NICE assessment of tofersen to being in 2026

Earlier this year, we welcomed MHRA approval of a new drug, tofersen, to treat a genetic subtype of MND.

The National Institute for Health and Care Excellence has advised Biogen, the manufacturer of tofersen, that their assessment of tofersen will restart in the new year. Tofersen will now be considered by the National Institute for Health and Care Excellence (NICE) to determine whether it can be provided by the NHS in England.

Biogen, the manufacturer of tofersen, has worked closely with NICE and NHS England to support progress on the appraisal of tofersen for the treatment of SOD1 MND. Following NICE’s confirmation today, Biogen also confirmed It is their intention to submit to the Scottish Medicines Consortium (SMC) later in 2026 via the Orphan Pathway to be considered for people with SOD1-MND in Scotland.

In the meantime, the tofersen Early Access Programme (EAP) 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.

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.

Evaluating treatments for very rare conditions is inherently complex, and it will be essential for all parties to work collaboratively and with flexibility. Most importantly, the voices of patients, their families and the clinical community must be central throughout the appraisal.

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.