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The management of myasthenia gravis (MG) has been improved due to immunotherapy advances, but 20% of individuals with MG are refractory to the conventional therapy, and the need for novel biological drugs remains. The Japanese clinical guidelines for MG published in May 2022 include the concept that treatment is often lifelong and should aim to maintain a sufficient quality of life and mental health. We provide an overview of the therapeutic strategy for generalized MG in Japan, in comparison with the international consensus. We summarize the clinical efficacy, safety, and tolerability of efgartigimod, the first approved anti-neonatal Fc receptor inhibitor for MG. A phase III study showed that efgartigimod was well-tolerated and efficacious in patients with generalized MG. Efgartigimod is a promising biological drug for patients with moderate to severe generalized MG with or without anti-acetylcholine receptor antibodies in Japan.

Efgartigimod for generalized myasthenia gravis with or without anti-acetylcholine receptor antibodies: a worldwide and Japanese perspective