only).
There is also a recently published paper on the safety of omaveloxolone in 236 people with FA in the US who had been taking the drug for one year. Read it here.
8. Even if approved in the UK, we have been told that a person with FA at a more advanced stage (e.g., non-ambulant) may not get the drug as it was tested only in people in the early stages of FA. Does this mean that they cannot get the drug?
The trial was conducted in ambulant (walking) patients aged 16-40 with FA. The MHRA has approved the drug for all adults with FA regardless of age and severity. Ataxia UK were very pleased to hear this outcome.
However, we do not know if NICE will limit the availability of the drug on the NHS to only a subgroup of people with FA. During the initial scoping phase of the NICE process, NICE asked stakeholders whether the evaluation of omaveloxolone should be limited to a subgroup of adults with FA (eg: of a certain age). Ataxia UK argued strongly that the drug should be considered for all adults, and we were pleased that NICE agreed with this recommendation and decided to evaluate the drug for all adults.
Having said this, when NICE makes its decision on whether omaveloxolone is approved for reimbursement on the NHS, they could still limit it to a subgroup of patients. If this were to happen, with time, if the company are able to collect positive evidence of the effectiveness of the drug in different subgroups they may change their decision and make the drug available for all. Ataxia UK will continue to encourage Biogen and NICE to make the drug available to all adults.
9. Is the children’s trial of omaveloxolone taking place in the UK?
Yes, the trial is planned to take place through the three UK children’s ataxia centres in London, Sheffield and Oxford. Read more here.
10. Can people from a different country take part in the UK children’s trial?
This can happen, but this will need to be a conversation with the clinicians at the trial centres to see if taking part is feasible and the child will be able to complete all of the trial visits.
11. What is the cost of omaveloxolone if you buy it privately?
- The listed price is $370,000 per year. There may be neurologists who are able to offer the drug privately. Biogen have said that they will work as quickly as possible to bring stock to the UK for those who may wish to buy the drug privately. This is a carefully regulated process and they will provide more details on timelines as soon as possible. Following this, individuals seeking to self-fund should speak to their consultant to be assessed and if appropriate, prescribed for.
- Individuals seeking to access the medicine via private healthcare insurance, should contact their insurer to discuss terms of coverage. If it is covered, individuals should arrange a referral to an appropriate consultant within their network to be assessed and if appropriate, prescribed for.
12. In which European countries has omaveloxolone been approved to treat FA?
Ataxia UK is a member of Euro-ataxia, the membership organisation of 22 ataxia patient groups across Europe. Austria, Luxembourg, Switzerland and Slovenia have had approval from their health services that they will reimburse the cost of omaveloxolone for people with FA. Some other countries in Europe have early access schemes in place for people to receive omaveloxolone, including Germany, France and Greece.
