The US Food and Drug Administration (FDA) has granted approval to efgartigimod, a first-in-class, targeted therapy for adults with generalized myasthenia gravis (gMG) testing positive for the antiacetylcholine receptor (AChR) antibody.

Efgartigimod is an antibody fragment which decreases the IgG antibodies and inhibits IgG recycling process in patients with gMG. The drug was found to be effective and well-tolerated in the phase 3, randomized, placebo-controlled ADAPT trial. A total of 187 adults with gMG, irrespective of acetylcholine receptor antibody status, were recruited in the study. The trial participants had a Myasthenia Gravis-Activities of Daily Living score of at least 5 (> 50% nonocular) on a stable dose of at least one drug for MG. Over a duration of 26 weeks, 84 patients were randomized to receive efgartigimod 10 mg/kg, while 83 were given matching placebo. Efgartigimod led to a reduction in disease burden and improvement in strength and quality of life in patients with gMG across four scales. The findings from the trial were published in Lancet Neurology… (Medscape)