Research Progress in Pharmacological Targeted Therapy for Myasthenia Gravis.

Myasthenia gravis (MG) is the prototypical disorder of the neuromuscular junction (NMJ) mediated by antibodies, producing fluctuating, fatigable skeletalmuscle weakness. 1 In the acetylcholine receptor (AChR)-Ab-positive subtype (≈80-85% of generalized cases), IgG1 and IgG3 antibodies fix complement to cause postsynaptic injury through the membrane attack complex (MAC), accelerate AChR internalization and block acetylcholine (ACh) binding. In muscle-specific kinase (MuSK)-Ab-positive MG, IgG4 antibodies disrupt the agrin- low-density lipoprotein receptor-related protein 4 (LRP4)-MuSK-Dok7 signalling complex required for AChR clustering, largely independently of complement. Pathogenesis of LRP4-Ab-positive and double-seronegative MG remains only partially elucidated, with possible roles for additional autoantibodies (agrin, cortactin, ryanodine receptors). 2 , 3 Additionally, CD4+ T helper cells can secrete proinflammatory cytokines (eg, interleukin-2, interferon-γ) to activate B lymphocytes, thereby facilitating autoantibody biosynthesis and exacerbating the cascade of autoimmune responses.

MG management follows a stepwise hierarchy. First-line treatment combines cholinesterase inhibitors and corticosteroids, with maintenance non-steroidal immunosuppressants (azathioprine, mycophenolate mofetil, tacrolimus); intravenous/subcutaneous immunoglobulin and plasma exchange are used for crises and in selected patients for chronic maintenance, and thymectomy in selected AChR-Ab-positive patients. 2 , 4–8 These regimens are limited by delayed onset (6–12 months for azathioprine and longer for mycophenolate mofetil), steroid-related morbidity, and suboptimal disease control. 9–14 Second-line and later-line targeted therapies, which include B/T cell-targeted agents, complement inhibitors, FcRn antagonists, cytokine-targeted agents and emerging cellular therapies, have therefore been developed for refractory, steroid-dependent or intolerant patients and are increasingly considered for earlier introduction in active generalized disease.

Over the past few years, as research into the pathogenesis of MG has become increasingly in-depth, most notably with the gradual clarification of the crucial driving effect of dysregulated Bcell activation on disease onset and progression, targeted therapy has evolved into a prominent research frontier in the MG field. 3 An array of innovative biologics (eg, monoclonal antibody-based agents) and cellular therapeutic technologies (eg, chimeric antigen receptor T-cell [CAR-T] therapy) has been successively developed, offering novel approaches to overcoming the limitations of conventional therapeutic regimens for MG. 15 , 16 Targeted biologics and cellular therapies also introduce class-specific adverse events: reactions at the infusion or injection site, hypogammaglobulinemia and reactivation of latent infection with B cell depletion; increased Neisseria meningitidis risk with complement inhibitors; hypoalbuminemia and dyslipidemia with selected FcRn antagonists; neutropenia, transaminitis and rare gastrointestinal perforation with interleukin-6 receptor (IL-6R) inhibitors (predominantly tocilizumab); and cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome (ICANS) with cellular therapies. These risks underscore the need for careful risk-benefit assessment and long-term pharmacovigilance.

Several aspects of MG targeted therapy are now well established. The pathogenic roles of postsynaptic autoantibodies, complement-mediated NMJ injury, FcRn-dependent IgG recycling and dysregulated B cell activation are sufficiently clarified to support rational drug design. 2 , 3 , 15 Phase III evidence has firmly established complement inhibitors (eculizumab, ravulizumab, zilucoplan) and FcRn antagonists (efgartigimod, rozanolixizumab, nipocalimab) in AChR-Ab-positive generalized myasthenia gravis (gMG), and rozanolixizumab in MuSK-Ab-positive disease, 17–23 and these strategies are incorporated into international consensus guidance. 5 Observational data support a disproportionately greater benefit of B cell depletion in MuSK-Ab-positive MG. 24–26 Unresolved issues include the optimal positioning of biologics within the treatment algorithm, the opposing rituximab results in BeatMG and RINOMAX, 27 , 28 limited subtype-tailored evidence (MuSK-, LRP4-, double-seronegative, ocular, juvenile, very-late-onset MG), immature long-term safety data, and the absence of predictive biomarkers and head-to-head trials.

Existing narrative reviews are typically confined to a single mechanistic class. 15 , 16 This review adds value in three respects. First, by considering all major mechanistic classes side by side, with their routes and dosing regimens, a horizontal comparison is provided. Second, recent evidence is incorporated: Phase III nipocalimab (Vivacity-MG3), 23 the pediatric eculizumab study, 29 the inebilizumab Phase III MINT trial, 30 the iscalimab Phase II results, 31 the subcutaneous efgartigimod ADAPT-SC programme, 32 real-world telitacicept data, 33 and a Bayesian network meta-analysis of innovative monoclonal antibodies. 34 Third, this evidence is linked to the practical clinical questions of biologic selection by mapping data onto patient-level dimensions (age, time from onset, severity, antibody subtype).

This narrative review summarizes advances in pharmacological targeted therapy for MG, focusing on B/T cell-targeted agents, complement inhibitors, FcRn inhibitors, and cytokine-targeted agents, with emphasis on mechanisms, efficacy, safety, and clinical positioning. Additionally, it discusses the developmental prospects of other newly emerging therapeutic modalities, aiming to provide evidence-based insights for optimizing clinical management of MG and defining future research priorities. The characteristics of the included studies are summarized in Supplementary Material 1 . Figure 1 shows the molecular targets and administration routes of each drug.

The pivotal Phase II and Phase III trials of agents directed at B and T cells, complement inhibitors, FcRn antagonists and IL-6R inhibitors covered here did not enrol participants who were naive to treatment; most patients were already on oral corticosteroids (either alone or alongside azathioprine, mycophenolate, tacrolimus, methotrexate or cyclosporine), with prior plasma exchange or intravenous immunoglobulin (IVIG) common. These agents were therefore assessed as add-on therapy in disease that was active, refractory or dependent on steroids. To prevent confounding by the slow onset of conventional immunosuppression (six to twelve months for azathioprine, a year or more for mycophenolate), protocols imposed a stable run-in (typically thirty days or more for corticosteroids; weeks to months for nonsteroidal immunosuppressants) and required these doses to remain unchanged throughout the randomised period, with the placebo-controlled design balancing any residual background effect between arms. The caveat carries more weight when interpreting open-label, retrospective and real-world data on telitacicept, tocilizumab, blinatumomab, CAR-T and Hematopoietic Stem Cell Transplantation (HSCT).

Targeting B Cells or Plasma Cells

Rituximab

Rituximab was the first anti-CD20 monoclonal antibody applied in the treatment of MG. It is a chimeric human-murine monoclonal antibody. Its mechanism of action mainly involves the specific depletion of CD20+ B cells. This depletion is mediated via complement-dependent cytotoxicity (CDC), antibody-dependent cellular cytotoxicity (ADCC) and other pathways. 35 , 36 CD20 is highly expressed on mature B cells and memory B cells, thus rituximab can effectively eliminate these cell subsets; in contrast, B cell progenitors and plasma cells residing in the bone marrow and secondary lymphoid organs are barely affected due to the lack of CD20 expression. 37 , 38

