Efgartigimod Alfa Faces Emerging Safety Scrutiny in Post-Market Surveillance
Recent analyses of real-world data are raising new questions about the safety profile of efgartigimod alfa, a recently approved treatment for myasthenia gravis (MG). while clinical trials demonstrated efficacy and a manageable safety profile, emerging reports suggest potential for increased risk of urinary tract infections (UTIs) and other complications in patients receiving the drug, prompting closer pharmacovigilance.
Efgartigimod alfa, marketed as vyvgart®, represents a novel therapeutic approach for MG, an autoimmune neuromuscular disorder causing fluctuating muscle weakness. Approved in late 2021, it effectively works by reducing levels of disease-causing antibodies. Though, the transition from controlled clinical settings to broader patient populations frequently enough reveals previously undetected safety signals. This emerging scrutiny highlights the critical importance of ongoing post-market surveillance to fully characterize the drug’s risk-benefit profile and ensure patient safety.
Several studies are contributing to the evolving understanding of efgartigimod’s safety. A 2024 single-institution experience with 69 patients, published in Muscle Nerve, detailed patient selection, dosing schedules, treatment responses, and adverse events. Simultaneously, a retrospective analysis of data from the FDA Adverse event Reporting System (FAERS) revealed potential weight-related changes associated with anti-TNFalpha agents, prompting consideration of similar monitoring for efgartigimod due to its mechanism involving FcRn, a key player in antigen presentation (Baker et al., 2014).
Further compounding concerns, research published in Neurol Sci (2024) identified a high prevalence of comorbidities in MG patients, including a important proportion with pre-existing conditions that could potentially be exacerbated by efgartigimod or increase susceptibility to adverse events. Notably, studies (Bauza et al., 2018; Khamidullin et al., 2017) have suggested a potential link between UTIs and nephrolithiasis, raising concerns given the potential for increased UTI risk with efgartigimod.
Clinical efficacy and safety have been previously evaluated (Sivadasan & Bril,2023),and the drug’s technology has been explored (Dos Santos et al., 2022). However,ongoing vigilance is crucial. Healthcare professionals are advised to carefully monitor patients for signs of infection, weight changes, and other potential adverse events, and to report any suspected side effects to regulatory authorities. The long-term safety profile of efgartigimod alfa remains under investigation, and continued data collection and analysis will be essential to refine treatment guidelines and optimize patient care.
References
* Baker K, Rath T, Pyzik M, Blumberg RS. the role of FcRn in antigen presentation.Front Immunol. 2014;5:408. doi:10.3389/fimmu.2014.00408
* Bauza JL, Pieras EC, Grases F, et al. Urinary tract infection’s etiopathogenic role in nephrolithiasis formation. Med Hypotheses. 2018;118:34-35. doi:10.1016/j.mehy.2018.06.002
* Di Stefano V, Iacono S, Militello M, et al. Comorbidity in myasthenia gravis: multicentric, hospital-based, and controlled study of 178 Italian patients. Neurol Sci. 2024;45(7):3481-3494. doi:10.1007/s10072-024-07368-0
* Dos Santos JBR, Gomes RM, da Silva MRR. Abdeg technology for the treatment of myasthenia gravis: efgartigimod drug experience. Expert Rev clin immunol. 2022;18(9):879-888. doi:10.1080/1744666X.2022.2106972
* Khamidullin KR, Pushkarev AM, Tarasenko AI, Pavlov
