The Medicines & Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation for tofersen, a new drug for the treatment of SOD1 motor neuron disease (MND).

Read the MHRA announcement here.  

MND Scotland, along with the MND Association and My Name’5 Doddie Foundation, welcomes the approval of the first treatment for MND in the UK in almost 30 years.  

SOD1-MND is a rare genetic subtype of MND, accounting for about 2-3 % of total MND cases in Scotland. Tofersen is a targeted treatment that stops the faulty SOD1 code from being used to make SOD1 protein, reducing the amount of toxic material being made. 

Tofersen has been developed by Biogen.

In November 2024, the National Institute for Health and Care Excellence (NICE) confirmed tofersen would be appraised via the Highly Specialised Technologies (HST) route, following a successful ‘Prescribe Life’ campaign driven by the MND Association, and supported by MND Scotland and My Name’5 Doddie Foundation. NICE evaluates new health technologies for NHS use in England and Wales, considering clinical effectiveness and value for money.     

Tofersen will now be considered by NICE and SMC (Scottish Medicines Consortium) to determine whether it can be provided by the NHS.  In the meantime, the tofersen Early Access Programme (EAP) in the UK, which is made available by Biogen, remains open to eligible patients. The MND Association is currently leading a campaign to ensure everyone that is eligible can access tofersen via the EAP. 

Dr Jane Haley, Director of Research at MND Scotland, said: “This is a major step forward in finding effective treatments for MND. Biogen has developed a brand-new type of treatment for MND, targeting a genetic change, and the ambition is that it will be available for everyone with SOD1-MND throughout the UK.” 

The process by which a new drug is evaluated for use in the UK has been outlined in an infographic developed by the three main UK MND charities. You can find these here.

The three main UK MND charities are committed to working collaboratively to pursue every avenue available to us to make proven treatments available to people with MND as quickly as possible and on an equitable basis.