Rituximab carries the broadest clinical experience across AChR- and MuSK-Ab-positive disease. 25 , 26 , 39 The BeatMG Phase II study (n=51 over 52 weeks) fell short on its composite primary endpoint, which required both a 75% or greater prednisone reduction and an Myasthenia Gravis Composite (MGC) increase no larger than two points. 27 RINOMAX, by contrast, enrolled newly diagnosed AChR-Ab-positive patients within twelve months of onset and saw a greater proportion of rituximab recipients reach minimal disease manifestations. 28 In a blinded multicentre prospective cohort of MuSK-MG, Burns et al recorded minimal manifestation (MM)-or-better Post-Intervention Status in 58% of rituximab-treated patients against 16% of controls. 24 Divergent rituximab outcomes in MG reflect differences in patient selection, antibody subtypes, disease duration and endpoint stringency, not inconsistent drug effects. BeatMG enrolled long-standing steroid-dependent AChR-positive patients, while RINOMAX focused on newly diagnosed disease targeting minimal manifestations, and the Burns cohort included only MuSK-positive cases. Biology offers a parsimonious explanation: MuSK-MG depends on IgG4 antibodies generated by short-lived CD20-expressing plasmablasts that anti-CD20 therapy readily eliminates, whereas IgG1 and IgG3 anti-AChR antibodies are largely produced by long-lived bone-marrow plasma cells lacking CD20 and therefore inaccessible to direct depletion. 40 , 41 Tolerability is generally good, with mild infusion reactions and a modest rise in respiratory and urinary infections. 25 Three evidence tiers thus emerge: a firmly established role in MuSK-Ab-positive disease (72–82% achieving MM or better 24 , 25 , 42 ); supportive standing when introduced early in newly diagnosed AChR-Ab-positive disease via RINOMAX; 28 and an unresolved position against stringent for sparing steroids endpoints in longstanding AChR-Ab-positive disease following BeatMG. 27 The dedicated meta-analysis by Chayanopparat et al (twelve studies, 111 MuSK patients) pooled to 82% achieving MM or better, 56% complete or pharmacological remission, an average glucocorticoid reduction of 17.15 mg, and a single serious adverse event. 42

Inebilizumab

Inebilizumab is a humanized, afucosylated IgG1κ monoclonal antibody that targets and depletes CD19+ B cells, including plasmablasts and plasma cells responsible for generating autoantibodies specific to MG. 43–46 This breadth of target expression, extending beyond the CD20+ mature B cell pool depleted by anti-CD20 agents to encompass earlier pro-B cells and plasmablasts that secrete antibody and short-lived plasma cells, provides the mechanistic rationale for its evaluation in autoimmune diseases mediated by antibodies, including MG.

This agent was approved in June 2020 for the treatment of aquaporin-4 antibody (AQP4)-positive neuromyelitis optica spectrum disorder (NMOSD). 47 , 48 Clinical trials involving NMOSD patients have demonstrated that inebilizumab is well tolerated, with mild to moderate adverse effects consistent with the safety profile of other B-cell depleting agents. Findings from a Phase III clinical study (MINT) indicated that compared with the placebo group, inebilizumab yielded greater clinical improvement at week 26, with larger mean reductions in the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale score (−4.2 vs −2.2 points) and in the Quantitative Myasthenia Gravis (QMG) score (−4.8 vs −2.3 points), with the primary and key secondary endpoints met. Adverse events reported in the inebilizumab group included arthralgia, diarrhea, headache and hypertension, with serious adverse events less frequent than in the placebo group (8.4% vs 13.4%). 30 Several converging lines underpin the agent’s clinical positioning. Earlier Phase I work in healthy volunteers and in patients with systemic sclerosis or relapsing multiple sclerosis defined inebilizumab’s pharmacokinetic profile and showed deep, sustained CD19+ depletion at therapeutic exposures. 43 , 44 The N-MOmentum programme in AQP4-IgG-positive NMOSD reduced attack risk and held up across four-plus years of follow-up. 45 , 46 Within MINT itself, AChR-Ab-positive responders retained benefit through week 52, and 87.4% of treated participants had been brought down to a prednisone dose of 5 mg/day or less by week 26, a clinically meaningful steroid-sparing signal. 30 Food and Drug Administration (FDA) approval followed on 11 December 2025, covering both AChR-Ab-positive and MuSK-Ab-positive gMG and making this the third autoimmune indication for the agent, while European Medicines Agency (EMA) and Pharmaceuticals and Medical Devices Agency (PMDA) evaluations were still in progress. A Bayesian network meta-analysis additionally placed B-cell-depleting strategies favourably overall. 34

Belimumab

Belimumab, a recombinant human IgG1λ monoclonal antibody, exerts neutralization activity against soluble B cell activating factor (BAFF, otherwise designated as B lymphocyte stimulator [BLyS]). BAFF occurs in two discrete isoforms: membrane-bound and secreted variants, both produced by non-B cellular subsets such as monocytes, dendritic cells, and macrophages. Dysregulated expression of BAFF impairs B cell activation, proliferation, survival, and immunoglobulin secretion, thereby contributing to the pathogenesis of autoimmune disorders. 49 , 50

Belimumab has been approved by the US FDA for the treatment of systemic lupus erythematosus (SLE), exhibiting favorable efficacy and a reliable safety profile. 51–53 A 24-week double-blind placebo-controlled Phase II study in forty generalised MG patients nonetheless found no separation from placebo on QMG and a similar adverseevent burden between arms. 54 Biology accounts for much of this negative result: belimumab neutralises soluble BAFF and exerts its principal effect on naive and transitional B cells, whereas the long-lived plasma cells responsible for most pathogenic anti-AChR antibody draw their survival signals chiefly from a proliferationinducing ligand (APRIL), and memory B cells operate largely independently of BAFF, 50 , 55 in marked contrast with SLE, where short-lived plasmablasts and naive-cell hyperactivity dominate. A small cohort, mixed antibody subtypes, no stratified analysis, heavy background immunosuppression and a brief 24-week window further blunted statistical power. No Phase III followed, and development of B-cell-axis agents in MG has gravitated toward broader pharmacodynamic footprints: anti-CD20, anti-CD19, anti-CD38, and dual BAFF/APRIL inhibition with telitacicept. 56 The trial therefore argues not against B-cell-axis modulation, but against selective soluble-BAFF blockade as a strategy in a disease sustained by long-lived, BAFF-independent plasma cells.

Telitacicept

Building on the mechanistic lesson of belimumab in MG, namely that selective neutralization of soluble BAFF leaves the long-lived plasma cell compartment that produces most pathogenic AChR autoantibodies largely intact, the development of telitacicept aimed to extend the pharmacodynamic footprint to APRIL-dependent plasma cells through dual cytokine neutralization. Telitacicept, a fusion protein engineered from transmembrane activator and CAML interactor (TACI) and Fc domains, is composed of the specific soluble extracellular region of TACI and the Fc segment originating from human IgG1. This biologic exerts dual targeting actions on both BAFF/BLyS and APRIL, thereby suppressing the proliferative activity and maturational process of B lymphocytes. 56 , 57 Two trimeric components of the tumor necrosis factor (TNF) superfamily, BAFF/BLyS and APRIL, exhibit extensive expression profiles across a broad spectrum of cell types. 55 BAFF/BLyS serves as a key mediator in maintaining the survival, differentiation, and maturation of B cells, whereas APRIL exerts a more prominent regulatory effect on the functional status and viability of long-lived plasma cells, ultimately modulating the process of antibody biosynthesis. 56

Data generated from the Phase II clinical investigation of telitacicept indicated that by the 12th week, the average reductions in QMG scores were 5.8 (±5.85) and 9.5 (±5.03) for the 160 mg and 240 mg dosage cohorts, respectively, alongside swift clinical improvement. 56 Most adverse events were categorized as mild to moderate in severity, and no severe adverse responses have been recorded. Such outcomes further suggest that telitacicept might possess long-term therapeutic potential, as sustained beneficial effects are maintained even after discontinuing the treatment. 33 Moreover, a multicenter, randomized, double-blind, placebo-controlled Phase III study of telitacicept ( NCT05737160 ) has been completed, but the results have not yet been published. Alongside the Lin et al (2024) retrospective series, a real-world study by Rong et al (2026) in 42 refractory AChR-Ab-positive gMG patients recorded a least-squares mean QMG reduction of approximately 2 points at month 5, with consistent gains across ocular, limb and bulbar domains, reductions in concurrent immunosuppression, and a clean safety record. 58

Emerging Monoclonal Antibodies Targeting B Cells or Plasma Cells

Obinutuzumab is a novel anti-CD20 monoclonal antibody that has been approved for the treatment of chronic lymphocytic leukemia (CLL). 59 To date, a single case report has illustrated that obinutuzumab yielded therapeutic efficacy in a CLL patient complicated with MG. 60

Ofatumumab, a fully humanized anti-CD20 monoclonal antibody, has been extensively administered for the management of multiple sclerosis (MS). 61 In terms of MG treatment, only case reports are available in the literature, 62 , 63 and no investigational trials have been initiated for MG to date.

