New NHS treatment approved for myasthenia gravis

New NHS treatment approved for myasthenia gravis

A new targeted treatment for myasthenia gravis has been approved for use on the NHS in England, marking a major step forward for people living with the condition.

The decision by National Institute for Health and Care Excellence (NICE) means that rozanolixizumab will now be available to eligible patients, following a period in which several similar treatments had been rejected despite strong support from patients and clinicians.

Over the past two years, Muscular Dystrophy UK (MDUK) has worked alongside people with myasthenia gravis, healthcare professionals and the charity myaware to campaign for improved access to effective therapies. Campaigners argued that access to the right treatment could make a significant difference to daily life.
 

What is myasthenia gravis?

Myasthenia gravis is a rare condition in which the immune system attacks the point where nerves and muscles communicate. This disrupts signals from the brain, leading to muscle weakness, fatigue and difficulties with everyday activities.

For many people, the condition can have a substantial impact on independence. Some may need to give up hobbies, reduce working hours or carefully plan daily routines to manage symptoms.

Initial treatments typically include steroids or other immunosuppressants. While these can be effective for some, they do not work for everyone and may cause significant side effects. Targeted therapies such as rozanolixizumab offer an alternative for people whose symptoms remain difficult to control.
 

Why this decision matters

Rozanolixizumab has been approved for people with generalised myasthenia gravis who have acetylcholine receptor (AChR) or MuSK antibodies.

It is the third targeted therapy for the condition to be assessed by NICE. Earlier treatments, including efgartigimod and zilucoplan, were not recommended, leaving patients without access to newer options.

Earlier this year, MDUK successfully appealed NICE’s decision on zilucoplan. As part of that appeal, the panel agreed it was unreasonable to exclude Minimal Symptom Expression (MSE) from its decision-making process.

MSE refers to a state where a person has no or very few symptoms, something many people with myasthenia gravis describe as life changing. Following the appeal, the NICE committee assessing rozanolixizumab gave greater weight to this outcome, helping to inform its positive recommendation.

MDUK said the decision better reflects the lived experience of people with the condition and the outcomes that matter most to them.
 

A milestone, but more to do

While the approval represents a major milestone, MDUK emphasised that not all patients respond to the same treatment and that further options are still needed.

Some targeted therapies remain unavailable on the NHS, meaning choice is still limited for many people.
 

Andy Fletcher, CEO, Muscular Dystrophy UK:

“This is hugely important news for the myasthenia gravis community. We’ve consistently heard how much access to the right treatment matters and today brings long-awaited progress. We’re proud to have worked alongside people living with myasthenia gravis to help make this happen – and we will continue pushing until everyone with a muscle wasting or weakening condition has access to the treatment that’s right for them.”

What does this mean across the UK?

In Wales and Northern Ireland, NHS bodies often follow NICE guidance, so access to rozanolixizumab is likely to be introduced in line with the decision in England, although local implementation can take time.

In Scotland, decisions are made separately by the Scottish Medicines Consortium (SMC). At present, there has been no confirmed decision on rozanolixizumab from the SMC, so availability there is still uncertain.

More broadly, people with myasthenia gravis across the UK may still face variation in access to newer targeted treatments, depending on local approvals and commissioning decisions.
 

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