Second treatment for myasthenia gravis not approved for NHS use in England
26 June 2025
Zilucoplan (Zilbrysq), a treatment for myasthenia gravis, has not been approved for use on the NHS in England. Coming just weeks after efgartigimod, another treatment for myasthenia gravis, was also turned down, this second decision is a major setback for people living with the condition.
Although NICE (the National Institute for Health and Care Excellence) recognised zilucoplan “improves symptoms and people’s ability to carry out their normal activities”, they decided that the benefit it offers isn’t currently enough to justify the cost to the NHS.
This is the second treatment for myasthenia gravis to be described as not cost-effective in recent weeks. Earlier this month, efgartigimod was also denied NHS funding.
Both zilucoplan and efgartigimod were seen as promising options for people who don’t respond well to current treatments. In myasthenia gravis, the immune system mistakenly attacks the way messages travel from the brain to the muscles. While the two treatments work in different ways, each targeting a different part of the immune system, they share the goal of reducing symptoms and improving quality of life.
Now that both treatments won’t be available on the NHS, people with myasthenia gravis are left with fewer options for managing their condition.
“This is a double blow for people living with myasthenia gravis – a long-term, chronic condition with no cure. In just weeks, two promising treatments have been ruled out for NHS use. That’s not just disappointing, it’s devastating for those living with myasthenia gravis. It is another example of how the system fails people with rare conditions. People living with myasthenia gravis deserve better, they deserve access to potentially life-changing treatments.”
Andy Fletcher
Our Chief Executive
How these treatments could help
Efgartigimod and zilucoplan are not cures, and can cause side effects. But for many people with myasthenia gravis, they have made a difference.
Those who have used efgartigimod talk about reclaiming daily independence – walking, dressing, and taking part in family life again. Some have even been able to go back to work or school, feeling hopeful for the future.
Similarly, people on zilucoplan describe less fatigue, stronger muscles, and the ability to walk safely. Many have gone from needing help all the time to doing things on their own, like washing, dressing, and exercising. For some, it means they can safely meet friends, travel, and even play sports again.
These aren’t small improvements. They show just how powerful the right treatment can be.
Lesley, a grandmother from Watford, was diagnosed with myasthenia gravis in 2002. The condition has been debilitating, often leaving her exhausted. She began taking zilucoplan in 2021 and describes the treatment as “life-changing.”
“Since taking the treatment I’ve never felt better. It’s completely changed me and given me my life back. I feel so much more confident, optimistic and have a positive outlook. After taking zilucoplan, I could reduce other medications, such as steroids, which was ruining my life. I reacted badly when taking them and ended up in hospital with severe electrolyte deficiency. After cutting them out I lost 40kg in weight. The thought of a return to the uncertainty, fear and worry that this condition can bring would be catastrophic for me. I’m devastated that others eligible now won’t be able to experience the benefits too.”
Lesley
Benefited from zilucoplan treatment
Access to treatment going forward
UCB, the manufacturer of zilucoplan, has confirmed that people currently receiving the treatment through an early access programme will continue to have access, for as long as their doctor feels it’s needed. However, new patients won’t be able to start the treatment.
Similarly, Argenx, the manufacturer of efgartigimod, has confirmed people already receiving it via early access can continue treatment, but no new patients will be eligible.
We welcome UCB’s and Argenx’s commitment to continuing treatment for those already on it and appreciate their ongoing efforts to find a way forward for everyone who could benefit from these treatments.
What this means across the UK
Wales and Northern Ireland typically follow NICE guidance, so the decisions are likely to apply there as well. Scotland has a separate medicines assessment process, so these rulings do not apply to people with myasthenia gravis in Scotland. We’ll continue to work alongside the myasthenia charity Myaware to ensure the voices of those affected are heard throughout this process. Both companies are currently in discussions with the relevant authorities in Scotland.
Next steps
We’re deeply frustrated and very concerned by this decision. It denies people the chance to try new treatments that could improve their lives. It also highlights a wider issue – the system continues to fall short when it comes to assessing treatments for rare and complex conditions.
We’re committed to standing with the myasthenia gravis community as we navigate the impact of this news. This means working closely with doctors, partner organisations, and people living with the condition to understand the impact and explore what can be done to help improve access in the future. Our priority remains clear – we want to ensure the voices of people with muscle wasting and weakening conditions are heard and push for fairer access to potentially life-changing treatments.
If you have been affected by this news and would like support please do reach out to our helpline, call 0800 652 6352 or email info@musculardystrophyuk.org.