Daratumumab, a monoclonal antibody targeting CD38, has been granted approval for managing multiple myeloma. 64 CD38 is present on plasma cells, plasmablasts, along with subsets of natural killer (NK) cells and regulatory T cells, 65 thereby rendering it a viable therapeutic target for autoimmune diseases. Retrospective investigations have indicated that daratumumab serves as an off-label therapy for a range of autoimmune disorders, MG included, 66 whereas no registered randomized controlled trials dedicated to MG have been conducted so far.

Mezagitamab is a humanized monoclonal antibody targeting CD38. A 16-week Phase II trial has been completed, and the negative outcomes regarding the MG-ADL scale and QMG score have been posted on clinicaltrials.gov ( NCT04159805 ), though the results have not been formally published yet. A Bayesian network analysis indicated that mezagitamab ranked low in terms of both efficacy and safety profiles. 34

Tolebrutinib is a Bruton’s tyrosine kinase (BTK) inhibitor that was once investigated for the treatment of MG. BTK serves as a critical link between cell surface receptors and downstream signaling pathways, playing a pivotal role in B cell development, migration and activation. 67 , 68 Nevertheless, due to the occurrence of a small number of drug-induced liver injury cases in other clinical trials of tolebrutinib, its Phase III trial ( NCT05132569 ) was partially put on clinical hold by the US FDA in 2022.

Targeting Both B Cells and T Cells

Cladribine

Cladribine is a purine analog that exerts selective depletion effects on both B and T lymphocytes.

This agent has been successfully implemented in the management of relapsing-remitting multiple sclerosis (RRMS). 69 , 70 A prospective, open-label study enrolling 13 patients with refractory MG provided evidence supporting the efficacy of subcutaneous cladribine administration: 11 participants achieved significant clinical improvement (a reduction of ≥ 3 points in the MGC score) after 6 months of treatment. 71 Nevertheless, a case report documented that one RRMS patient receiving cladribine treatment developed concurrent AChR-positive MG accompanied by a severe myasthenic crisis. 72 A Phase III trial comparing the efficacy and safety of a novel oral cladribine formulation with placebo in gMG was initiated in 2024 ( NCT06463587 ).

Blinatumomab

Blinatumomab is a bispecific T cell engager (BiTE), whose N-terminal single-chain variable fragment (scFv) targets CD19 on B cells while the C-terminal scFv binds to CD3ε on T cells. Upon bridging these two cell populations, the activated T cells release perforin to induce B cell apoptosis. 73

To date, a case report involving two patients has been published, documenting that both individuals achieved rapid and sustained clinical improvement, with remarkable reductions in the scores of MG-ADL, QMG and 15-item Revised Myasthenia Gravis Quality of Life Scale (MG-QoL15r). Both patients developed grade 1 cytokine release syndrome during the initial treatment phase, whereas no neurotoxicity or serious adverse events were observed. 74

Iscalimab

Iscalimab is a humanized monoclonal antibody targeting CD40, a cell surface molecule widely expressed on B cells, antigen-presenting cells (such as dendritic cells and macrophages), and T cells. By specifically binding to the CD40L-binding site of CD40, this agent blocks the CD40-CD40L-dependent signaling pathway, thereby concomitantly modulating the functions of both T and B cells without inducing the depletion of CD40-positive cells. 75

Findings from a 52-week Phase II randomized controlled trial demonstrated that iscalimab failed to exhibit superior efficacy over placebo with respect to the primary endpoint (QMG score at week 25). Nevertheless, positive signals were observed in the subgroup of patients who had not undergone thymectomy. 31

Why response differs by thymectomy status deserves a closer look. The Phase II study (n=44) returned an overall median QMG change at week 25 of −4.07 with iscalimab against −2.93 with placebo; in the prespecified stratum of patients who had not undergone thymectomy (n=29) the gap widened to −4.35 versus −2.26, with a numerical separation between arms favouring iscalimab on MG Composite at week 13 and on MG-ADL at week 21. 31 As a nondepleting anti-CD40 antibody, iscalimab blocks the CD40 and CD40L interactions that sustain B cell activation that depends on T cells and germinal centre output; 75 its clinical effect should therefore track residual disease activity dependent on CD40. The early-onset AChR-Ab-positive thymus contains ectopic structures resembling germinal centres that drive AChR-antibody production 3 , 49 and are driven by CD40; surgical removal of the thymus eliminates that niche and shifts antibody output to long-lived plasma cells that are independent of CD40 in the marrow. Enrichment of MuSK-Ab-positive or seronegative patients, whose IgG4 class switching is itself dependent on CD40 and CD40L, within the same stratum offers a competing explanation, but the numbers are too small to settle the question and a definitive result stratified by subtype will require larger prospectively designed studies.

Complement Inhibition

Eculizumab

As the first approved C5 inhibitor for the treatment of refractory gMG, eculizumab exerts its core therapeutic effect on MG by blocking the terminal activation process of the classical complement pathway. 17 This agent can specifically inhibit the enzymatic cleavage of the C5 molecule, preventing its breakdown into the proinflammatory mediator C5a and C5b—the precursor of the membrane attack complex (MAC). Consequently, it abrogates the assembly and deposition of MAC, thereby effectively preserving the structural integrity of the postsynaptic membrane at the NMJ. 76 , 77

In the REGAIN trial, eculizumab failed to reach the primary endpoint, defined as the difference in mean ranks of the total MG-ADL score changes from baseline to week 26 between the eculizumab and placebo groups. However, responder analyses of MG-ADL and QMG scores revealed that a higher proportion of patients in the eculizumab group achieved clinically meaningful responses compared with the placebo group. 17 Data from the open-label extension (OLE) phase of the REGAIN trial demonstrated marked reductions in the rates of acute exacerbations, rescue therapy utilization and hospitalization among eculizumab-treated patients. 78 Findings from a Phase III open-label study evaluating eculizumab for pediatric MG indicated that the agent exhibited efficacy in both primary endpoints, namely QMG and MG-ADL scores. 29 Eculizumab has a favorable safety profile, with the most common adverse events including infections, headache, diarrhea and nausea. 79 Real-world data reinforce the trial signal. The global MG SPOTLIGHT Registry (Howard et al 2025; n=189) showed an average MG-ADL fall of 4.2 points at a median of eighteen months, with the share of patients in Myasthenia Gravis Foundation of America (MGFA) class 0-II climbing from 43.7% before initiation to 86.4%, and gains were retained after transition to ravulizumab. 80 A Japanese registry analysis (Tokuyasu et al 2024; n=36 refractory AChR-Ab-positive patients) found that 70% met responder criteria. 81 An Italian multicentre comparison by Pane et al (2024; 32 eculizumab versus 31 efgartigimod recipients) recorded comparable real-world MG-ADL outcomes between the two agents. 82

A handful of practical points around eculizumab administration are worth setting out. The licensed adult schedule comprises an induction phase of 900 mg intravenously weekly for four weeks, 1200 mg at week five, and 1200 mg every fortnight thereafter. 17 The 28 February 2025 FDA paediatric extension brought patients aged six and above into a regimen tiered by body weight. 29 The signal safety concern is a thousand- to two-thousand-fold rise in the risk of invasive meningococcal disease, prompting the FDA boxed warning and a risk evaluation and mitigation strategy (REMS) framework. 83 Pre-treatment MenACWY and MenB vaccination is required, with pneumococcal and Haemophilus influenzae type b (Hib) added. 83 Initiation is barred in the setting of unresolved Neisseria meningitidis infection. 78 , 79

