Myaware are beyond delighted to confirm that the targeted therapy for myasthenia gravis, Rozanolixizumab, has been approved for use via NHS England for refractory patients.

The charity has worked with our fellow stakeholders in the sector and NICE to capture the unmet need of generalised myasthenia gravis patients around the UK for over three years and we are overwhelmed to finally reach this momentous day.

Rozanolixizumab is an FcRn inhibitor that is administered via injection in six weeks cycles and has been proven to be highly effective in improving the symptoms of generalised myasthenia gravis. It is manufactured by the pharmaceutical company UCB and was initially developed here in the United Kingdom. It will be the first targeted medicine for gMG approved for reimbursement via NHS England.

This news is a first step in opening access for gMG patients all around the UK and offering much needed relief from the burden of the disease. We could not be happier for refractory patients and their families who will feel the direct benefit of this medicine.

Marc Smith, CEO myaware says: 'As a charity, we would like to extend our heartfelt thanks to the community who have supported us throughout the appraisals of many new medicines - and we have no intention of relaxing as we continue to fight for the betterment of patient access around the UK.

Without our members, we would be unable to authentically capture the reality of living with gMG effectively enough to convince decision makers of this unmet need.

We thank you and promise to continue to represent your voice.'

From the final draft guidance:

Rozanolixizumab, as an add-on to standard treatment, can be used as an option to treat generalised myasthenia gravis in adults who test positive for anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibodies. It can only be used if:
• the condition is classified as Myasthenia Gravis Foundation of America (MGFA) class 2 to 4a
• the condition is uncontrolled after 2 or more treatments, excluding acetylcholinesterase inhibitors, and
• intravenous immunoglobulin (IVIg) or plasma exchange (PLEX) would otherwise be offered, or has been tried and stopped because of side effects or because it did not work well enough.

To read the final draft guidance released by NICE, click here. To learn more about Rozanolixizumab, visit our treatment page here.