Tofersen statement

It is completely unacceptable that the drug, tofersen, is only being given to some of those who are eligible to receive it. This drug, which is specifically for people living with SOD1 motor neuron disease (MND), caused by mutations in the SOD1 gene, is the first truly effective life-extending treatment for anyone with the condition.

Tofersen has been licensed by the UK’s MHRA for use in SOD1 MND patients, and is currently available free of charge from its manufacturer, Biogen. However, it cannot be accessed in many parts of the UK, because individual NHS Trusts and NHS Boards are choosing not to fund its delivery to people living with MND.

This has led to the ultimate postcode lottery and must change.

Tofersen is currently under review by NICE (National Institute for Health and Care Excellence), which will decide whether it can be provided by the NHS in England, Wales and NI. We expect a decision in the summer. In Scotland, these decisions are made by the Scottish Medicines Consortium (SMC), but Biogen has not yet submitted data for them to review.

While these reviews happen, NHS Boards and Trusts have the power to provide the resources that would allow their local patients with SOD1 MND to receive the drug.

Several NHS Trusts and Boards have not done so, including the whole of Scotland.

This must change immediately. This is the first drug that can make a meaningful difference for a certain group of people living with MND. It shows the potential of research to change the current reality of an MND diagnosis. Although it is only for around 1-2% of people living with the disease, progress like this offers hope to the whole community. For many people that hope is currently being extinguished. 

We will continue to highlight this issue by speaking with key stakeholders including the NHS, government and MND clinicians across the UK, as well as other charities including MND Association and MND Scotland.

There are many more genetic therapies being developed for MND. This means we need to address the delivery of these treatments now, to ensure this situation does not keep happening as they are developed. 

We will continue to fund the right research into MND. However, it is essential that any discoveries reach patients as quickly and fairly as possible. This is only possible with the support of the NHS.