Ravulizumab

Ravulizumab is a long-acting derivative of eculizumab. Engineered histidine substitutions in the complementarity-determining regions (CDRs) confer pH-dependent C5 binding (high affinity at physiologic pH, dissociation in acidic endosomes for FcRn recycling), and two Fc substitutions enhance FcRn binding. This dual engineering yields a terminal half-life 3–4 times longer than eculizumab, permitting dosing every 8 weeks while maintaining sustained terminal complement inhibition. 84

In the CHAMPION MG trial, ravulizumab yielded greater mean reductions in total MG-ADL and QMG scores from baseline to week 26 compared with placebo, meeting the primary endpoint and leading to its regulatory approval in 2022 for the treatment of AChR-positive MG. 19 Data from the OLE phase of the CHAMPION MG trial demonstrated that all scores of patients receiving ravulizumab treatment remained improved over 60 weeks. 22 The most common adverse events associated with ravulizumab include headache, diarrhea and nausea. 85 In a German prospective real-world study (Stascheit et al 2025), ravulizumab and efgartigimod produced comparable MG-ADL/QMG improvements in AChR-Ab-positive gMG; mechanistic profiling confirmed ravulizumab inhibited AChR-Ab-dependent terminal complement activation while efgartigimod predominantly reduced circulating IgG, in the first head-to-head real-world comparison between these classes. 86

Zilucoplan

Zilucoplan is a macrocyclic peptide inhibitor with high specificity for complement component C5. It exerts a dual mechanism of action to block the activation of the terminal complement pathway. First, it inhibits the cleavage of C5 into the proinflammatory mediator C5a and C5b (the precursor of the MAC). Second, it hinders the binding of C5b to C6 via steric hindrance, thereby preventing MAC formation through these two distinct pathways. 87

In the RAISE clinical trial, zilucoplan-treated patients demonstrated a more substantial decrease in MG-ADL scores between baseline and week 12 than their counterparts in the placebo group. 21 Throughout the OLE phase of this trial, the overall therapeutic effect remained stable and satisfactory; patients who transitioned from placebo to zilucoplan attained prompt clinical amelioration. 88 The adverse events most frequently linked to zilucoplan encompass injection site responses, headache, and diarrhea. 21 , 88 , 89

Emerging Complement Inhibitors

Gefurulimab, a bispecific antibody fragment consisting of anti-C5 domains and antigen-binding variable heavy chain (VHH) segments derived from human serum albumin, can selectively bind to complement component C5 and abrogate its activation cascade. 90 Distinguished from currently available C5 inhibitors, this agent boasts a key merit of subcutaneous delivery; a Phase III randomized controlled trial evaluating its efficacy for MG is currently in progress ( NCT05556096 ).

The combined therapeutic regimen of Pozelimab and Cemdisiran adopts a dual-action strategy that incorporates a C5 inhibitor and a small interfering RNA (siRNA) targeting the same complement component. Specifically, Pozelimab exerts selective binding to C5 to block its terminal pathway activation, whereas Cemdisiran suppresses hepatic biosynthesis of C5. 91 This synergistic treatment modality is under investigation for managing paroxysmal nocturnal hemoglobinuria (PNH), 92 and a Phase III randomized controlled trial assessing its therapeutic potential for MG is underway ( NCT05070858 ).

Iptacopan hydrochloride, a low-molecular-weight inhibitor of complement Factor B, targets specifically the alternative complement pathway. By inhibiting Factor B activity, it impairs the activity of C3 convertase linked to the alternative complement pathway. 93–95 Already approved for the treatment of IgA nephropathy, this agent is currently undergoing a Phase III randomized controlled trial for MG ( NCT06517758 ).

Vemircopan, a small-molecule antagonist of complement Factor D, specifically targets the alternative complement pathway and prioritizes its amplification loop. By curbing Factor D activity, it blocks the aberrant activation of this alternative complement cascade. 96 The Phase II trial of vemircopan in MG ( NCT05218096 ) did not reach its prespecified efficacy threshold. Saccà et al (2026) reported a 57% response rate on the primary MG-ADL endpoint in both vemircopan dose groups, compared with 64% in the placebo group, with no significant differences observed across secondary endpoints. 97

Although all complement inhibitors aim to attenuate MAC deposition at the postsynaptic membrane, they differ in cascade intervention level and clinical characteristics. Eculizumab, ravulizumab, zilucoplan and gefurulimab target C5 in the terminal pathway; iptacopan and vemircopan act upstream on factors B and D of the alternative pathway; pozelimab-cemdisiran combines anti-C5 antibody with C5 siRNA. Molecular formats include humanized monoclonal antibodies, a macrocyclic peptide (zilucoplan), a bispecific antibody fragment (gefurulimab), a fully human IgG4 antibody (pozelimab), an siRNA (cemdisiran), and oral small molecules (iptacopan, vemircopan). Routes range from intravenous (IV) biweekly (eculizumab) or every 8 weeks (ravulizumab) to subcutaneous (SC) (zilucoplan, gefurulimab, pozelimab-cemdisiran) and oral (iptacopan, vemircopan). Eculizumab, ravulizumab and zilucoplan are approved for AChR-positive gMG; gefurulimab, pozelimab-cemdisiran and iptacopan are in Phase III (pozelimab-cemdisiran positive NIMBLE results); vemircopan’s Phase II was terminated. Safety profiles share Neisseria meningitidis risk requiring pretreatment vaccination, headache and gastrointestinal symptoms; SC agents add injection site reactions.

Targeting the FcRn

Efgartigimod

Efgartigimod is an Fc fragment antibody derived from human IgG1 that targets FcRn. Its core mechanism of action involves competitive binding to FcRn, which blocks the FcRn-mediated IgG recycling pathway, facilitates the degradation of pathogenic IgG autoantibodies, and thus achieves a significant reduction in serum IgG levels. In comparison with full-length anti-FcRn monoclonal antibodies, this agent retains pH-dependent binding properties, enabling self-recycling and thereby prolonging its in vivo half-life. 98 , 99

In the ADAPT Phase III randomized controlled trial, the proportion of MG-ADL responders-defined as patients who achieved a reduction of at least 2 points in the MG-ADL score within the first treatment cycle, with the improvement sustained for a minimum of 4 weeks-was higher in the efgartigimod group than in the placebo group. 18 Specifically, 67.7% of efgartigimod-treated AChR-Ab-positive patients were MG-ADL responders compared with 29.7% on placebo. A parallel benefit was observed for QMG responders (63.1% vs 14.1%), and 40.0% of efgartigimod-treated patients achieved minimal symptom expression (MG-ADL of 0 or 1) compared with 11.1% of placebo recipients. Notably, 84.1% of MG-ADL responders and 82.9% of QMG responders exhibited an onset of effect within the first two weeks of the initial cycle, underlining the rapid onset of action of FcRn blockade. 18 The ADAPT-SC non-inferiority trial further verified that the efficacy of its subcutaneous formulation was comparable to that of the intravenous formulation. 32 In terms of safety, efgartigimod is generally well tolerated, with the most common adverse events including infections, headache and nausea; additionally, injection site reactions are observed specifically with subcutaneous administration. The dosing regimen of efgartigimod adopts a cyclic strategy, which can be administered either intravenously or subcutaneously (once weekly for 4 consecutive weeks, followed by a drug-free interval of at least 4 weeks). 18 , 32 Currently, trials investigating alternative dosing schedules (eg, consecutive administration every 2 weeks without an interval) are underway ( NCT04980495 , ADAPT-NXT). The open-label 6-month study by Khateb et al (2025) in 30 adults with moderate to severe double-seronegative gMG, using weekly induction for four weeks followed by biweekly maintenance for five months, met its prespecified primary endpoint (MGII change at six months), with the Myasthenia Gravis Impairment Index (MGII) decreasing by 11.92 points; the secondary endpoint of MG-ADL also showed parallel improvement; adverse events were predominantly mild, providing the first prospective evidence that efgartigimod is well tolerated and efficacious in this historically underrepresented antibody subtype. 100 Real-world series corroborate the ADAPT and ADAPT-SC findings. A Chinese multicentre cohort (Luo et al 2024; n=61, 92% AChR-Ab-positive) documented clinically meaningful improvement in 97% of patients within 1.3 ± 0.7 weeks; 101 the UK Early Access experience (Moniz Dionísio et al 2025; n=48 with long-standing refractory AChR-Ab-positive disease), 102 a larger Chinese cohort (Hao et al 2025; n=77), 103 and a multicentre analysis in patients aged eighty and above (Hong et al 2025) 104 all reproduced the picture of rapid, durable MG-ADL benefit and acceptable tolerability, broadening the evidence base to thymoma-associated disease, myasthenic crisis, very-late-onset and seronegative MG.

