Update: 28th January 2026

In July 2025 NICE published final draft guidance (FDG) for the drug zilucoplan, not recommending it for reimbursement on the NHS. Myaware, alongside our colleagues at MDUK, the Association of British Neurologists (ABN), and the company UCB appealed this decision based on several grounds. 

We are pleased to say that as of today this appeal has been upheld by the committee at NICE, as described in an appeal outcome letter by NICE:

The appeal panel (therefore) upheld the appeal on UCB’s appeal point 1(a)3, ABN’s appeal point 2.3, MDUK’s appeal point 2.3, Myaware’s appeal point 2.3 and UCB’s appeal point 2.5.

The appraisal is remitted to the appraisal committee who must now take all reasonable steps to share further details of the NHS England paper regarding treatment switching rates with UCB as well as further details as to how these were integrated with the data in the Clarivate expert elicitation report to arrive at its preferred assumptions about treatment switching rates to be applied in the model; re-consider approaches to the inclusion of MSE in the modelling and provide UCB with explicit recommendations as to how it may do this in order to achieve more plausible outcomes; and reconsider the results of the modelling in light of the inclusion of MSE.

This statement identifies that two appeal points have been upheld, one ground 1 point from the company and a combinatorial ground 2 point from myaware, MDUK, the ABN, and UCB. By definition, a ground 1 point is where:

"In making the assessment that preceded the recommendation, NICE has:

(a) Failed to act fairly; and/or

(b) Exceeded its powers."

A ground 2 point is described as:

"The recommendation is unreasonable in light of the evidence submitted to NICE."

What this means is that there will now be another committee meeting after a period of engagement between NICE and the company UCB. At this meeting, it is hoped that any further clarification needed by NICE regarding zilucoplan can be provided and we can proceed to a positive outcome for all. As soon as we hear any further information we will ensure to share this with our members.

Myaware's upheld appeal point 2.3  was submitted as follows:

"NICE's conclusion that minimal symptom expression should be removed from the model is unreasonable."

Myaware argued that the impact of minimal symptom expression (defined as an MG-ADL score of 0 or 1) for patients who have lived with symptomatic myasthenia is a significant factor in proving the benefit of the drug. The preferred assumption of NICE that it should be removed from the model as a factor of consideration was something we felt particularly strongly about and we'd directly like to thank our patient expert Abby Mabil who spoke so clearly and passionately about her experience. We have no doubt this will have gone a long way in helping the appeal committee understand our viewpoint. Myaware had a fantastic team of Abby, our legal advisor Sarah Love, and clinical expert Dr Channa Hewamadduma taking part in the appeal hearing and we are incredibly grateful for their efforts in making this appeal successful. We'd also like to express our appreciation for our fellow stakeholders at MDUK, the ABN, and UCB who all worked tirelessly in ensuring the patient voice was heard on the day.

Myaware will continue to engage with all stakeholders involved in the appraisal of this medicine, which has been shown to be lifechanging for those who have been treated with it through clinical trial. We are pleased that we have been effective in this instance in championing the patient voice and will continue pushing for access to new treatments for all patients in the target treatment population. Our next steps will be to assist in any way we can ahead of the newly agreed committee meeting to discuss zilucoplan with NICE and how we can obtain a positive recommendation for reimbursement on the NHS.