Post-Marketing Safety Concerns with Efgartigimod alfa: A Pharmacovigilance Analysis Based on the Food and Drug Administration Adverse Event Reporting System Database.

AIM: Efgartigimod alfa (EA) is a novel US Food and Drug Administration (FDA) approved neonatal Fc receptor-targeting drug; however, its real-world adverse event (AE) profile remains underexplored.

METHODS

AE reports primarily related to EA were retrieved from the US FDA Adverse Event Reporting System database for the fourth quarter of 2021 to the third quarter of 2024. Disproportionality analysis using Reporting Odds Ratio (ROR), Proportional Reporting Ratio (PRR), Bayesian Confidence Propagation Neural Network, and Multi-item Gamma Poisson Shrinker algorithms was employed to detect signals of AEs.

RESULTS

Our study processed 3,182 AE reports related to EA, revealing 57 signals that met the criteria of the ROR, PRR, Bayesian Confidence Propagation Neural Network, and Multi-item Gamma Poisson Shrinker algorithms across 14 system organ classes. Notably, the most significant signal in the System Organ Class was "Surgical and medical procedures", whereas the most significant signal in Preferred Term was "Bulbar Palsy". Some unexpected over-the-counter AEs, including falls, choking, sepsis, nephrolithiasis, and atrial fibrillation, were also observed. The median onset time of EA-related AEs was 101.5 d (interquartile range 27-260). The AE risk model associated with EA should be referred to as "early failure", with the likelihood of AEs decreasing over time.

CONCLUSION

This study highlights the potential AEs and risks associated with the clinical use of EA; the analysis provides significant evidence regarding the clinical safety of EA.