NICE revises decision regarding tofersen
18 November 2024
Tofersen, a breakthrough drug for people with the form of motor neuron disease (MND) caused by genetic changes in the SOD1 gene, is now a step closer to being approved, following a change in how the National Institute of Health and Care Excellence (NICE) will assess the treatment.
In March this year we released a statement voicing our disappointment that NICE had decided not to appraise tofersen through the Highly Specialised Technologies (HST) route, which would have brought it closer to approval.
Since then, My Name’5 Doddie Foundation, in partnership with MND Association, MND Scotland and the MND clinical and research community has been campaigning for NICE to rethink how they assess tofersen. This included discussions with NICE, Genomics England, the Medicines and Healthcare Regulatory Agency (MHRA) and Biogen as well as supporting MNDA’s Prescribe Life campaign. Over 15,000 people signed a petition urging NICE to evaluate tofersen through their HST route. Today (18 November) NICE has announced that they will now assess tofersen through this route, bringing tofersen closer to approval and availability on the NHS.
Approximately 2% of the MND population, (60-100 people in the UK), have the SOD1 genetic change. Developed by Biogen, tofersen has been proven in trials to slow, and in some cases halt, MND disease progression in people who have the SOD1 genetic alteration.
Tofersen is an antisense oligonucleotide (ASO) which comes under the umbrella of “gene therapies.” Gene therapy approaches target disease-linked genes or RNA, aiming to stop the production of toxic proteins. Stopping the production of toxic SOD1 protein, through treatment with tofersen, can lead to potential improvement in symptoms of MND in those who have the SOD1 mutation. We have created an infographic explaining how this therapy works.
Today’s announcement by NICE said ‘topic routing was discussed at the NICE Prioritisation Board in October’ and ‘the Board concluded that the topic was suitable for Highly Specialised Technology.’
Although this decision is promising, there is still a long way to go until tofersen is available for people with SOD1 MND in the UK. Any drug or treatment prescribed in this country needs to receive marketing authorisation by the Medicines and Healthcare Agency (MHRA). If tofersen is approved and receives marketing authorisation, then decisions will be made on whether it will be available on the NHS. The Scottish Medicines Consortium makes this decision in Scotland, but has not yet stated on how it will approach its appraisal.
Our CEO Nicola Roseman said: “This is a very promising announcement and we are extremely pleased to hear NICE has decided to assess tofersen through the HST route. People with MND need access to proven effective treatments as quickly as possible. This decision by NICE should bring that closer for those with the SOD1 form of MND.”
“Despite this promising news, we can’t say for certain whether tofersen will definitely be approved, but we are hopeful that it will ultimately be available on the NHS. We will continue to work in collaboration on behalf of those who can benefit from this therapy.”
My Name’5 Doddie Foundation, in partnership with MND Association and MND Scotland, have produced a set of infographics on how drugs are discovered, developed & approved.
