AstraZeneca’s Gefurulimab Achieves Phase III Success In Generalized Myasthenia Gravis, Meeting All Endpoints

AstraZeneca recently announced updates on a global Phase III clinical trial, gefurulimab demonstrated positive high-level results in adults with anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG). The randomized, double-blind, placebo-controlled study showed that the drug met its primary and all secondary endpoints. 

Kelly Gwathmey, MD, Associate Professor of Neurology, Chief of Neuromuscular Division, Virginia Commonwealth University, Richmond, VA, Vice Chair of the MGFA Medical & Scientific Advisory Council and principal investigator in the trial, said, “Rapidly fluctuating symptoms and the unpredictable disability associated with gMG can affect nearly every aspect of a patient's life, making early intervention and sustained disease control a critical treatment goal. A once-weekly, self-administered C5 treatment option would offer patients greater convenience and independence in managing their condition, empowering them to have more control over their therapy.”

Patients receiving gefurulimab experienced a statistically significant and clinically meaningful improvement in Myasthenia Gravis Activities of Daily Living (MG-ADL) total score at week 26 compared to those receiving placebo. Generalized myasthenia gravis is a rare, chronic autoimmune neuromuscular disorder marked by severe muscle weakness, fatigue, and difficulty in performing everyday functions such as swallowing, speaking, or breathing. 

Marc Dunoyer, Chief Executive Officer, Alexion, AstraZeneca Rare Disease, commented, “Building on Alexion’s pioneering leadership in gMG, these positive results from the PREVAIL Phase III trial demonstrate the potential for gefurulimab to offer rapid and sustained disease control for this patient community. These data, reflecting patient participation across 20 countries, reinforce the established safety profile and efficacy of C5 inhibition and show the potential for gefurulimab as a first line biologic, with the convenience of a self-administered option.”

Symptoms often worsen over time and can lead to life-threatening complications like respiratory failure. Gefurulimab was well-tolerated during the trial, with a safety profile consistent with previous C5 inhibitors and no new safety concerns. Full data will be presented at an upcoming medical meeting and submitted to global regulatory authorities to support potential approval.