Families call for NHS access to approved Friedreich’s ataxia treatment

October 24, 2026

Families affected by the rare genetic condition Friedreich’s ataxia (FA) have taken their campaign for NHS access to an approved treatment to Downing Street, following a petition signed by more than 100,000 people.  

FA is a progressive, degenerative condition primarily affecting parts of the nervous system and the heart. 

The campaign calls for wider access to omaveloxolone, the only approved drug treatment for FA, which remains unavailable through the NHS despite receiving UK regulatory approval in 2025. Clinical trial data has demonstrated that the drug significantly improved neurological function and significantly reduced physical impairment in people with FA compared to a placebo group.   

Omaveloxolone, developed by biotechnology company Biogen, has been approved for patients aged 16 and over. However, discussions over NHS funding have not resulted in an agreement, leaving patients facing an estimated private treatment cost of £300,000 per year.  

The National Institute for Health and Care Excellence (NICE), the body that evaluates whether drugs offer value for money to the NHS, said that Biogen withdrew its evidence submission to it in May 2025. In a letter to the patient foundation Ataxia UK, Biogen stated they withdrew from the appraisal process after NICE indicated that they would not proceed to committee stage of the process.  

A petition calling for NHS access to omaveloxolone closed in September 2026 after attracting more than 100,000 signatures, meeting the threshold for consideration for a parliamentary debate. Families affected by FA have since delivered the petition to Downing Street, seeking government intervention. At the time of writing, the petition remains amongst those waiting for a debate in Parliament.  

Although patients with FA can access support services such as occupational, speech and physical therapy through the NHS, omaveloxolone is the only approved pharmacological therapy available to patients. The availability of an approved medicine for FA but an inequitable path to accessing it presents a challenging and unsatisfactory situation for the FA community in the UK and is representative of some of the challenges affecting rare disease drug development.  

This petition highlights the need not only for approved rare diseases therapies to be available to patients in the UK through the NHS or via an alternative attainable reimbursement pathway, particularly where the cost of accessing the medicines privately is prohibitive, but also the need for more effective drugs to continue to be developed for rare diseases. 

The mission of the Oxford-Harrington Rare Disease Centre (OHC) is to deliver new medicines to the clinic to address the huge unmet need facing the majority of people living with rare conditions that lack approved therapies. In a philanthropically funded programme, the OHC is advancing FA research towards identification and development of new candidate therapies for FA. 

Carlo Rinaldi, Professor of Molecular and Translational Neuroscience at University of Oxford and scientific lead of OHC’s FA programme said: “Ensuring that approved treatments for FA and other rare diseases reach UK patients who desperately need them is of paramount importance, and will require both reform of the reimbursement process and continued innovation to bring more efficacious therapies into the pipeline.” 

In a recent update to Ataxia UK, Biogen has reaffirmed its commitment to securing broader access to omaveloxolone, including scoping a potential omaveloxolone-specific service. NICE has stated it will review its decision to not make a recommendation on omaveloxolone if Biogen decides to make a new submission.