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Drugs. 2022 Feb;82(3):341-348. doi: 10.1007/s40265-022-01678-3.
Efgartigimod (efgartigimod alfa-fcab, Vyvgart) is a first-in-class neonatal Fc receptor antagonist being developed by argenx for the treatment of autoimmune diseases including myasthenia gravis. In December 2021, intravenous efgartigimod received its first approval in the USA for the treatment of generalized myasthenia gravis in adults who are anti-acetylcholine receptor (AChR) antibody positive. Intravenous efgartigimod has also been evaluated for generalized myasthenia gravis in various other countries, with the agent subsequently approved in Japan in January 2022 for generalized myasthenia gravis patients regardless of antibody status and in preregistration stage in the EU. Several clinical studies of intravenous and subcutaneous formulation of efgartigimod are also being investigated for other autoimmune diseases including bullous pemphigoid, chronic inflammatory demyelinating polyradiculoneuropathy, immune thrombocytopenia, autoimmune myositis and pemphigus. This article summarizes the milestones in the development of efgartigimod leading to this first approval for generalized myasthenia gravis.
依氟鸟氨酸（efgartigimod alfa-fcab，Vyvgart）是一种新型的新生儿 Fc 受体拮抗剂，由 argenix 公司研发，用于治疗包括重症肌无力在内的自身免疫性疾病。2021 年 12 月，静脉注射依氟鸟氨酸在美国首次获批，用于治疗乙酰胆碱受体（AChR）抗体阳性的成年全身性重症肌无力患者。静脉注射依氟鸟氨酸也在其他国家评估用于治疗全身性重症肌无力，该药随后于 2022 年 1 月在日本获批，用于治疗无论抗体状态如何的全身性重症肌无力患者，并在欧盟处于预注册阶段。几项静脉注射和皮下制剂的依氟鸟氨酸的临床试验也正在研究用于治疗其他自身免疫性疾病，包括大疱性类天疱疮、慢性炎症性脱髓鞘性多发性神经病、免疫性血小板减少症、自身免疫性肌炎和天疱疮。本文总结了导致依氟鸟氨酸首次获批用于治疗全身性重症肌无力的关键开发里程碑。
