Previously, we shared that NICE had published individual initial draft guidance not recommending the use Ravulizumab or Efgartigimod on the NHS. A second committee meeting was scheduled in November to discuss the points raised in both documents but unfortunately, the company Alexion withdrew Ravulizumab from the appraisal process. Unusually, Efgartigimod has been scheduled to be assessed in a third committee meeting in May following further draft guidance not recommending it on the NHS. Normally a decision would be made by NICE after the second committee meeting, so this is most unusual.

We would like to emphasise that regardless of the guidance issued, those who have been taking part in the clinical trial for either medicine will continue to receive it until their neurologist decides otherwise.


Myaware quickly got in touch with Alexion to find out more about their decision to withdraw. The Alexion representative we spoke with assured that they are still looking to support and be involved with the myasthenia community, but the current landscape has made it difficult for them to proceed with the appraisal.


Argenx, the company responsible for Efgartigimod, have been in regular contact with myaware and we understand they are working to meet the terms of NICE’s second draft guidance. Currently, there remains concern regarding the degree to which Efgartigimod would reduce the need for maintenance IVIg in the target population of patients. In addition to this, the impact of carers has also been questioned despite myaware submitting a wealth of evidence on the matter.

We as a charity know through our members the impact this medicine has had on some of you and are committed to doing all we can to push for a positive outcome. We know treatment with Efgartigimod can be lifechanging. We also believe that the myasthenia community has patiently waited for a targeted therapeutic for a long time and this is a significant unmet need for patients.

We are regularly liaising with NICE and Argenx to encourage compromise on both sides so that Efgartigimod can become available for use on the NHS. We are hopeful that the third committee meeting, while not common, is a sign there is a path forward to a positive decision from NICE.

Read NICE’s second draft guidance for Efgartigimod

We are preparing our response to the second draft guidance for Efgartigimod. We want to ensure NICE hear from patients and that the need for new medicines is understood. If you have anything you would like to share, whether you have had Efgartigimod or not, please contact our research and partnerships officer Charlotte.