About Tofersen for motor neurone disease (MND)

Tofersen is a genetic therapy developed for a rare inherited form of motor neurone disease (MND) caused by mutations in the SOD1 gene, which affects around 2% of people with MND. It is designed to reduce production of the faulty SOD1 protein believed to contribute to motor neurone damage, although the extent of its clinical effectiveness is still being evaluated.

The MHRA approved Tofersen in July 2025 for adults with SOD1‑related MND; however, it has not been approved by NICE for routine NHS use, and the NICE appraisal process is ongoing.

It is only when NICE approval has been given that funding becomes available from NHSE to deliver it.

Although the manufacturer currently provides the drug free of charge through early access schemes, there is currently no NHSE funding to support the delivery of the service required to administer the treatment, meaning some NHS Trusts are not routinely able to offer it at this time.

University Hospital Southampton’s position

Like all NHS Trusts, we are facing challenging times and are required to live within our financial means.

Funding from NHS England to deliver Tofersen to patients is dependent on NICE approval which has not yet been given.

While this drug is currently being offered for free by a manufacturer through an early access programme, unfortunately we have not received any funding to pay for the increased service capacity we need to safely deliver this treatment and closely monitor a patient’s ongoing progress.  

We have therefore made the difficult decision that we are unable to provide Tofersen at this time, because doing so would mean taking resources away from other patients. 

We understand this will be deeply disappointing to families affected. The Trust will work towards being able to offer this treatment as soon as possible, if and when it is approved by NICE and funding is made available from NHS England.