28 April 2022 07:00 BST
Ultomiris approved in the US for adults with generalised myasthenia gravis
First and only long-acting C5 complement inhibitor to demonstrate clinical improvement in patients with generalised myasthenia gravis
Ultomiris showed early effect and lasting improvement in activities of daily living and has potential to reduce treatment burden with dosing every 8 weeks.
Ultomiris (ravulizumab-cwvz) has been approved in the US for the treatment of adult patients with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive, which represents 80% of people living with the disease.1-5
The approval by the Food and Drug Administration (FDA) was based on positive results from the CHAMPION-MG Phase III trial, in which Ultomiris was superior to placebo in the primary endpoint of change from baseline in the Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score at Week 26, a patient-reported scale that assesses patients' abilities to perform daily activities.1
This FDA action marks the first and only approval for a long-acting C5 complement inhibitor for the treatment of gMG.
gMG is a rare, debilitating, chronic, autoimmune neuromuscular disease that leads to a loss of muscle function and severe weakness.6 The diagnosed prevalence of gMG in the US is estimated at approximately 90,000.7
Professor James F. Howard, Jr, MD, Department of Neurology at The University of North Carolina School of Medicine and lead primary investigator in the CHAMPION-MG trial said: “Despite recent advances, managing gMG is complex. Earlier intervention can preserve function and quality of life. This approval offers patients, including those with milder symptoms, a long-acting C5 inhibitor with early onset and reliable efficacy.”
Samantha Masterson, Chief Executive Officer, Myasthenia Gravis Foundation of America (MGFA), said: ” gMG takes a physical and emotional toll on those living with the disease. We are grateful for continued innovation and research into new treatment and dosing options to meet the needs of more patients and reduce the treatment burden. With the approval of Ultomiris, we're excited that MG patients now have another option to consider as part of their personalised treatment strategies that may offer more convenience and improve muscle weakness . “
Marc Dunoyer, Chief Executive Officer, Alexion, said: “Since bringing forward the first complement inhibitor, we've continued to listen to the community and focused innovation on the needs of gMG patients. We're proud to deliver on this commitment with today's approval. Ultomiris, the only long-acting C5 inhibitor, will benefit a broader range of patients, including those with milder symptoms. As presented at the 2022 American Academy of Neurology Annual Meeting, Ultomiris has demonstrated clinical benefit through 60 weeks, with treatment every eight weeks, compared to Soliris every two weeks.”
In the trial, the safety profile of Ultomiris was comparable to placebo and consistent with that observed in Phase III trials of Ultomiris in paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS). The most common adverse reactions in patients receiving Ultomiris were upper respiratory tract infection and diarrhoea.1
Results from the CHAMPION-MG trial were recently published online in NEJM Evidence and presented at the 2022 American Academy of Neurology Annual Meeting in April.
Regulatory submissions for Ultomiris for the treatment of gMG are currently under review with multiple health authorities, including in the European Union (EU) and Japan.