Today, Thursday the 26th of June 2025, NICE have released negative final draft guidance (FDG) for the drug zilucoplan as an add-on therapy in the treatment of AChR antibody positive myasthenia gravis (MG). This disappointing news comes shortly after the unsuccessful appeal of the release of negative FDG for efgartigimod. 

Zilucoplan has proven efficacy and safety in the treatment of MG. We are unfortunately seeing a similar pattern where MG patients are denied access based on cost effectiveness rather than how well they work to treat the disease. Myaware has been an involved stakeholder for the two committee meetings held to assess zilucoplan, and delivered distinct testimony through a key patient expert who had experience of the treatment. Again, NICE has stated they understand the burden of the disease and recognise the unmet need of MG patients to have access to novel therapies that target their disease. However, MG patients continue to be denied access to these medicines through the NHS.

Patients who received access to zilucoplan before this decision will continue to do so, as promised by the company UCB. However, myaware will continue to push for this access to be made available to all patients who meet the proposed criteria. As a charity, we vow to champion the rights of patients to have equitable access to therapy. We will look to appeal this decision, as we did with efgartigimod, and once again look to our membership to help us raise our voice in disagreement. 

We will be providing template letters for open use to allow patients, carers, and their families to contact their local MPs and make them aware of this evolving issue. We encourage any and all able to engage with decision makers across the country and let them be aware that the UK is being left behind in terms of clinical innovation, and this directly affects the most vulnerable patients. 

Yours Sincerely,

Trevor Ranson (Chairman), Bethan Peach (Medical Committee Chair), and Charlotte Campbell (Research and Partnerships Manager)