Rozanolixizumab

Rozanolixizumab is a humanized monoclonal antibody targeting the IgG-binding domain of FcRn, which inhibits FcRn-mediated IgG recycling by specifically blocking the binding of FcRn to IgG. 105

In the MycarinG Phase III randomized controlled trial, both treatment dose groups (7 mg/kg and 10 mg/kg) were superior to the placebo group in terms of the primary endpoint (change in MG-ADL score from baseline to day 43), demonstrating definite therapeutic benefits. 20 This agent has been approved by regulatory authorities in Europe and the United States for the treatment of AChR-positive and MuSK-positive gMG, making it the first monoclonal antibody approved for MuSK-positive gMG. Safety data indicate that it is well tolerated, with the most common adverse events including headache, diarrhea, fever and nausea. 20 , 106

Emerging FcRn Antagonists

Batoclimab, a fully human monoclonal antibody endowed with selective FcRn-targeting activity, can reduce circulating IgG levels by approximately 70%. 107–109 Evidence obtained from a Phase III clinical trial indicated that the rate of durable MG-ADL score improvements in the batoclimab-treated group was higher than that observed in the placebo cohort throughout the first course of treatment, with noticeable intergroup difference in scores manifesting as early as week 2. 109 The most commonly reported adverse events include infections, peripheral edema, diarrhea, MG symptom exacerbation and hypercholesterolemia. At present, a 24-week Phase III randomized controlled trial, designated as the FLEX trial, is underway in Europe, the United States and Japan ( NCT05403541 ). 110

Nipocalimab, an aglycosylated monoclonal antibody, targets specifically the IgG-binding domain of FcRn. Findings from the Phase II Vivacity-MG trial revealed that none of the individual dosage cohorts of nipocalimab achieved a clear improvement in MG-ADL scores when compared with the placebo arm; by contrast, a marked enhancement in MG-ADL scores was detected after pooling all nipocalimab dosage groups for a head-to-head comparison with the placebo cohort. 111 Data accrued from the Phase III randomized controlled trial Vivacity-MG3 indicated that the mean total MG-ADL and QMG scores in the nipocalimab-treated group were reduced from baseline levels at weeks 22, 23, and 24. 23

Although the four FcRn antagonists share FcRn-mediated IgG recycling blockade, they differ clinically. Efgartigimod is a human IgG1-derived Fc fragment; rozanolixizumab and batoclimab are full-length monoclonal antibodies; nipocalimab is aglycosylated with minimized Fcγ-receptor effector function. Efgartigimod retains pH-dependent self-recycling; full-length antibodies achieve longer exposure and stronger IgG reduction (≈70% with batoclimab). Routes: efgartigimod IV and SC, rozanolixizumab/batoclimab SC, nipocalimab IV. Dosing: efgartigimod cyclic (4 weekly + drug-free interval); the others continuous weekly/biweekly. Efgartigimod and rozanolixizumab are approved for AChR-positive gMG (rozanolixizumab also for MuSK-positive); nipocalimab approved on the basis of the Phase III Vivacity-MG3; batoclimab remains investigational. Adverse events overlap (headache, infections, gastrointestinal (GI)) with class-specific signals: injection-site reactions for SC, peripheral edema and hypercholesterolemia for batoclimab.

Targeting Cytokines

Satralizumab

Satralizumab is a humanized monoclonal antibody targeting the IL-6R. As a pleiotropic cytokine with key roles in inflammation and immune regulation, interleukin-6 (IL-6) can be secreted by various cell types including B cells and T cells. It initiates downstream signaling pathways through binding to membrane-bound IL-6R and soluble IL-6R, thereby participating in pivotal pathological processes such as the activation and differentiation of naive T cells into proinflammatory helper T cells, as well as the differentiation of B cells into plasmablasts that secrete antibodies. 112 Serum IL-6 levels have been reported to be elevated in some studies of treatment-naive MG patients, with reductions described in parallel with disease activity following immunosuppressive therapy in selected cohorts, although elevation is not universal across studies and correlations with disease activity have been variable. 113 Satralizumab can specifically block the aforementioned signal transduction mediated by IL-6, suppress the activation and differentiation of T cells as well as the transformation of B cells into plasmablasts, and may indirectly reduce the activity of autoreactive B cells.

Satralizumab has been approved for the treatment of seropositive NMOSD. 114 , 115 Findings from the Phase III clinical trial LUMINESCE evaluating satralizumab for MG indicated that at week 24, compared with placebo, the satralizumab group achieved a modest improvement in MG-ADL scores, meeting the primary endpoint. The proportion of patients who experienced at least one adverse event during the double-blind period was slightly higher in the satralizumab group than in the placebo group. 116

Tocilizumab

Tocilizumab is also a humanized monoclonal antibody targeting the IL-6R. By blocking the binding of IL-6 to IL-6R, it inhibits proinflammatory responses mediated by the IL-6 pathway and may modulate the activity of B cells and autoantibody production, thereby exerting its therapeutic effects. 117

Tocilizumab has been approved for the treatment of rheumatoid arthritis, giant cell arteritis, polyarticular and systemic juvenile idiopathic arthritis, interstitial lung disease associated with systemic sclerosis, cytokine release syndrome induced by CAR T cells, and severe COVID-19. 117 A retrospective case series study enrolling 5 patients with very late-onset (onset age > 65 years in all cases) AChR-positive MG demonstrated that tocilizumab combined with low-dose oral glucocorticoids yielded favorable efficacy and safety profiles. 118 Findings from a prospective, open-label, single-arm study indicated that during tocilizumab treatment, the MG-ADL and QMG scores decreased, the dosage of prednisone was also reduced, and no serious adverse events were observed during the follow-up period. 119 Additionally, a single-centre prospective cohort study reported preliminary signals of efficacy and acceptable short-term tolerability of tocilizumab in reducing MG-ADL scores and decreasing prednisone dosage in patients with refractory AChR-positive gMG, although in the absence of a randomized placebo-controlled comparator and with a limited sample size, definitive conclusions regarding the safety and efficacy of tocilizumab in MG cannot yet be drawn. 120 A multicentre, randomized, double-blind, placebo-controlled Phase II trial of intravenous tocilizumab in approximately 64 patients with AChR-Ab-positive gMG (MGFA classes II–IV; tMG, NCT05067348 ) is currently recruiting in China, with an open-label Phase II/III extension trial (tMG-E, NCT05716035 ) planned for participants completing the randomized phase, and the results of these trials are awaited to provide the first randomized controlled evidence on tocilizumab in MG.

