Table 1

New and emerging treatments for myasthenia gravis by therapeutic class

ClassMechanism of actionDrug nameRouteRecommendationsStudies*
B cell depletionTarget CD20, lead to B cell depletionRituximab: chimeric monoclonal antibodyIntravenousEarly consideration in MuSK+MG, third line for AChR+gMGBlinded prospective study for MuSK+MG and retrospective studies. Phase 2 BeatMG trial
Target CD19, lead to B cell depletionInebilizumab: humanised monoclonal antibodyIntravenousTBDPhase 3 randomised controlled trial of AChR+gMG and MuSK+MG ongoing
Complement inhibitionBlock cleavage of C5 into C5a and C5b and prevent formation of membrane attack complexEculizumab: humanised monoclonal antibodyIntravenousRefractory AChR+gMGPhase 3 REGAIN trial completed. FDA approval for AChR+gMG in 2017
Ravulizumab: humanised monoclonal antibodyIntravenousAChR+gMGPhase 3 CHAMPION study completed. FDA approval for AChR+gMG in 2022
Zilucoplan: 15 amino acid peptideSubcutaneousTBDPhase 3 RAISE trial AChR+gMG completed, and primary and key secondary endpoints met
Neonatal Fc receptor inhibitionBind and block neonatal Fc receptor, reduce IgG recycling and level of IgG antibodiesEfgartigimod alfa: Fc fragment of human IgG1IntravenousAChR+gMGADAPT trial completed. FDA approval for AChR+gMG in 2021
Rozanolixizumab: humanised IgG4SubcutaneousAChR+MG and MuSK+MG are enrolledPhase 3 MycarinG study completed, and primary and key secondary endpoints met
Batoclimab: humanised IgG1SubcutaneousTBDPhase 3 randomised controlled trial of generalised myasthenia gravis ongoing
Nipocalimab: humanised IgG1IntravenousTBDPhase 3 randomised controlled trial of generalised myasthenia gravis ongoing
Interleukin 6 inhibitionBlock interleukin 6 receptor, reduce proinflammatory T cell and B cell differentiationSatralizumab: humanised IgG2SubcutaneousTBDPhase 3 randomised controlled trial of AChR+gMG ongoing
Chimeric antigen receptor T cell treatmentModify T cells and their regulation of B cell maturationDescartes-08SubcutaneousTBDPhase 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.