17 March 2025 News
Last autumn tofersen, a breakthrough drug proven to substantially slow progress of MND in people with the SOD1 genetic change, moved a step closer to being approved following our successful campaign, Prescribe Life.
Since then, we’ve met with Biogen, the pharmaceutical company responsible for developing tofersen. Biogen has committed to keeping its early access programme (EAP) open so people with SOD1 MND can access the drug.
Unfortunately, tofersen isn’t an easy drug to take – it’s not just a case of swallowing a tablet. It has to be injected monthly via a lumbar puncture.
This involves a needle being passed between the bones in the spine to put the drug directly into the spinal fluid. It has to be done in a clinic or hospital, by a medical professional.
Because of that, even though Biogen is providing the drug for free, there is a cost to the NHS of administering tofersen. Staff must be trained and paid, clinic time has to be paid for, and it is an invasive procedure so support staff need to be on hand to help.
At the moment, none of those services are formally in place so clinics are offering treatment on a ‘goodwill’ basis. That means there is a limit on the number of people who can receive the drug. Now, in some areas, that limit appears to have been met and we have started to receive reports of a small number of people with SOD1 MND who can’t access it.
As soon as we were made aware of this issue, we began compiling information to understand the full picture. From that research, which includes speaking to specialist clinics, neurologists and people with MND, we estimate there might be roughly around 10 people who have SOD1 MND can’t currently access tofersen because the NHS doesn’t have the capacity to deliver it.
Tofersen gives not just a glimmer of hope to people with SOD1 MND, but a future. It is the difference between life and death. The drug itself costs nothing at the moment - yet we risk people dying for want of the NHS not being able to pay for the cost of administering it.
We are hopeful tofersen will pass through the necessary regulatory process and be available on the NHS in the future, with no barriers.
But while we wait for that process to run its course, we need to make sure people with SOD1 MND can access this life-changing drug.
We have alerted Biogen to this issue. They have confirmed they are willing to continue to provide tofersen to any clinician who requires it for a person with SOD1 MND.
We are also pleased that NHS England has now agreed to meet with us to discuss this issue, and we will do everything we can to help them to find a solution.
We will continue to share updates.
