Treatment with efgartigimod in patients with myasthenia gravis (MG) has resulted in significant and expeditious health-related quality-of-life (HRQoL) improvements, a new study published in the Journal of Neurology revealed.

Efgartigimod alfa, also known as efgartigimod, is a new antibody fragment and the first of its kind to be developed as a neonatal Fc receptor (FcRn) antagonist. Efgartigimod works by blocking FcRn and reducing the levels of circulating immunoglobulins, including the pathogenic autoantibodies.

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The research team conducted a phase 3 ADAPT study, a randomized, double-blind, placebo-controlled, multicenter study, carried out in 56 neuromuscular centers in North America, Europe, and Japan, which supported the regulatory approvals of efgartigimod. The study was conducted to review the HRQoL outcomes from individuals with AChR-Ab+ generalized MG (gMG) as found in the ADAPT study across the two cycles of treatment and follow-up. 

Myasthenia gravis (MG) is a chronic autoimmune disease that occurs when pathogenic immunoglobulin G (IgG) targets the neuromuscular junction, disrupting normal neuromuscular transmission. Patients with MG often experience significant disease-related burdens and decreased quality of life.

Study results suggest that efgartigimod was well tolerated and that participants treated with efgartigimod had statistically considerable improvements in the disease severity scores. Moreover, efgartigimod showed a rapid impact on the HRQoL, with considerable improvements as early as the first week of treatment in each treatment cycle (TC). The observed improvements were compatible across multiple measures with similar TC 1 and TC 2 effects. Furthermore, maximum benefit was noticed at week 4 or 5 in each TC, and the observed benefits with efgartigimod were found to be durable and reproducible.

The authors noted several limitations in the study. The assessment tools used may not have fully captured the impact of fatigue on gMG; second, the findings were based on clinical studies with limited data/follow-up and a specific dosing schedule; third, the design of the study, which evaluated the duration of effect after each treatment cycle, fourth, the study did not allow for the extrapolation of the HRQoL impact on long term real-life scenarios in which patients are treated based on the neurologic evaluation.

“Future studies using real-world data will be needed to further validate the benefit on HRQoL of efgartigimod in gMG,” the authors highlighted.

Reference

Saccà F, Barnett C, Vu T, et.al. Efgartigimod improved health-related quality of life in generalized myasthenia gravis: results from a randomized, double-blind, placebo-controlled, phase 3 study (ADAPT)J Neurol. 2023. Published online January 4, 2022 doi: 10.1007/s00415-022-11517-w