#ACCESSOMAVNOW

Prime Minister, you stood with families affected by Friedreich’s ataxia. Now you can turn that support into action.

Dear Prime Minister,

On behalf of Ataxia UK, and the ataxia community we represent, may I begin by congratulating you on assuming the office of the Prime Minister.

As you enter Downing Street, I am writing to remind you of the support you gave last year to our #AccessOmavNow campaign when, as Mayor of Greater Manchester, you signed Ataxia UK’s open letter calling for urgent access to omaveloxolone (Omav), the only licensed treatment for Friedreich’s ataxia (FA).

Your name was one of the almost 11,000 added in support of our Open Letter to the Department of Health and Social Care, which called for:

1. The introduction of a new funded, temporary compassionate programme to help people with this devastating progressive condition.
2. A variation in the criteria for the NICE assessment of medications for rare diseases, like FA, that are more common than the 300 patients required by the Highly Specialised Technology appraisal system, but still extremely rare.

FA is a rare, degenerative, life-limiting and severely disabling neurological condition that progressively affects the nervous system leading to loss of balance and coordination of movement, as well as weakening of the heart and other complications and significantly reducing life expectancy to around an average of just 37 years. Approximately 1,100 people in the UK, and around 600 adults in England, are affected by FA, while more than 11,000 are living with all forms of ataxia.

Omaveloxolone (Omav, trading as Skyclarys) is the only licensed treatment for FA. The MHRA approved Omav in April 2025 for people aged 16 and over but it is not available to patients in England and Northern Ireland.

For a medicine to be available through the NHS in England, it must receive a positive recommendation from NICE after assessment of its clinical and cost-effectiveness. However, in April 2025, Biogen, the drug manufacturer, withdrew its submission following discussions with NICE. During their discussions NICE had indicated to Biogen that they would not proceed the evaluation to the committee stage, where the patient and clinical community would have had the opportunity to highlight the unmet need in Friedreich’s ataxia and potential of the treatment. This meant that NICE could not appraise the drug and so would not issue a final decision.

The drug is available to patients in many European countries, through state reimbursement or interim access programmes.

In Scotland, in March 2026, the SMC (NICE equivalent), issued a negative recommendation for omaveloxolone. However, a pathway (called PACS2) exists in Scotland through which clinicians may submit individual requests seeking access to omaveloxolone. Similarly, in Wales, clinicians can make individual funding requests for access to omaveloxolone in the absence of an appraisal recommendation. However, there is no interim access in England and Northern Ireland.

Reimbursement of omaveloxolone at a national level has been agreed in 11 European countries:

Austria, Croatia, Germany, Italy, Luxembourg, Poland, Portugal, Slovakia, Slovenia, Spain, Switzerland.

It is also available via interim access schemes in 8 additional European countries and has been available in the US since 2023.

Since you signed our open letter in June 2025, we, along with members of the FA community, met with Dr Zubir Ahmed, then Parliamentary Under-Secretary of State for Health. In his letter to us dated 6 May 2026, Dr Ahmed suggested that Biogen should formally seek a resubmission to NICE and engage with NHS England on commercial terms. He also indicated that the Innovative Medicines Fund (IMF) could be considered for managed access if the NICE process restarts with NHS England ready to support.

At a recent Ataxia UK Parliamentary Drop-In Event at Portcullis House, hosts Jonathan Brash MP and Mary Glindon MP were joined by dozens of MPs and peers who expressed their solidarity and support for people living with FA and their families.

During the event, Ataxia UK presented the following key recommendations

1. Provision of an interim access pathway for Omav while dialogue continues between stakeholders for a long-term solution. Exploring the use of the Innovative Medicines Fund (IMF) to support eligible patients.
2. Reform of the drug appraisal system for NHS reimbursement so that people with rare diseases are not left behind.

The ataxia and rare disease community welcomed the announcement on 2 July 2026 that there will be a series of pilot schemes to test ways to get innovative medicines to NHS patients faster, and support investment in the UK. The announcement explains that the Innovative Medicines Fund would be used to fund managed access agreements of medicines directly post marketing authorisation. We ask for an urgent meeting to explore whether and how Omav might be included in this pilot scheme.

Georgia Hart, a young woman affected by FA says: "Living with FA means living with a fear of knowing that the condition will keep progressing. It is incredibly hard to bear the thought that there’s a treatment available in many European countries and the USA, but you can’t access it here. Why?”

Arabella Faulkner, who was diagnosed with FA at the age of 15, says: “While I do not expect miracles, I do hope for fairness and equality. Every day matters with FA and we should not have to wait for a medicine that is already licensed.”

