We won this one

Update 17th Sept - When the Great British PAC first became aware of this petition it was just over 35,000 signatures (4th Sept 26) with an expiry date of 24th September. We set an aim to get it over 100,000. ON the 16th September the petition exceeded 100,000 and has been update to say it will be considered for debate in Parliament. We cant prove that our input made a difference but this is a great example of where people power lies.

The government has provided an official response to this petition. Details can be found on the government website however we have provided part of the response here for transparency.

Petition: Fund NHS access to Omaveloxolone for patients with Friedreich's Ataxia

The National Institute for Health and Care Excellence (NICE) is the independent body responsible for developing evidence-based recommendations for the NHS on whether new, licensed medicines represent a clinically and cost-effective use of NHS resources. The NHS in England is legally required to fund medicines recommended by NICE.

NICE initiated the appraisal of omaveloxolone for the treatment of Friedreich’s ataxia for people aged 16 and over in anticipation of a licence being granted by the Medicines and Healthcare products Regulatory Agency (MHRA). The MHRA is the independent body responsible for licensing medicines for use in the UK, based on an assessment of their safety, quality and efficacy.

NICE’s appraisal process requires the manufacturer of the drug to make an evidence submission. NICE has unfortunately been unable to develop guidance for the NHS on the use of omaveloxolone for treating Friedreich's ataxia in people 16 years and over because the company, Biogen, withdrew its evidence submission. NICE will reopen the appraisal if the company decides to make a new evidence submission.

Fund NHS access to Omaveloxolone for patients with Friedreich's Ataxia

Friedreich's Ataxia patients need access to the drug Omaveloxolone. Without treatment, this degenerative disease can get worse. Patients & families may have to see their loved ones deteriorate when they are aware there is a drug available to stop the progression of this disease & in many cases, to improve the symptoms.

What we demanded

1
We call on the Government to fund NHS access to Omaveloxolone in the UK for the treatment of Friedreich's Ataxia.

The petition as it was published

We call on the Government to fund NHS access to Omaveloxolone in the UK. We believe the drug should also be considered under the HST programme, which we feel is the only appraisal route which reflects the realities of Friedreich's Ataxia. This is a degenerative disease & time is of the essence.

Friedreich's Ataxia patients need access to the drug Omaveloxolone, the only drug available for this disease. Without treatment, this degenerative disease can get worse. Patients & families may have to see their loved ones deteriorate when they are aware there is a drug available to stop the progression of this disease & in many cases, to improve the symptoms. The drug is available in EU countries & America. It passed the safety test in April 2025 and we believe it should be available on the NHS!