Tofersen Access – UK Government failing to ensure effective drug reaches eligible patients

Almost 2 years ago we posted about the first truly effective treatment for any form of MND, tofersen (for SOD1 gene mutation mediated disease) and the growing delays in getting to UK patients.

Mutations in the SOD1 gene are the cause of MND in about 2% of all sufferers. That represents roughly 100 at any one time in the UK. In addition, 2 new patients every month are diagnosed on average. So even though 2% sounds small, these are both real numbers and very real lives.

Imagine being told you have MND.

Then imagine the joy of being told there is an effective treatment!

But then imagine being told that you can’t get it because of a post-code lottery in the UK!

That is exactly what around 20 patients and more monthly (the newly diagnosed) face being told in 2026.

To really comprehend how the delays have impacted patients we have to note that tofersen was FULLY approved in the USA just under 3 years ago now! And yet patients in the UK are still waiting.

Tofersen was approved in the UK by the MHRA on the 28th July 2025. We now wait on NICE to complete their review. However, Biogen are supplying free of charge whilst this review completes.

Are our regulators, the MHRA and NICE really fit for purpose in the 21st century?

Is this what our UK government wants from healthcare in the UK?

Is this the ambition of a government that wants to lead the world in research?

This horrific and emerging scandal is happening right now. How can it be solved? It certainly wouldn’t be tolerated in cancer care. It is simply money and there is no need for a scientific breakthough or discovery.

Yesterday, Dr Rachel Jakeman, step mother to Lillia, a 19 year old, with SOD1 MND, presented a petition and open letter to No. 10 Downing St to ask for their immediate intervention into this post-code lottery for the receiving of tofersen in the UK.

You can read below, Rachel’s open letter to the Prime Minister which we in United2endmnd fully support and have co-signed. You can also download a PDF copy of the letter here.

We are extremely grateful for both Geoff Burrow’s and Kevin Sinfield’s support along with leading researchers and neurologists.