Prime Minister, we ask you to:

1. Direct the Department of Health and Social Care (DHSC) to convene an urgent meeting between NICE and Biogen to explore the potential for Omav to be part of the newly announced pilot scheme
2. Failing this, direct the DHSC to bring NHS England and Biogen together to navigate an urgent interim access pathway for Omav.
3. Direct NICE to make changes to the way in which rare disease medicines are appraised.
   • Change to the definition of ultra-rare conditions so more rare conditions qualify for the HST route.
   • Development of a proportionate evaluation route for rare neurological conditions that fall between existing Single Technology Appraisal and Highly Specialised Technology frameworks, to ensure these conditions are not structurally disadvantaged.

Patients like Georgia, Victoria and the many more living with this cruel condition simply can’t wait any longer. Why should patients in England be left with no access to a life-changing drug which is available in so many countries in Europe and in the United States of America?

As Georgia put it so clearly at our meeting with the DHSC last December:
“I used to feel like I was part of a global community of FA patients who would share experiences online and support each other to be resilient and just be young people together sharing our lives. That’s all gone now for me. Watching so many of my friends from other countries document the positive ways Omav is transforming their lives just makes me feel even more isolated. It’s crushing.”

Yours sincerely,

Sue Millman
Chief Executive
Ataxia UK

The recommendations in this letter have been endorsed at the time of publication by the following Parliamentarians:

Paula Barker MP

Jonathan Brash MP

Paul Davies MP

Mary Glindon MP

Dame Chi Onwurah MP

Rebecca Smith MP

Luke Taylor MP

Download a copy of the Open Letter here 

September 2026

Dear Ataxia UK,

Thank you for your request for an update on Biogen's engagement in efforts to secure reimbursement of omaveloxolone for the treatment of Friedreich's ataxia (FA) in people aged 16 years and older within the NHS in the United Kingdom (UK).

Firstly, I would like to reaffirm Biogen's commitment to the FA community across the country. We recognise that FA is a devastating condition and appreciate the profound burdens it places on people living with the disease and their families.

Biogen remains committed to working with health authorities in England to identify solutions that could enable broad access to omaveloxolone. We will continue to work in collaboration with stakeholders across England to support a positive outcome for the FA community. Encouragingly, national reimbursement for omaveloxolone has already been achieved in twelve comparable European countries, and we know how important it is to deliver similar progress for patients in England.

In response to your enquiry regarding Biogen's engagement in commercial discussions with NHS England, we continue to maintain regular dialogue with both NHS England and the National Institute for Health and Care Excellence (NICE) regarding omaveloxolone.

During the past year, Biogen representatives have met with senior leaders at NICE, NHS England and the Department of Health and Social Care. We are also developing an innovative solution, including scoping a potential omaveloxolone-specific service, and will continue to engage with the clinical community on how we move forward with NICE and NHS England.

In July, the Government announced four pilot programmes designed to explore ways of providing NHS patients with faster access to innovative medicines. One of the pilot programmes will seek to reform managed access arrangements and will include therapies for rare diseases.

While further details of the pilot have yet to be announced, Biogen has approached NICE and NHS England system leaders to seek exploratory discussions regarding the pilot and its potential implications for access to innovative treatments such as omaveloxolone.

Biogen will continue to pursue all appropriate opportunities to support access for people living with FA in England.

It is Biogen’s intention to resubmit to NICE. We will do so once we are confident that the submission will progress to a committee meeting, ensuring the appropriate level of stakeholder input is considered as part of the assessment.

Finally, I would like to extend my thanks to Ataxia UK and the FA community for their ongoing collaboration and support as we work together to advance access to omaveloxolone across England.

Yours sincerely,

Kylie Bromley BSc (Hons), PhD
General Manager and Managing Director, United Kingdom & Ireland
Biogen

Biogen-295649 | Sep 2026

APPENDIX

Further information requested.

1. Does the approval of omaveloxolone by the Health Service Executive (HSE) in Ireland have any implications for the UK?

We are very happy with the HSE’s decision to approve Skyclarys (omaveloxolone) and Biogen is proud to be able to bring this option to the Friedreich’s ataxia community in Ireland.

Biogen remains committed to continue working with health authorities in the UK to identify solutions that could enable broad access to omaveloxolone. We will continue to work in collaboration with stakeholders to support a positive outcome for the FA community.

Encouragingly, national reimbursement for omaveloxolone has now been achieved in twelve comparable European countries, and we know how important it is to deliver similar progress for patients across the UK.

2. Would you be able to provide more details on the "innovative solution, including scoping a potential omaveloxolone-specific service" and explain how this might help our FA community access treatment?

Biogen is working hard to develop the innovative solution; at this stage we are unable to provide further details.

This is in part because we are in dialogue with clinicians and in part due to the importance of commercial confidentiality, which has helped deliver reimbursement in twelve countries across Europe.

With the innovative solution, we are seeking to prepare a proposal for the Government and the NHS that will help to provide predictability as to the budget impact of the medicine.

