Tofersen receives MHRA approval | MND Association

Tofersen, the primary drug for 30 years confirmed to sluggish development of a type of MND, is one step nearer to being out there on prescription. 

The drug has as we speak been granted marketing authorisation by the Medicines and Healthcare products Regulatory Agency (MHRA) for the therapy of SOD1 MND. This means it’s now licensed to be used within the UK. 

Biogen, the corporate liable for the event of tofersen, has confirmed it would now submit the drug to the National Institute for Health and Care Excellence (NICE) to find out if it is going to be made out there on the NHS. 

Thanks to our profitable Prescribe Life marketing campaign, NICE will appraise tofersen by way of the Highly Specialised Technologies (HST) route, which we and different consultants consider provides it the perfect likelihood of being accepted to be used on the NHS.  

MND Association Director of Engagement Richard Evans stated: “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 at the moment being made out there by means of Biogen’s Early Access Programme (EAP). While round 40 persons are being given it every month by way of a lumbar puncture, the MND Association is aware of of round 20 people who find themselves being refused entry as a result of the NHS doesn’t have the sources to manage it. 

On July 17, alongside Seckin McGuirk, one of many 20 folks at the moment being refused entry, we hosted a day of motion as a part of our Prescribe Life marketing 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 course of by which a brand new drug is evaluated to be used within the UK has been outlined in an infographic developed by the MND Association, MND Scotland and My Name’5 Doddie Foundation. You can discover these right here. 

The three UK MND charities are dedicated to working collaboratively to pursue each avenue out there to us to make confirmed remedies out there to folks with MND as rapidly as potential and on an equitable foundation.