Charities welcome MHRA approval of drug to treat genetic subtype of MND

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).

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

SOD1-MND is a rare genetic subtype of MND, accounting for about 3% of total MND cases. 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 My Name’5 Doddie Foundation and MND Scotland. 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 in the UK, which is made available by Biogen, remains open to eligible patients.

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.