Efgartigimod, as a novel FcRn inhibitor, can improve neuromuscular transmission in patients with generalized myasthenia gravis (gMG) by accelerating the degradation of pathogenic IgG antibodies. Although clinical trials have confirmed its efficacy and good tolerability, its safety profile in the real-world setting requires comprehensive evaluation. This study utilized data from the U.S. FDA Adverse Event Reporting System (FAERS) to investigate and assess potential adverse event (AE) signals associated with Efgartigimod. The aim was to evaluate its real-world safety characteristics and provide evidence for clinical safe medication use and risk monitoring.

Reports listing Efgartigimod as the primary suspected drug were extracted from Q1 2020 to Q4 2023. After deduplication and terminology standardization, disproportionality analysis was conducted using three methods: Reporting Odds Ratio (ROR), Bayesian Confidence Propagation Neural Network (BCPNN), and Empirical Bayes Geometric Mean (EBGM), to identify significant signals at both the System Organ Class (SOC) and Preferred Term (PT) levels. Important Medical Events (IMEs) were also screened.

A total of 788 individual case safety reports involving 13 551 AEs were included. At the SOC level, the strongest signals were for nervous system disorders (ROR=5.12) and neoplasms (ROR=4.78). At the PT level, multiple strong signals were identified, including myasthenic crisis (ROR=2 269.29), bulbar paralysis (ROR=275.64), and thymoma (ROR=170.02). IME analysis further confirmed these events as high-priority safety concerns.

This study identified several potential new safety signals for Efgartigimod in the neurological, oncological, and immunological domains, suggesting that clinicians should remain vigilant about adverse reactions related to these organ systems and providing a basis for further targeted safety research.