DOI: 10.1111/ene.16037 ISSN:

Advancements in Targeted Therapies for Generalized Acetylcholine Receptor Antibody Positive Myasthenia Gravis: Beginnings of a Paradigm Shift

Ali A. Habib, Christiane Schneider Gold
  • Neurology (clinical)
  • Neurology

Abstract

In recent years, a new era of biologic therapeutics has emerged for the treatment of generalized myasthenia gravis (gMG). Compared to traditional immune modulatory therapies (steroids, non‐steroidal immunosuppressive agents), these agents have more specific target engagement via inhibition of complement activation (eculizumab, ravulizumab, zilucoplan) or blockade of IgG recycling via the neonatal Fc receptor (FcRn; efgartigimod, rozanolixizumab). Phase 3 trials with each of these agents have shown rapid clinical improvement and good safety and tolerability. B‐cell targeting with rituximab was safe and well‐tolerated but with less impressive efficacy in two phase 2 studies. As a result, four new therapies are now approved for use (eculizumab, ravulizumab, efgartigimod, rozanolixizumab), another (zilucoplan) is in regulatory approval process and several others are in pivotal trials. A paradigm shift is afoot.

The challenge is figuring out where these therapies may fit in gMG treatment algorithms.

More from our Archive