RNS -24 Jul 2025 08:43
Positive high-level results from a global, randomised, double-blind, placebo-controlled Phase III trial in adults with anti-acetylcholine receptor (AChR) antibody-positive (Ab+) generalised myasthenia gravis (gMG) showed that gefurulimab met its primary and all secondary endpoints. Data demonstrated a statistically significant and clinically meaningful improvement from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) total score at week 26 compared to placebo.
gMG is a rare, debilitating, chronic, autoimmune neuromuscular disease that leads to a loss of muscle function and severe weakness.1 Those living with gMG may initially experience slurred speech, double vision, droopy eyelids and weakness, with symptoms becoming more severe as the disease progresses, including extreme fatigue, difficulty swallowing, choking and respiratory failure.2,3
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."
Marc Dunoyer, Chief Executive Officer, Alexion, AstraZeneca Rare Disease, said: "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."
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