Introduction: Myasthenia gravis is characterized by fluctuating muscle weakness that improves with rest and worsens with effort or throughout the day.
Areas covered: Efgartigimod is a human IgG1-derived Fc fragment modified at five residues to increase its affinity for the neonatal Fc receptor by Abdeg technology. Thus, efgartigimod binds to the neonatal Fc receptor and decreases the levels of IgG, including autoantibodies of this isotype. For acetylcholine receptor (AChR) antibody-positive patients, efgartigimod had a higher proportion of MG-ADL responders than placebo in the first treatment cycle. The mean changes of multiple outcomes from baseline were better for efgartigimod than placebo from weeks 1 to 7 in the first treatment cycle. The decrease of IgG and AChR autoantibodies was 61.3% and 57.6% one week after the first treatment cycle ends, respectively. The most common adverse events were headache, nasopharyngitis, nausea, and diarrhea, which occurred in the same proportion in the efgartigimod and placebo groups. Urinary and upper respiratory tract infections were twice as frequent in efgartigimod-treated patients.
Expert opinion: Efgartigimod was efficacious and safe for generalized myasthenia patients with AChR antibody-positive patients. These findings need to be confirmed in AChR antibody-negative patients, and long-term safety studies are currently ongoing.
Keywords: Efgartigimod; generalized myasthenia gravis; neonatal Fc receptor antagonist; review; treatment.
Myasthenia gravis patients have a high level of autoantibodies, which can cause fluctuating muscle weakness. In this regard, efgartigimod is a new drug approved to treat myasthenia gravis that decreases antibody levels and symptom improvement. Furthermore, this drug was safe for these patients.[Figure: see text].