Other Therapeutic Modalities

CAR-T Therapy

CAR-T cells are genetically engineered T lymphocytes that express synthetic chimeric antigen receptor (CAR) on their cell surface. This receptor enables T cells to directly recognize and bind to target antigens independent of antigen-presenting cells, and the process of target antigen recognition triggers T cell activation, thereby achieving precision elimination of antigen-positive target cells. 121

Currently, a variety of B cell-associated targets are currently being investigated in early-phase clinical trials. In the field of MG, current research has focused on two core targets, namely B-cell maturation antigen (BCMA) and CD19. 122 , 123 CAR-T cell therapy in MG should at present be regarded as an investigational, early-phase intervention rather than an established clinical treatment. The published evidence comprises small open-label Phase I studies and a recently completed Phase 2b randomized, double-blind, placebo-controlled trial of the BCMA-directed mRNA-engineered Descartes-08 construct in 26 patients with gMG, in which 66.7% of treated participants achieved at least a 5-point MGC improvement at month 3 versus 27.3% of placebo recipients, 124 together with a single-centre Phase I trial of a bispecific BCMA/CD19 CAR-T construct in 18 patients with refractory gMG. 125 No CAR-T product is yet approved for MG, and these agents have not been incorporated into mainstream international consensus guidance. Broader clinical adoption among neuromuscular experts therefore awaits the results of larger, multicentre randomized controlled trials and the maturation of long-term safety data. Safety concerns related to this therapy mainly include the following aspects: adverse reactions induced by lymphodepletion conditioning regimens (typically fludarabine combined with cyclophosphamide), increased risk of infections secondary to hypogammaglobulinemia caused by B cell depletion, as well as the potential occurrence of cytokine release syndrome (CRS) and ICANS. 126

Autologous HSCT

HSCT is currently the only therapeutic modality capable of eradicating all autoreactive T and B lymphocytes in the body completely, thereby achieving immune reconstitution, namely the remodeling of the immune system.

In the management of MG, pertinent clinical cases have been reported in existing literature thus far. 127–132 These case data demonstrated that the overwhelming majority of patients attained complete stable remission or minimal manifestation status, and could cease all therapeutic drugs associated with MG. The benefit and risk profile of HSCT has not been completely clarified. Primary safety considerations primarily center on three key aspects: systemic toxic effects triggered by intensiveconditioning regimens, increased risk of severe infections, and mortality associated with treatment. Potential long-term sequelae encompass secondary autoimmune disorders, reproductive impairment, and secondary malignant tumors. 131

Bortezomib

Bortezomib, a proteasome inhibitor, can trigger apoptotic cell death in cells with elevated protein turnover activity. Therefore, plasma cells- which are required to maintain continuous antibody secretion and thus display high protein turnover- are particularly vulnerable to its cytotoxic actions. 133

After securing supportive experimental data from MG animal models, 134 case reports describing the use of bortezomib for MG therapy have been published so far. 135 , 136 A Phase IIa clinical trial (the TAVAB trial, NCT02102594 ) aimed at investigating bortezomib’s efficacy in antibody-mediated autoimmune diseases- including systemic lupus erythematosus, rheumatoid arthritis, and MG- was halted due to recruitment challenges.

Unresolved Issues and Future Research

All of the agents discussed in this review are positioned as second-line or later-line options, used as add-on or alternatives to conventional first-line regimens in patients with refractory, steroid-dependent, or otherwise inadequately controlled disease, with several already integrated into international consensus guidance for subtypes defined by antibody status and others still in clinical development. However, the strength of evidence supporting these classes varies markedly, and the agents should not be regarded as clinically equivalent options. The most robust evidence base, comprising multiple Phase III randomized placebo-controlled trials and incorporation into international consensus guidance, supports complement inhibitors (eculizumab, ravulizumab, and zilucoplan) and FcRn antagonists (efgartigimod, rozanolixizumab, and nipocalimab) for AChR-Ab-positive gMG, as well as rozanolixizumab for MuSK-Ab-positive gMG. 5 , 17–23 Agents that deplete B cells form a rapidly maturing intermediate tier: rituximab has one positive phase III trial (RINOMAX) in AChR-positive disease and robust real-world data in MuSK-positive disease, 24–28 , 42 while inebilizumab became the first approved CD19-targeted therapy for both subtypes in December 2025. 30 IL-6R inhibitors have limited evidence, 116 , 118–120 and agents targeting T cells, CAR-T and autologous HSCT remain investigational, with most showing negative or preliminary results. 31 , 71 , 74 , 122–125 , 137 This hierarchy guides clinical practice: only complement inhibitors, FcRn antagonists and selected agents that deplete B cells are established second-line options for defined subtypes, while others remain promising but unproven. Collectively, these therapies broaden treatment options for patients with different antibody profiles and disease severity, and provide a mechanistic basis for further investigation of MG pathogenesis. Several limitations nevertheless remain. While complement inhibitors, FcRn antagonists, inebilizumab and satralizumab have established short-term efficacy and safety in phase III randomized trials, 17–23 , 30 , 116 and rituximab has been evaluated in two multicenter randomized studies, 27 , 28 all other mechanistic classes - including dual BAFF/APRIL inhibition, CD38 targeting and CAR-T constructs - remain limited to phase II or earlier trials, single-arm studies and small case series. Even for agents approved on the basis of phase III trials, critical limitations remain: no head-to-head comparative trials exist, pediatric data are extremely scarce (only eculizumab has a dedicated pediatric phase III trial, and nipocalimab is approved for adolescents ≥12 years), and evidence stratified by subtype for MuSK, LRP4 and double-seronegative disease is still emerging. The durability of the response to FcRn antagonists, relapse patterns after discontinuation, and the broader applicability of CAR-T and agents targeting CD40 all require higher levels of evidence. Furthermore, while AChR-positive and MuSK-positive gMG now have multiple approved options, treatment choices remain limited for rarer subtypes including LRP4-antibody-positive, double or triple seronegative, pediatric and very-late-onset disease. Randomized data specific to subtype for these underserved populations are just becoming available, most notably from the eculizumab pediatric trial 29 and the recently completed ADAPT-SERON study of efgartigimod. Two pairings of subtype and drug deserve emphasis: rituximab achieves a disproportionately greater response in MuSK- than AChR-Ab-positive disease (82%/56% in the dedicated meta-analysis; 42 72% vs 30% in Tandan et al 25 58% vs 16% in Hehir et al 24 ), and efgartigimod has accrued the strongest evidence in double-seronegative gMG (Khateb et al 2025: 11.92 point MGII reduction and 72% responder rate; 100 corroborated by ADAPT-SERON). The belimumab miss in MG illustrates a wider point: the molecular target itself, rather than the pathogenic relevance of the cell lineage it touches, can decide clinical outcome. This experience has steered B-cell-axis development toward broader pharmacodynamic strategies including dual BAFF/APRIL inhibition and direct plasma cell depletion.

While head-to-head trials are lacking, several considerations at the patient level can guide rational biologic use. Age influences tolerability and choice: only eculizumab has a dedicated Phase III paediatric study; in late-onset disease, comorbidity and infection risk favour, agents that act faster and are less broadly immunosuppressive agents (FcRn antagonists, complement inhibitors). Time from onset matters: in newly diagnosed active disease, the delayed onset of conventional immunosuppressants (≥6 months) supports earlier introduction of fast-acting biologics; in long-standing or refractory disease, deeper or more durable effects (rituximab in MuSK-positive, depletion of B cells and plasma cells, or cellular therapies in selected cases) become more relevant. Severity and antibody status refine selection: FcRn antagonists and complement inhibitors in crisis/impending crisis; complement inhibitors/FcRn antagonists for AChR-positive; rituximab and rozanolixizumab for MuSK-positive; efgartigimod supportive in double-seronegative. These are orientations informed by evidence, not substitutes for individualized judgment and consensus guidance. In particular, rituximab’s role is firmly established for MuSK-Ab-positive disease and selectively supportive for early or refractory AChR-Ab-positive disease, but not yet definitively established against stringent steroid-sparing endpoints in long-standing AChR-Ab-positive disease.

Matching evidence to disease subtype is central. In AChR-Ab-positive gMG, complement inhibition and FcRn antagonism are backed by Phase III data and consensus guidance, with eculizumab the sole paediatric option from six years of age. For MuSK-Ab-positive disease, rituximab remains the best-substantiated targeted approach (72–82% MM-or-better), rozanolixizumab is the first FcRn antagonist licensed specifically for this subtype, and inebilizumab’s December 2025 FDA approval became the first to cover both AChR- and MuSK-Ab-positive disease in a single decision. No agent has yet earned a regulatory indication in LRP4-Ab-positive or in double or triple seronegative disease. In thymoma-associated MG, complement inhibitors and FcRn antagonists have proven workable in real-world cohorts. Juvenile MG remains markedly underserved: eculizumab is alone in carrying a paediatric Phase III, and nipocalimab is licensed from age twelve. Very-late-onset disease (over 65) lends itself well to FcRn antagonism or complement blockade, with small series additionally supporting tocilizumab. Across refractory disease, that deplete B cells and plasma cells strategies have been investigated but the evidence is still chiefly observational.

