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AstraZeneca’s Ultomiris recommended for approval in the EU

Marc Dunoyer

Ultomiris (ravulizumab) has been recommended for marketing authorisation in the European Union (EU) as an add-on to standard therapy for the treatment of adult patients with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive.

If authorised, Ultomiris would be the first and only approved long-acting C5 complement inhibitor for the treatment of AChR antibody-positive gMG in the EU.

“This recommendation is a major milestone in our commitment to help a broader range of gMG patients, including those with milder symptoms, and expand access to Ultomiris. As we listen to the patient community, we’re focused on understanding and meeting the needs of gMG patients, providing them with effective and accessible treatment options, and the positive opinion is a great step forward,” says Marc Dunoyer, Chief Executive Officer, Alexion.

The CHAMPION-MG Phase III trial

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency based its positive opinion on results from the CHAMPION-MG Phase III trial.

In this trial, 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.

gMG is a rare, debilitating, chronic, autoimmune neuromuscular disease that leads to a loss of muscle function and severe weakness.2 The diagnosed prevalence of gMG in the EU is estimated at approximately 89,000.

In CHAMPION-MG, 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 drug reactions are diarrhoea, upper respiratory tract infection, nasopharyngitis and headache.

The CHMP recommended approval as an add-on to standard therapy for the treatment of adult patients with gMG who are AChR antibody-positive.

Ultomiris was approved in the US for adults with gMG who are AChR antibody-positive in April 2022, and regulatory reviews are ongoing in additional countries, including Japan.

Photo of Marc Dunoyer: AstraZeneca