GEFURULIMAB significantly improved disease severity in adults with anti acetylcholine receptor antibody positive generalised myasthenia gravis compared with placebo, with clinical benefits emerging rapidly and persisting throughout 26 weeks of treatment.
The Phase III PREVAIL trial enrolled adults aged 18 years and older with anti acetylcholine receptor antibody positive generalised myasthenia gravis, Myasthenia Gravis Foundation of America class II–IV disease, and a Myasthenia Gravis Activities of Daily Living total score of five or greater. Conducted across 113 sites in 20 countries, the double blind, placebo-controlled study randomised 260 participants to receive either once weekly subcutaneous self administered gefurulimab (n=131) or placebo (n=129). Overall, 249 participants completed the 26 week treatment period.
The primary outcome assessed change from baseline in the Myasthenia Gravis Activities of Daily Living total score at week 26, while the key secondary outcome evaluated change in the Quantitative Myasthenia Gravis total score.
All primary and secondary end points were achieved with statistical significance. Improvements in the Myasthenia Gravis Activities of Daily Living score were observed within 1 week of treatment, while improvements in the Quantitative Myasthenia Gravis score were evident by week 4. Both measures remained improved through week 26.
At week 26, the least squares mean change from baseline in the Myasthenia Gravis Activities of Daily Living score was: −4.2 versus−2.6; treatment difference:−1.6; 95% CI:−2.4–−0.8; p<0.001. Improvements in the Quantitative Myasthenia Gravis score were also greater with gefurulimab than placebo:−4.5 versus−2.4; treatment difference:−2.1; 95% CI:−3.1–−1.1; p<0.001.
Among 405 patients screened, 145 did not meet eligibility criteria. The mean age of participants was 52.8 years, with females accounting for 60.4% of the study population. The incidence of adverse events was similar between treatment groups.
The most frequently reported treatment emergent adverse events with gefurulimab were injection site reactions, headache, back pain, and nasopharyngitis. No meningococcal infections were reported during the study.
The findings demonstrated that gefurulimab produced both rapid and sustained clinical improvement in adults with anti acetylcholine receptor antibody positive generalised myasthenia gravis while maintaining a safety profile comparable with placebo. The investigators concluded that the results support gefurulimab as a potential once weekly self administered treatment option for this patient population.
Reference
Gwathmey KG et al. Efficacy and Safety of Gefurulimab in Generalized Myasthenia GravisThe PREVAIL Phase 3 Randomized Clinical Trial. JAMA Neurology. 2026.
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