Targeted treatment of double or triple seronegative gMG has long been hampered by low recruitment into pivotal trials and a higher failure rate on conventional immunosuppression. FcRn antagonism is changing that picture. An open-label six-month prospective study of IV efgartigimod in 30 adults with moderate-to-severe double-seronegative gMG delivered clinically meaningful gains on MG-ADL and QMG with tolerability matching that seen in seropositive patients. 100 The Italian real-world cohort by Pane et al (2024) included six seronegative cases whose response paralleled the rest of the series. ADAPT-SERON (n=119; spanning MuSK-, LRP4-Ab-positive and triple-seronegative subtypes) met its primary MG-ADL endpoint at day 29, the first Phase III read-out for an FcRn antagonist in this population, with a planned supplemental Biologics License Application (BLA). A small Japanese case series (n=16) reported a more modest response, yet the overall body of work positions FcRn antagonism as the strategy with the strongest evidence in seronegative disease. Dedicated registries are still a priority.

As framed in the Introduction, interpretation of the open-label, retrospective and real-world data summarized here (particularly for rituximab in MuSK-MG, telitacicept in refractory AChR-MG, tocilizumab in very-late-onset AChR-MG, blinatumomab and CAR-T in refractory disease, and HSCT) is constrained by concomitant background immunosuppression and the absence of placebo control, reinforcing the need for randomized placebo-controlled trials in subtypes with predominantly observational evidence.

Although targeted biologics largely sidestep the cumulative harm of long-term corticosteroids, 13 , 14 each class brings its own adverse event signature. Agents directed at B cells and plasma cells (rituximab, inebilizumab, ofatumumab, obinutuzumab, telitacicept and daratumumab) produce infusion or injection reactions, 25 , 30 respiratory and urinary infections, hypogammaglobulinaemia after sustained anti-CD20 exposure, 138 hepatitis B reactivation, 139 and the occasional case of progressive multifocal leukoencephalopathy. Tolebrutinib, a BTK inhibitor, has been linked to drug-induced liver injury (alanine aminotransferase (ALT) more than three times the upper limit of normal in 4.0% of MS Phase III participants), 140 which triggered the FDA’s 2022 partial clinical hold and the URSA MG termination. Cladribine raises a concern around prolonged lymphopenia, and blinatumomab additionally around cytokine release syndrome. 74 FcRn antagonists give rise to headache, infections and a theoretical longer-term infection risk from sustained IgG depletion; 18 , 20 batoclimab and nipocalimab can produce transient hypoalbuminaemia and hypercholesterolaemia, 141 and peripheral oedema was reported in 38.8% of batoclimab recipients in Phase III. 109 IL-6R inhibitors are associated with infections, neutropenia, transaminase elevation, dyslipidaemia and, uncommonly but recognised (particularly with tocilizumab), gastrointestinal perforation. 116 , 142 CAR-T therapy is dominated by lymphodepletion-related toxicity, cytokine release syndrome, ICANS, prolonged cytopenias and, since January 2024, a class-wide FDA boxed warning for secondary T-cell malignancy; 143 autologous HSCT carries an appreciable treatment-related mortality together with risks of secondary autoimmunity and malignancy; 131 bortezomib adds peripheral neuropathy and thrombocytopenia. 133 Long-term pharmacovigilance for these newer agents remains essential.

Mortality figures across these classes, though reported with varying levels of granularity, are reassuring on balance. Within the complement inhibitors, the REGAIN open-label extension (n=117) recorded three deaths unrelated to the study drug; 17 , 78 the CHAMPION-MG extension (n=169) reported five (cerebral haemorrhage, drug toxicity, three from COVID-19), none considered attributable by the investigators; 22 and RAISE-XT (n=200) logged six treatment-emergent deaths, none treatment-related and none caused by meningococcal infection. 88 Among FcRn antagonists, ADAPT+ (n=145, 217.6 participant-years) registered five fatal serious adverse events (AEs), none attributed to efgartigimod; 32 MycarinG reported no deaths during randomisation; 20 and Vivacity-MG3 recorded one nipocalimab fatality (myasthenic crisis) and two on placebo. 23 On the B-cell side, MINT (n=190) noted one death on inebilizumab against two on placebo, 30 while BeatMG and RINOMAX recorded no treatment-related deaths. 27 , 28 LUMINESCE recorded none during double-blind treatment, 116 and the Phase II telitacicept programme (n=29) reported no mortality. 56 The Phase II vemircopan study included one death from hepatic failure. Among cellular therapies, the Descartes-08 Phase 2b cohort (n=26) had no treatment-related deaths during randomisation, 124 whereas the largest refractory MG HSCT series (Beland et al 2025; n=21) reported a 100-day treatment-related mortality of 9.5%, making HSCT the modality with the highest procedural mortality in this review. 137 Treatment-related deaths are therefore uncommon overall and concentrate around cellular and stem cell procedures.

Long-term safety experience with targeted biologics in MG, while still building, already supports a handful of class-level conclusions. On the complement side, the REGAIN extension (up to four years; median 22.7 months in 117 patients) showed a profile in line with the randomised period and no meningococcal infections at interim analysis; 78 the CHAMPION-MG extension (median 759 days) confirmed durable benefit; 22 and RAISE-XT documented stable efficacy and tolerability of self-administered subcutaneous zilucoplan. 32 Among the FcRn antagonists, ADAPT+ accumulated 217.6 participant-years across up to seventeen treatment cycles, with treatment-emergent AEs in 84.8% (most often headache, COVID-19 and nasopharyngitis 144 ), and multi-year experience is now accruing with rozanolixizumab, nipocalimab and batoclimab. The N-MOmentum inebilizumab extension supplies more than four years of acceptable long-term exposure relevant to MG, 46 while long-term rituximab data record infections, hypogammaglobulinaemia, hepatitis B virus (HBV) reactivation and a small tail of late events. The telitacicept extension reported sustained 48-week benefit with no new safety signals. 56 Long-term surveillance for cellular therapies has not yet caught up with the FDA’s class-wide boxed warning for secondary T-cell malignancy issued in January 2024. 143 The signals most worth monitoring across the agents are cumulative infection burden, hypogammaglobulinaemia after sustained B-cell depletion or FcRn blockade, encapsulated bacterial infection on complement inhibition, metabolic disturbance with selected FcRn antagonists, and late haematological or oncological events after cellular therapy. Structured pharmacovigilance, registry participation and serial laboratory monitoring remain indispensable.

Beyond failure of effect and unacceptable toxicity, several further considerations shape decisions on starting, switching and stopping these agents. When generalised disease is newly diagnosed and active, the slow onset of conventional immunosuppression (six to twelve months for azathioprine, a year or more for mycophenolate) argues for early use of a faster-acting biologic such as an FcRn antagonist or a complement inhibitor. In patients already on conventional therapy, reasonable triggers to escalate include sustained moderate-to-severe activity (MG-ADL of six or more, or QMG of eleven or more) despite an adequate trial, recurrent exacerbations, ongoing dependence on plasma exchange or IVIG, intolerance or contraindications to the standard regimen, and a steroid maintenance dose that cannot be brought to acceptable levels. Antibody status sharpens choice: rituximab for MuSK-Ab-positive disease, complement inhibitors or FcRn antagonists for AChR-Ab-positive disease, and rozanolixizumab spanning both. Reasons to discontinue include sustained pharmacological remission or MM status, progressive hypogammaglobulinaemia or low IgG, recurrent serious infections, the need for live vaccines, pregnancy or breastfeeding planning, malignancy, and regulatory or programme withdrawal, with vemircopan in MG and tolebrutinib in MS being recent examples. Switching is generally driven by suboptimal response, poorly tolerated reactions, route or dosing preference, a change in disease phenotype, or accrual of class-specific toxicity. Each decision should be tailored to the individual.

