A real-world pharmacovigilance study of efgartigimod alfa in the FDA adverse event reporting system database

Objective Efgartigimod alfa, approved for treating generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive, has uncertain long-term safety in large populations This study analyzed adverse events (AEs) linked to efgartigimod alfa using data from the FDA Adverse Event Reporting System (FAERS). Methods We collected and analyzed efgartigimod alfa-related reports from the FAERS database from the first quarter of 2022 through the second quarter of 2024. Disproportionality analysis was used in data mining to quantify efgartigimod alfa-related AE signals. Results A total of 3,040 reports with efgartigimod alfa as the primary suspect and 12,487 AEs were retrieved from FAERS. The most frequently reported serious outcome was hospitalization (53.22%), and death occurred in 270 cases (8.88%). Disproportionality analysis detected 137 AE signals, with the most common in nervous system disorders (22.69%), general disorders and administration site conditions (16.90%), and infections and infestations (14.05%). Notably, in addition to infection-related AEs identified during clinical trials, this study detected unexpected signals, including inappropriate schedule of product administration (ROR 2.60, PRR 2.53, IC 1.34, EBGM 2.53) and nephrolithiasis (ROR 8.13, PRR 7.99, IC 2.99, EBGM 7.95). The median onset time of AEs was 81.0 days. Conclusion Our study provides a comprehensive assessment of the post-marketing safety of efgartigimod alfa and highlights the need for continued vigilance regarding infection-related adverse events. Additionally, the detection of inappropriate schedules of product administration underscores the importance of enhanced training and pharmacist involvement in medication management. Further research is warranted to explore the potential association between efgartigimod alfa and nephrolithiasis.