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AstraZeneca’s Ultomiris approved in the US

Marc Dunoyer

Ultomiris has been approved for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive, which represents 80% of people living with the disease.

“Since bringing forward thefirst 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, Ultomirishas demonstrated clinical benefit through 60 weeks, with treatment every eight weeks, compared to Soliris every two weeks,” says Marc Dunoyer, Chief Executive Officer, Alexion.

The first and only approval for a long-acting C5 complement inhibitor for the treatment of gMG

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. The diagnosed prevalence of gMG in the US is estimated at approximately 90,000.

The CHAMPION-MG Phase III trial

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, states the company.

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 Ultomiriswere upper respiratory tract infection and diarrhoea.

Regulatory submissions for Ultomirisfor the treatment of gMG are currently under review with multiple health authorities, including in the European Union (EU) and Japan.

Photo of Marc Dunoyer: AstraZeneca