Efgartigimod Alfa in Generalised Myasthenia Gravis: A Profile of Its Use

Intravenous efgartigimod alfa (also known as efgartigimod alfa-fcab in the USA; Vyvgart (R)) is the first neonatal Fc receptor antagonist approved in several countries worldwide, including the USA and EU for the treatment of generalised myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) antibody positive, and in Japan for the treatment of gMG regardless of antibody status. In the double-blind, placebo-controlled phase 3 ADAPT trial in patients with gMG, efgartigimod alfa significantly and rapidly reduced disease burden and improved muscle strength and quality of life compared with placebo. The clinical benefits of efgartigimod alfa were durable and reproducible. Furthermore, in an interim analysis of the ongoing open-label phase 3 ADAPT+ extension trial, efgartigimod alfa provided consistent clinically meaningful improvements in patients with gMG. Efgartigimod alfa was generally well tolerated, with most adverse events being mild to moderate in severity. Plain Language Summary Generalised myasthenia gravis (gMG) is a chronic, autoimmune neuromuscular disorder that can significantly impair quality of life. Several novel targeted therapeutic approaches have emerged to provide faster onset of action compared with conventional immunosuppressive therapy, favourable tolerability profile and the potential for a sustained disease control for patients with gMG. Intravenous efgartigimod alfa (also known as efgartigimod alfa-fcab in the USA; -Vyvgart (R)) is the first neonatal Fc receptor antagonist approved in several countries worldwide, including the USA and EU for the treatment of gMG in adults who are anti-acetylcholine receptor (AChR) antibody positive, and in Japan for the treatment of gMG regardless of antibody status. In the pivotal clinical trial in patients with gMG, efgartigimod alfa rapidly reduced disease burden and improved muscle strength and quality of life. The beneficial effects of efgartigimod alfa occurred early and were durable and reproducible. Longer term, efgartigimod alfa provided consistent clinically meaningful improvements in patients with gMG. Efgartigimod alfa is generally well tolerated, with most adverse events being mild to moderate in severity. Thus, efgartigimod alfa is a novel, effective and generally well-tolerated treatment option for patients with gMG.

Automated summary

Efgartigimod alfa is a novel and effective treatment for generalised myasthenia gravis that has shown significant and durable clinical benefits in clinical trials.