We hope it will help the FA community access treatment, as it has in other countries where the medicine is nationally reimbursed.

3. Are you able to provide an approximate timeline of when you plan to resubmit to NICE?

It is Biogen’s intention to resubmit to NICE. We will do so once we are confident that the submission will progress to a committee meeting, ensuring the appropriate level of stakeholder input is considered as part of the assessment.

Biogen will continue to pursue all appropriate opportunities to support access for people living with FA in England.

4. What is involved in Biogen preparing for a resubmission to NICE? For example, does this involve further evidence gathering?

Uncertainty will remain a factor in the appraisal process, but we are continuing to gather evidence from patients treated with omaveloxolone in other countries which will be helpful in demonstrating outcomes when used in clinical practice.

The burden of illness study that Ataxia UK contributed to will also be useful in demonstrating the impact of FA on patients and their carers.

We have now seen the medicine approved in twelve different countries in Europe, and are hopeful a successful process could also be feasible in England.

We know that we will be able to bring a proposal to NICE and NHSE that will be in line with what has been approved in other countries, including Ireland, most recently.

Biogen-295649 | Sep 2026

5. Please can you provide further information on the pilot schemes.

The pilot schemes are still in development. We are seeking dialogue with NICE and NHS England on this topic.

Biogen would like to see the Managed Access Pilot provide an opportunity to take a different approach to the assessment of rare disease medicines.

These medicines often struggle to meet NICE's standard cost-effectiveness thresholds, which are applied in the same way to rare conditions as they are to more common diseases.

6. Are you able to provide an update on devolved nations? Do Biogen plan to submit to the Scottish Medicines Consortium (SMC) after NICE?

We will continue to explore opportunities to secure NHS funding in Wales, Northern Ireland and Scotland both through routine commissioning and individual patient funding routes.

In Scotland, Biogen submitted omaveloxolone to the SMC for a full Health Technology Assessment (HTA) for the treatment of FA in adults and adolescents aged 16 years and older in 2025.

Following its assessment, in March 2026, the SMC did not recommend omaveloxolone for use within NHS Scotland.

Biogen continues to explore opportunities through the SMC. We have meetings scheduled with representatives of the Scottish NHS and Government and will keep the community updated on next steps.

In Scotland, we also provide information to clinicians, at their request, to support their requests for funding for individual patients as required.

Biogen statement for Ataxia UK September 2026

PRESS RELEASE

Ataxia UK urges action as Republic of Ireland approves access to Friedreich’s ataxia treatment still unavailable to patients in Northern Ireland and the rest of the UK

London, 28 August 2026: Ataxia UK is calling again on people across the UK to add their name to the over 5,000 who have already signed our Open Letter to Andy Burnham after the Health Service Executive (HSE) in Republic of Ireland approved reimbursement for omaveloxolone, branded as Skyclarys, for eligible people living with Friedreich’s ataxia.

The decision means patients over the age of 16 in the Republic of Ireland are set to gain publicly reimbursed access to the only licensed treatment for Friedreich’s ataxia, while patients and families in Northern Ireland, England, Scotland and Wales still cannot access the same treatment through the NHS.

Friedreich’s ataxia is a rare, degenerative, life-limiting and severely disabling neurological condition. It progressively affects the nervous system, leading to loss of balance and coordination, weakening of the heart and other complications, and significantly reduced life expectancy.

Omaveloxolone is the first and only licensed treatment for Friedreich’s ataxia and it significantly slows the progression of the condition. The MHRA approved omaveloxolone in April 2025 for people aged 16 and over in the UK, but the medicine is still not routinely available to patients through the NHS in England, Scotland or Wales, or through HSC in Northern Ireland.

Ireland’s decision shows that progress is possible when health services, government and the manufacturer work collaboratively to find a route to patient access. Ataxia UK believes families in Northern Ireland and across the UK should not be left behind simply because reimbursement processes have stalled.

The charity is therefore renewing its call for urgent action and asking supporters to add their names to the Open Letter to Andy Burnham, urging him to help bring NHS England, the Department of Health and Social Care, NICE and the manufacturer, Biogen, together to explore an interim access pathway to omaveloxolone for people with Friedreich’s ataxia.

Sue Millman, Chief Executive of Ataxia UK, said: “The HSE decision is welcome news for families in the Republic of Ireland, but it also makes the inequality facing families in Northern Ireland and the rest of the UK impossible to ignore. People living with Friedreich’s ataxia do not have time on their side. We need urgent leadership to turn regulatory approval into real access for patients.”

The Open Letter asks Andy Burnham to use his voice and influence to help secure a practical interim access route while formal reimbursement decisions continue.

Ataxia UK is encouraging patients, families, clinicians, parliamentarians and members of the public to sign and share the letter. You can read and sign the Open Letter here.

ENDS

Download our press release here