The treatment is indicated for gMG patients who are anti-acetylcholine receptor (AChR) antibody-positive.
In July, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) provided a positive opinion recommending a grant of marketing authorisation for Ultomiris.
The EC granted the approval based on data from the randomised, international, placebo-controlled, double-blind, multicentre Phase III CHAMPION-MG trial that assessed the safety and efficacy of Ultomiris in adult gMG patients.
On the trial’s primary endpoint of change from baseline in the Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score at week 26, Ultomiris was found to be superior to placebo.
A subject-reported scale, MG-ADL evaluates the ability of participants to carry out day-to-day activities.
According to the extended follow-up findings from the open-label extension, Ultomiris demonstrated clinical benefit through 60 weeks.
A long-acting C5 complement inhibitor, Ultomiris provides quick and lasting complement inhibition.
It obtained approvals in the US and Japan for use in some adult patients with gMG.
Regulatory reviews of the therapy are progressing in further regions.
Alexion CEO Marc Dunoyer said: “This approval in Europe of the first and only long-acting C5 inhibitor is an important step towards realising our vision of improving the lives of people living with gMG and increasing access to Ultomiris worldwide.
“We’re proud to offer a new treatment option that provides more convenience in dosing and has shown clinical benefit in a broader range of patients, including those who remain symptomatic despite their initial standard of care treatment.”
In September last year, the EU granted expanded approval for Ultomiris to treat paroxysmal nocturnal haemoglobinuria in children and adolescents.