Efficacy and Safety of Efgartigimod for Patients With Myasthenia Gravis in a Real‐World Cohort of 77 Patients

ABSTRACT Aims Efgartigimod, a first‐in‐class neonatal Fc receptor antagonist, is approved for generalized myasthenia gravis (gMG). Its safety and efficacy across MG subtypes remain unclear. Methods This single‐center real‐world study (September 2023–July 2024) analyzed patients from an MG registry study in China. The primary efficacy outcome is the mean MG‐ADL score changes from baseline at weeks 4, 8, and 12, analyzed via generalized estimating equations. Safety was assessed by adverse events. Results Among 77 patients (mean age 56.1 ± 15.2 years; 59.7% male), 76 completed at least one treatment cycle (20 completed 2 cycles; 1 completed 3 cycles). After efgartigimod treatment, MG‐ADL scores decreased significantly by week 4 (mean difference −6.4, 95% CI −7.2 to −5.6, p < 0.001), sustaining through week 12 (−6.9, −7.8 to −6.1, p < 0.001). After the second cycle, MG‐ADL scores at week 12 trended lower than the first cycle (mean difference: −0.8, 95% CI: −2.0 to −0.5, p = 0.061). Efficacy was consistent across MGFA classes and thymoma status. In refractory patients, efgartigimod reduced MG‐ADL scores ( p < 0.001). Adverse events occurred in 3.9% (3/77). Conclusion Efgartigimod safely improved MG‐ADL scores and reduced steroid use across MG subtypes, with sustained efficacy through multiple treatment cycles. These findings support its potential when conventional therapies fail.