Prescribe Life - frequently asked questions

Tofersen is a ground-breaking new treatment for a small proportion of people with motor neurone disease (MND), a devastating and life-limiting neurological condition. In the UK there is currently only one treatment available (riluzole) which extends life by a matter of months.

Please see this short video for an introduction to tofersen and why it matters.

Tofersen is only effective for people who have a type of MND caused by a variation in a gene called SOD1, which is about 2% of people with MND. The only way to find out if you have a gene change, such as SOD1, associated with MND is to have genetic testing. 

If you are interested in this, please talk to your GP or neurologist. Deciding to have genetic testing can be difficult, as it can impact family members, as well as yourself.

Therefore, we always recommend having genetic counselling before you decide to have genetic testing. The counsellor will help explain the possible outcomes and impacts, to help you choose whether to go ahead. 

For more information, please read our booklet on inherited MND and genetic testing. 

If you have SOD1-type MND and are not receiving tofersen, ask your clinician to refer you to the Early Access Programme (EAP). Biogen, the pharmaceutical company that makes tofersen, is currently funding access to tofersen for SOD1 patients through the EAP.

We know however that some people who desperately need this treatment are missing out, because their local services don’t have the capacity to give it to them via the required monthly lumbar puncture – even though the drug itself is available for free. 

Asking the Government to step in and fix this is the new focus of our Prescribe Life campaign for Spring 2025 – see the main campaign page for more information and how to take action.

If you are experiencing difficulties receiving tofersen through the Early Access Programme and want to share your story with us please let us know on [email protected] or or 02038758910.

Last year, it looked like tofersen would not be made available on the NHS. The National Institute for Health and Care Excellence (NICE) is the body that decided which treatments the NHS would fund. Earlier in 2024 NICE had decided not to evaluate tofersen using the approach that gave it the best chance of being approved, saying MND wasn’t rare enough to qualify.

Yet NICE’s own guidelines said it could make exceptions to the rule, and we believed tofersen was worth making an exception for.

Based on this, in October we launched a petition calling on NICE to reconsider and adopt the right approach (called the Highly Specialised Technology or HST route) to appraising tofersen, so that it had the best chance of being made available to patients on the NHS.

Our Director of Engagement, Richard Evans, explained the situation at the start of the campaign in more detail.

Your signature on this petition joined more than 15,000 other names to show NICE that this issue mattered hugely – to the MND community and beyond. 

The campaign was also covered in the media and championed on social media, and throughout we continued to talk with NICE and other decision-makers to make our case. 

We hoped that NICE would see both the strength of public opinion and the justice of our position. We are delighted that by November 2024 it had accepted that tofersen is worth making an exception to its normal criteria for, giving it a better chance of becoming available on the NHS.  

MND is a fatal, rapidly progressing disease that affects the brain and spinal cord. It kills a third of people within a year of diagnosis and more than half within two years. MND leaves people locked in a failing body, unable to move, talk and eventually breathe. Six people every day die from MND in the UK. Another six are diagnosed.

There is no cure. 

More information on MND.

The National Institute for Health and Care Excellence (NICE) is an independent public body which helps to get patients the best care, while ensuring value for money for the taxpayer.

One of its roles is to assess new treatments for diseases and decide whether to make them available on the NHS for people who could benefit from them.

We have worked well with NICE on other projects – they are, for example, responsible for the clinical guidelines for health and social professionals on MND care.

But we disagreed with their position on tofersen.

We briefed MPs on the issues with the Early Access Programme at the meeting of the All-Party Parliamentary Group (APPG) on MND in April 2025. The APPG has written to Government and we continue to work closely with them to resolve the problem.

We will continue to keep MPs updated on progress, especially those with constituents with SOD1 MND affected by this problem. If you have a good relationship with your MP, contact us at [email protected] or 02038758910, and we can provide you with a briefing to share with them.

At present, the delivery of tofersen is not available in Wales or Northern Ireland.

We are aware that there are some people living with SOD1 MND in Wales who are accessing the tofersen EAP through local services in England, and some people with SOD1 MND in Northern Ireland who are accessing it through local services in the Republic of Ireland.

We are also aware that there are people living in Wales and Northern Ireland who are now unable to access those services due to capacity issues.

Our campaign is calling for the UK Government to work with the NHS to resolve these issues and ensure everyone can access tofersen who wants to. This may increase the capacity to deliver tofersen for those who are able to travel to England to access services. However, we are acutely aware that health policy is devolved in both Wales and Northern Ireland, and we are pursuing conversations with health and social care professionals in the devolved nations to explore delivery of the drug as close to home as possible.

For more information on our approach in Wales and Northern Ireland, please contact [email protected] 

No. Biogen has not yet applied for marketing authorisation in the UK, meaning tofersen can’t be prescribed privately.

Tofersen has been authorised in the EU and US. Please speak to your GP or neurologist in the first instance. We are unable to provide medical advice.

MIROCALS is completely separate from tofersen. Read more about the MIROCALS trial here along with our September 2024 update.

Please get in touch with the Campaigns Team on [email protected] or 02038758910 and we will do our best to help.