Tofersen receives MHRA approval

Tofersen, the first drug for 30 years proven to slow progression of a form of MND, is one step closer to being available on prescription. 

The drug has today been granted marketing authorisation by the Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of SOD1 MND. This means it is now licensed for use in the UK. 

Biogen, the company responsible for the development of tofersen, has confirmed it will now submit the drug to the National Institute for Health and Care Excellence (NICE) to determine if it will be made available on the NHS. 

Thanks to our successful Prescribe Life campaign, NICE will appraise tofersen via the Highly Specialised Technologies (HST) route, which we and other experts believe gives it the best chance of being accepted for use on the NHS.  

MND Association Director of Engagement Richard Evans said: “Tofersen has been described as a game changer for the 2% of people whose MND is caused by a variation in the SOD1 gene, so the licensing of the drug is a really important step forward and one which, of course, we welcome.  

“However, it is important to stress that just because it has MHRA approval, it does not necessarily mean it will be available on the NHS, as it will be for NICE to decide if it considers it value for money. 

“What we need now is for tofersen to be appraised as quickly as possible by NICE and for it to be approved for use on the NHS.  

“Only then can we be confident that everyone with SOD1 MND – we think that’s around 60 to 100 people – can access the drug which could save their lives.” 

Tofersen is currently being made available through Biogen’s Early Access Programme (EAP). While around 40 people are being given it each month via a lumbar puncture, the MND Association knows of around 20 people who are being refused access because the NHS doesn’t have the resources to administer it. 

On July 17, alongside Seckin McGuirk, one of the 20 people currently being refused access, we hosted a day of action as part of our Prescribe Life campaign, highlighting this inequity.  

Richard added: “Although this announcement is positive news, sadly, people who desperately need tofersen now continue to be denied access through the Early Access Programme, despite Biogen making it available for free while it goes through the approval process. 

“Now that tofersen has been confirmed safe and effective by the MHRA, we urge the NHS and government to ensure that people with SOD1 MND can access tofersen through the EAP while regulatory processes continue.” 

The process by which a new drug is evaluated for use in the UK has been outlined in an infographic developed by the MND Association, MND Scotland and My Name’5 Doddie Foundation. You can find these here. 

The three 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.