Tofersen, a drug shown to slow the progression of SOD1-MND, is closer to approval following a successful campaign by the MND community.

Motor neuron disease (MND) is a progressive neurological condition with no current cure or effective treatment.

A small number of people have MND caused by a change in the SOD1 gene. A breakthrough drug called tofersen, which targets this type of MND has been shown in trials to slow and, in some cases, halt the progression of the disease.

Following intense discussions and requests lodged by leading researchers, clinicians, policy advocates, patients, and patient associations including the MND Association, the National Institute for Health and Care Excellence (NICE) has reconsidered how tofersen will be evaluated, taking it closer to approval for use in the UK.

 

NICE reconsiders tofersen approval process

Groups campaigned to ensure that tofersen is offered a route that ensures the drug’s best chance of approval.

NICE has now announced that it will use the HST pathway, a set of recommendations on the use of new and existing highly specialised medicines and treatments within the NHS in England.

Nicola Waters, who lives with MND, explained what the news means to patients like her: “We are delighted that neuroscientists working as part of the UK MND Research Institute have helped to convince NICE that SOD1-MND should be considered as a distinct disease for their assessment. This is great news for patients now and into the future.”

 

What this means for SOD1-MND patients

This represents a significant step forward, and we must ensure that tofersen is available on the NHS as quickly as possible.

We will encourage Biogen, the pharmaceutical company behind the drug, to resubmit its application as soon as it can.

The UK MND Research Institute welcomes the news, and this decision offers renewed hope for people with SOD1-MND and their families that tofersen may become available on the NHS.

Professor Ammar Al-Chalabi, who is co-Director of the UK MND Research Institute, further explained the importance of this news: “Prior to this announcement from NICE, tofersen was being assessed as if it were a standard therapy. Now it will be considered as the specialised, targeted treatment it is, which greatly improves the chances of approval for use on the NHS.”

Professor Christopher McDermott, co-Director of the UK MND Research Institute, added: “People with MND and researchers in the UK were part of the international trial that proved tofersen is an effective therapy for SOD1-MND. With tofersen now approved in the USA and Europe, it is a relief that NICE and the MHRA will look at the possibility of making this treatment available to people in England and Wales.

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