15 November 2024 News
A breakthrough drug, proven to substantially slow progression of the small proportion of people with motor neurone disease (MND) who have the SOD1 genetic change, is a step closer to being approved following a successful MND Association campaign.
The National Institute for Health and Care Excellence (NICE) has today (18 November) announced it has changed its mind about how it will assess tofersen, making it more likely it will eventually be approved.
The move comes a month after the MND Association launched its Prescribe Life campaign, alongside people affected by the disease, and working closely with the UK MND Research Institute and the UK MND Clinical Studies Group.
Over 15,000 people signed the charity’s petition urging NICE to evaluate tofersen using its Highly Specialised Technology (HST) route, which the charity said gave the drug the best chance of being greenlit as a treatment for SOD1 MND in the future.
Eleanor Dalley, 49, from Barnet is taking tofersen through the early access programme and has been a key member of the campaign team.
She said: “Today is really special for me – NICE has listened to the SOD1 MND community and heard us.
“By changing its original decision, NICE has taken another step down the road to securing this incredible drug, giving us time and hope, two things that often feel out of reach for people diagnosed with MND.
“I am so grateful to all the people who have felt driven to join the MND Association’s campaign by what we saw as the injustice of NICE’s original decision.”
NICE’s announcement on November 18 said that the ‘topic routing was discussed at the NICE Prioritisation Board in October’ and the ‘board concluded that the topic was suitable for Highly Specialised Technology’.
Tanya Curry, CEO at the MND Association, said: “We are absolutely delighted NICE has decided to change its mind on this.
"Tofersen is the first proven effective treatment for MND for many years, and we are incredibly proud that our campaign has helped our community get across how important it is that we give it the best possible chance of being approved.
“To be clear, there is still a long process to go through, and there is no guarantee that tofersen will be approved at the end of the process. But it does give our community renewed hope.
“While we disagreed with NICE’s original decision, it deserves credit for being flexible enough to reconsider.
“We now need Biogen, the pharmaceutical company that manufactures tofersen, to come back to the table and get the application process going again so we can move towards our ultimate goal - for people with SOD1 MND to access this life-changing treatment.”
In trials, tofersen has been shown to slow, and in some cases halt, the progression of MND in people with the SOD1 genetic variant, estimated to be around 60-100 people in the UK at any time.
The change in routing decision has now removed the logjam and while there is still a long way to go and there are no guarantees about the final outcome, the charity’s hope is that tofersen will now pass through the regulatory process.
You can read more thoughts from Richard Evans, our Director of Engagement, here.