Class | Mechanism of action | Drug name | Route | Recommendations | Studies* |
B cell depletion | Target CD20, lead to B cell depletion | Rituximab: chimeric monoclonal antibody | Intravenous | Early consideration in MuSK+MG, third line for AChR+gMG | Blinded prospective study for MuSK+MG and retrospective studies. Phase 2 BeatMG trial |
Target CD19, lead to B cell depletion | Inebilizumab: humanised monoclonal antibody | Intravenous | TBD | Phase 3 randomised controlled trial of AChR+gMG and MuSK+MG ongoing | |
Complement inhibition | Block cleavage of C5 into C5a and C5b and prevent formation of membrane attack complex | Eculizumab: humanised monoclonal antibody | Intravenous | Refractory AChR+gMG | Phase 3 REGAIN trial completed. FDA approval for AChR+gMG in 2017 |
Ravulizumab: humanised monoclonal antibody | Intravenous | AChR+gMG | Phase 3 CHAMPION study completed. FDA approval for AChR+gMG in 2022 | ||
Zilucoplan: 15 amino acid peptide | Subcutaneous | TBD | Phase 3 RAISE trial AChR+gMG completed, and primary and key secondary endpoints met | ||
Neonatal Fc receptor inhibition | Bind and block neonatal Fc receptor, reduce IgG recycling and level of IgG antibodies | Efgartigimod alfa: Fc fragment of human IgG1 | Intravenous | AChR+gMG | ADAPT trial completed. FDA approval for AChR+gMG in 2021 |
Rozanolixizumab: humanised IgG4 | Subcutaneous | AChR+MG and MuSK+MG are enrolled | Phase 3 MycarinG study completed, and primary and key secondary endpoints met | ||
Batoclimab: humanised IgG1 | Subcutaneous | TBD | Phase 3 randomised controlled trial of generalised myasthenia gravis ongoing | ||
Nipocalimab: humanised IgG1 | Intravenous | TBD | Phase 3 randomised controlled trial of generalised myasthenia gravis ongoing | ||
Interleukin 6 inhibition | Block interleukin 6 receptor, reduce proinflammatory T cell and B cell differentiation | Satralizumab: humanised IgG2 | Subcutaneous | TBD | Phase 3 randomised controlled trial of AChR+gMG ongoing |
Chimeric antigen receptor T cell treatment | Modify T cells and their regulation of B cell maturation | Descartes-08 | Subcutaneous | TBD | Phase 1/2 trial ongoing |
FDA=US Food and Drug Administration; AChR+MG=acetylcholine receptor antibody positive myasthenia gravis; AChR+gMG=acetylcholine receptor antibody positive generalised myasthenia gravis; MuSK+MG=muscle specific tyrosine kinase antibody positive myasthenia gravis; TBD=to be determined.
*Study names: BeatMG=B Cell Targeted Treatment in Myasthenia Gravis; REGAIN=Safety and Efficacy of Eculizumab in Refractory Generalised Myasthenia Gravis; CHAMPION=Safety and Efficacy Study of Ravulizumab in Adults With Generalised Myasthenia Gravis; RAISE=Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalised Myasthenia Gravis; ADAPT=An Efficacy and Safety Study of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalised Muscle Weakness: MycarinG=A Study to Evaluate Rozanolixizumab in Study Participants With Generalised Myasthenia Gravis.