#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