A breakthrough drug, proven to substantially slow the 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 recently announced it has reconsidered its routing decision for Tofersen. The move comes a month after the MND Association launched its Prescribe Life campaign, alongside people affected by the disease.
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 was diagnosed with SOD1 MND in July 2019. She has received Tofersen through an Early Access Programme since November 2022, but there were no guarantees about how long the programme would stay open to new patients.
Three members of Eleanor’s family have died of the disease. 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, which offers people like me 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 of 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.
However, following NICE’s original decision to appraise it using the Single Technology Appraisal framework instead of the HST, Biogen thought that the chance of it being approved at the price it needed was so low that it did not move forward with its application.
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 can now pass through the regulatory process.
Tanya added:
“People with MND don’t have time to waste, so the sooner they have the security of knowing the drug is available to them on the NHS the better.
“And of course, we want to say a massive thank you on behalf of everyone with SOD1 MND and their loved ones to every single person who signed the petition, shared it on social media or raised awareness.”
Professor Ammar Al-Chalabi, Director, of King’s Motor Neuron Disease Centre and co-director, of UK MND Research Institute, said:
“The effects of this treatment are truly remarkable. The HST route was only ever the right way to appraise this drug to ensure patients can access it in the future.
“It’s also a signal that NICE are focusing on the future of precision medicine – knowing that, and seeing a clear and obvious path to market, will encourage pharmaceutical companies to bring their future innovative science and clinical studies to this country.”
MND is a fatal, rapidly progressing disease that affects the brain and spinal cord. It can affect a person’s ability to walk, talk, eat and ultimately to breathe.
A third of people die within a year of diagnosis and half within two years. There is currently no effective treatment and no cure.
For more information about MND and the MND Association please visit: www.mndassociation.org.