How quickly switching can be assessed reflects the pharmacodynamics of the agent in use. With an FcRn antagonist (IgG nadir within one to two weeks), suboptimal response can be judged after one to two cycles, roughly eight to sixteen weeks; an intra-class switch (for example, from IV efgartigimod to subcutaneous rozanolixizumab, batoclimab or nipocalimab) or an inter-class move (to complement inhibition in AChR-Ab-positive disease, to B-cell depletion in MuSK-Ab-positive disease) can usually be settled within twelve to sixteen weeks. Complement inhibitors act within days to weeks, so primary non-response is evaluable inside twelve weeks; intra-class moves among C5 inhibitors tend to follow route or tolerability preferences, with inter-class switching reserved for genuine non-response. B-cell-depleting agents demand a longer view: autoantibody and clinical change typically take three to six months to declare themselves, and re-treatment or switching is usually decided at six to twelve months, with comparable windows applying to telitacicept and CD38-directed depletion. Inter-class change rarely arises with cellular therapies. Beyond the headline MG-ADL or QMG number, the depth and durability of response and the cumulative tolerability burden should also weigh in the decision.

Whether these agents enhance or undercut one another when combined or sequenced has not yet been put to a randomised test, but several mechanistic principles apply. Rational pairings include orthogonal complement inhibition together with depletion of B cells or plasma cells; FcRn blockade alongside B-cell depletion, marrying rapid IgG clearance with elimination of the cells that produce autoantibody; and an anti-CD20 or anti-CD19 phase followed by anti-CD38 or BCMA-directed CAR-T. Limited experience speaks to feasibility: sequential rituximab then efgartigimod in MuSK-Ab-positive MG, 145 real-world cohorts switching safely between classes, and the Pane et al series showing successful transitions from eculizumab to efgartigimod and the reverse. 82 On the cautionary side, FcRn antagonism can accelerate catabolism of co-administered IgG biologics, so timing relative to B-cell-depleting infusions or loading of a complement inhibitor deserves attention. Same-class pairings with overlapping target spectra make little biological sense. Cumulative immunosuppression - B-cell depletion stacked with FcRn antagonism and chronic corticosteroids - must be weighed. Current practice favours sequencing rather than simultaneous inter-class use.

Two practical constraints further limit wider adoption. First, cellular therapies still carry incompletely controllable risks of cytokine release syndrome and long-term oncogenicity. Second, targeted biologics are typically costly and inconsistently covered by health insurance, which limits access for patients in less-resourced settings and can undermine long-term treatment adherence. In addition, the determinants of long-term cellular-therapy safety, and the precise immunomodulatory effects of newer agents at the single-cell level, remain insufficiently characterized; this constrains accurate prediction of response and adverse events and, in turn, the individualization of therapy.

With head-to-head trials still missing, network meta-analyses have become a key reference. Chen et al (2023) placed FcRn antagonism and B-cell-depleting strategies favourably for MG-ADL gain, with safety broadly comparable across classes. 34 Ma et al (2024) confirmed superiority of FcRn antagonists and complement inhibitors over placebo on QMG and MGC, with batoclimab producing notably large effect estimates. 146 Zhong et al (2024) flagged the rapid onset of FcRn antagonism and complement inhibition, set against the slower but more enduring effects of B-cell depletion. 147 Smith et al (2024) generated number needed to treat (NNT) and number needed to harm (NNH) estimates for efgartigimod IV, rozanolixizumab, ravulizumab and zilucoplan against placebo plus standard of care. 148 Taken together, the analyses anchor FcRn antagonism and complement inhibition as the primary established options for AChR-Ab-positive gMG and B-cell-depleting agents as the leading choice in MuSK-Ab-positive disease, while leaving intra-class ranking unresolved.

Future research in MG targeted therapy should pursue four priorities. First, subtype-stratified, multicentre randomized controlled trials, complemented by real-world registries, are needed to define the optimal positioning of each targeted strategy and to consolidate long-term efficacy and safety data. Second, target discovery should extend beyond established mechanisms to candidates such as novel targets within the complement pathway beyond the already-validated C5 (including C1q, C3, and the alternative-pathway regulators Factor B and Factor D), 77 regulatory T-cell pathways, and agents specifically designed for rare subtypes; in parallel, development and process optimization of domestically produced biologics may reduce cost and improve access. Third, combination and sequencing strategies - for example, complement or FcRn blockade combined with B-cell or plasma-cell depletion, and stepwise application of small molecules and cellular therapies - warrant systematic evaluation, while safer cellular platforms, including mRNA-engineered CAR-T and reduced-intensity HSCT conditioning regimens, also merit further refinement. Fourth, integration of single-cell and multi-omic profiling into biomarker discovery should enable prediction of response and adverse events and support individualized regimens. Together, these advances should clarify the mechanisms of targeted therapy, strengthen the evidence base, and progressively improve outcomes for patients with MG.

Conclusions

Targeted biological and cellular therapies have substantially expanded the treatment landscape for gMG. Complement inhibitors (eculizumab, ravulizumab, and zilucoplan) are now established, guideline-supported options for AChR-antibody-positive disease, each supported by a Phase III randomised controlled trial. Among FcRn antagonists, efgartigimod is approved for AChR-antibody-positive gMG, while rozanolixizumab and nipocalimab are approved for both AChR-antibody-positive and MuSK-antibody-positive disease, together extending targeted therapy across the two most common antibody subtypes. B-cell-depleting agents, led by rituximab in MuSK-antibody-positive disease and inebilizumab across both major antibody subtypes, represent a rapidly maturing second tier. IL-6 receptor inhibitors, T-cell-targeted agents, CAR-T constructs, and autologous HSCT remain investigational, with most evidence confined to early-phase or observational studies. Despite this progress, critical gaps persist: no head-to-head comparative trials exist, evidence in seronegative, paediatric, and very-late-onset disease remains limited, long-term safety data are still accruing, and validated predictive biomarkers are absent. These limitations constrain individualised treatment selection and underscore the need for further research.

Future advances should prioritise subtype-stratified randomised trials to clarify optimal agent sequencing, expanded biomarker discovery using single-cell and multi-omic approaches to enable personalised therapy, systematic evaluation of rational combination strategies pairing orthogonal mechanisms, and development of safer cellular therapy platforms. Addressing these priorities will strengthen the evidence base, improve equitable access, and progressively translate mechanistic insights into better outcomes for all patients with MG.

Funding Statement

This work was supported by the Key Program of the Natural Science Foundation of Chongqing, China (Grant No. CSTB2024NSCQ-KJFZZDX0005), and the Chongqing Municipal Clinical Key Specialty Construction Project (Year 2024).

Data Sharing Statement

This is a narrative review article based entirely on previously published literature. No original experimental data, research materials, or custom datasets were generated or used in the preparation of thispaper. All relevant data cited in this work are available from the corresponding original published studies (see References for details), and any further information is available from the corresponding author (Zhaoyou Meng, mengzhaoyou@tmmu.edu.cn) upon reasonable request.

Author Contributions

Y.Z.: Conceptualization, Writing – original draft, Writing – review & editing. Y.W.: Conceptualization, Writing – original draft, Writing – review & editing. Z.M.: Conceptualization, Supervision, Writing – review & editing. Yue Zhou and Yutong Wu share first authorship. All authors gave final approval of the version to be published; have agreed on the journal to which the article has been submitted; and agree to be accountable for all aspects of the work.

Disclosure

The authors have no conflict of interests.

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Associated Data

Data Availability Statement

This is a narrative review article based entirely on previously published literature. No original experimental data, research materials, or custom datasets were generated or used in the preparation of thispaper. All relevant data cited in this work are available from the corresponding original published studies (see References for details), and any further information is available from the corresponding author (Zhaoyou Meng, mengzhaoyou@tmmu.edu.cn) upon reasonable request.