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NICE publishes draft guidance on zilucoplan for myasthenia gravis treatment

4 July 2024

The National Institute for Health and Care Excellence (NICE) has published draft guidance that zilucoplan, a potential treatment for antibody-positive generalised myasthenia gravis, is not recommended within its marketing authorisation as an add-on to standard treatment for generalised myasthenia gravis in adults who test positive for anti-acetylcholine receptor antibodies.

The NICE committee carrying out the appraisal concluded that clinical trial evidence suggests that zilucoplan plus standard treatment does improve symptoms and people’s ability to carry out their normal activities when compared to standard treatment alone, but that there are some uncertainties in the clinical evidence for which it would like further clarification. The draft guidance points specifically to zilucoplan not having been compared with intravenous immunoglobulin and plasma exchange, so it’s unclear how well it works compared with these. It also states that there is some uncertainty about the cost-effectiveness estimates for the treatment.  

This is not NICE’s final guidance on zilucoplan. The draft guidance outlines NICE’s current recommendations and is now open for public consultation.

What does this mean?

If this draft recommendation is accepted, it means people not currently receiving zilucoplan through the NHS in England will be unable to access it. However, people already being treated will be able to continue to receive it until their doctor considers it’s appropriate to stop.

The NICE appraisal consultation document can be read in detail and is now open for consultation until Thursday 25 July 2024.

Our response and next steps

We’ve worked closely with the myasthenia gravis charity Myaware throughout the zilucoplan appraisal process. We nominated and supported two people living with the condition and receiving zilucoplan as Patient Experts.

We’re disappointed by the draft recommendation but encouraged that the NICE committee has recognised zilucoplan is a potentially effective treatment. As part of the appraisal process, we heard directly from the myasthenia gravis community about the importance of access to treatments that can better control myasthenia gravis.

It’s very common for NICE to not recommend a treatment at this stage. The decision often changes after the consultation and a second committee meeting has taken place – for zilucoplan, this second committee meeting is yet to be scheduled and we’ll keep you informed once NICE confirm the date.

We’ll be taking part in the consultation and are currently looking at the key areas where we feel the views of our community will have the most impact.

We also urge NICE and the company that makes zilucoplan, UCB, to work closely together between now and the second appraisal committee meeting to address the evidence gaps raised in the draft guidance and to find a solution that means new patients can benefit from zilucoplan when clinically appropriate.

How you can share your views

We’ll continue to work with all parties involved and to present the patient voice in partnership with the community and other patient groups. Over the coming weeks, we’ll let you know how you can support our consultation response and how to make your own individual response.

In the meantime, you can share your views with us by emailing our Campaigns teams

If you’ve been affected by this announcement and would like support, get in touch with our helpline by calling 0800 652 6352 Monday to Thursday, between 10am-2pm,  or email

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