RARE Daily

Ultomiris Approved in Europe for the Treatment of Adults with Generalized Myasthenia Gravis

September 23, 2022

The European Commission has approved Ultomiris as an add-on to standard therapy for the treatment of adult patients with generalized myasthenia gravis who are anti-acetylcholine receptor antibody positive.

This decision marks the first and only approval for a long-acting C5 complement inhibitor for the treatment of generalized myasthenia gravis in Europe.

Generalized myasthenia gravis (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 EU is estimated at approximately 89,000. Eighty percent of people with gMG are anti-acetylcholine receptor (AChR) antibody positive meaning they produce specific antibodies (anti-AChR) that bind to signal receptors at the neuromuscular junction, the connection point between nerve cells and the muscles they control. This binding activates the complement system, which is essential to the body’s defense against infection, causing the immune system to attack the neuromuscular junction, leading to inflammation and a breakdown in communication between the brain and the muscles.

gMG can occur at any age, but it most commonly begins for women before the age of 40 and for men after the age of 60. Initial symptoms may include slurred speech, double vision, droopy eyelids, and lack of balance; these can often lead to more severe symptoms as the disease progresses such as, impaired swallowing, choking, extreme fatigue and respiratory failure.

The approval by the European Commission follows the positive opinion of the Committee for Medicinal Products for Human Use and is based on results from the CHAMPION-MG phase 3 trial, which were published online in NEJM Evidence. In the 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.9 Additionally, in prolonged follow-up results from the open-label extension, clinical benefit of Ultomiris was observed through 60 weeks.

“As physicians, we see first-hand how gMG can have a debilitating impact on quality of life. Today’s approval is a major advancement for treating gMG in Europe, offering patients and physicians a new, long-acting treatment option which has shown reliable efficacy and sustained improvements in activities of daily living,” said Renato Mantegazza, professor at the Department of Neuroimmunology and Neuromuscular Diseases, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy, and CHAMPION-MG trial investigator.

In CHAMPION-MG, the safety profile of Ultomiris was comparable to placebo and consistent with that observed in phase 3 trials of Ultomiris in paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. The most common adverse reactions in patients receiving Ultomiris were diarrhea, upper respiratory tract infection, nasopharyngitis, and headache.

Ultomiris was approved in the United States in April 2022 and Japan in August 2022 for certain adults with gMG. Regulatory reviews are ongoing in additional countries.

Author: Rare Daily Staff

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