Following a successful campaign by the MND Association, Tofersen, a promising drug for individuals with motor neurone disease (MND) who have the SOD1 genetic change, is nearing approval. The National Institute for Health and Care Excellence (NICE) has reconsidered its initial decision regarding the drug's evaluation process.
The decision comes after the MND Association launched its Prescribe Life campaign, which garnered over 15,000 signatures on a petition urging NICE to assess Tofersen via the Highly Specialised Technology (HST) route. The charity argued that the HST route would provide the drug with the best opportunity for approval as a treatment for SOD1 MND.
Eleanor Dalley, a patient diagnosed with SOD1 MND in July 2019, has been receiving Tofersen through an Early Access Programme since November 2022. She emphasized the significance of NICE's reconsideration, stating, "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."
Tanya Curry, CEO of the MND Association, expressed delight at NICE's change of mind, stating, "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." She also urged Biogen to proceed with the application process.
Clinical Evidence and Impact
Clinical trials have demonstrated that tofersen can slow, and in some cases halt, the progression of MND in individuals with the SOD1 genetic variant. This variant is estimated to affect approximately 60-100 people in the UK at any given time. The drug works by targeting the SOD1 protein, which is implicated in the disease's pathology.
Professor Ammar Al-Chalabi, Director of King’s Motor Neuron Disease Centre, commented on the significance of the HST route, stating, "The effects of this treatment are truly remarkable… It’s also a signal that NICE are focusing on the future of precision medicine."
The Need for New Treatments in MND
MND is a rapidly progressing and fatal disease affecting the brain and spinal cord, impacting a person's ability to walk, talk, eat, and breathe. A third of individuals with MND die within a year of diagnosis, and half within two years. Currently, there is no cure and limited effective treatments, highlighting the urgent need for innovative therapies like tofersen.
Next Steps
While NICE's decision is a positive step, the approval process for Tofersen is not yet complete. Biogen's decision to halt its application after NICE’s initial decision to appraise it using the Single Technology Appraisal framework. The change in routing decision has now removed the logjam, and the charity hopes that Tofersen can now pass through the regulatory process.
