A New Era in the Treatment of Myasthenia Gravis: Six New Medications in The Last 6 Years

This article presents a summary of the new medications approved for generalized myasthenia gravis (gMG) since 2017. Pivotal clinical trials that led to the approval of these medications and their open-label extension studies are reviewed. We also provide information on healthcare cost when available. Lastly, we propose an approach to selecting therapies.

Six new medications have been approved for acetylcholine receptor antibody positive gMG. These include the complement inhibitors eculizumab, ravulizumab, and zilucoplan and the neonatal Fc receptor blockers efgartigimod, efgartigimod and hyaluronidase, and rozanolixizumab. The latter is also approved for muscle-specific kinase gMG. The Myasthenia Gravis Activities of Daily Living (MG-ADL) has become the most commonly used primary outcome measure in gMG clinical trials.

All medications have shown safety and efficacy as measured by clinically meaningful changes in the MG-ADL. The medications differ in the need for meningococcal vaccination, the route and frequency of administration, and timing of treatments. Comparison studies are lacking and therefore there is limited evidence to guide the selection of therapy.