NICE confirms next steps for tofersen appraisal

NICE has confirmed the next stage in the appraisal process for tofersen, a treatment for the SOD1 form of motor neuron disease (MND). This follows NICE’s decision last year to assess the drug through its Highly Specialised Technologies (HST) route - a pathway used for treatments for very rare conditions.

In November 2024, NICE agreed to move tofersen onto the HST route after sustained campaigning from the MND community, including My Name’5 Doddie Foundation, MND Association and MND Scotland. This was an important moment, as it brought tofersen closer to potential NHS approval.

Tofersen is designed for a specific group of people living with MND caused by changes in the SOD1 gene. This affects around 2% of the MND population in the UK (approximately 60–100 people). Developed by pharmaceutical company Biogen, the drug works by reducing the production of a toxic protein that drives disease progression in this form of MND.

Biogen has confirmed today that the formal assessment process will begin in the new year, with a view to submitting its full evidence to NICE in June 2026. Biogen has also said it intends to submit tofersen to the Scottish Medicines Consortium later in 2026 through its Orphan Pathway.

Biogen has been working with NICE and NHS England over the past year to prepare for this next stage. They have acknowledged that assessing treatments for very rare conditions is complex, and have stressed the importance of collaboration and flexibility. They also highlighted how important it is for the voices of people with MND, their families and clinicians to be central to the process.

While this update represents progress, several steps still need to be completed before a final decision is made on routine NHS access. Any new treatment must first receive marketing authorisation from the Medicines and Healthcare products Regulatory Agency (MHRA). Decisions are then made about whether the treatment will be funded by the NHS in England and Wales, with Northern Ireland also following, and separately in Scotland.

Importantly, Biogen has confirmed it will continue to provide tofersen to eligible people through the Early Access Programme and Post-Trial Access routes while appraisal and reimbursement decisions are ongoing. This means new and existing patients who meet the criteria can continue to access the treatment during this period.

Our CEO Nicola Roseman said: “Last year’s decision to assess tofersen through the HST route was a crucial step forward. This update gives clarity on what happens next.”

“Although people affected by SOD1 MND still face a wait for a final decision, continued access through early and post-trial routes is vital. We will keep working with partners to push for progress and ensure people with MND and their families are heard at every stage.”

We will continue to keep the MND community informed as the appraisal moves forward.