Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio, ZAI Lab - LARVOL DELTA

A case report of sequential efgartigimod and rituximab treatment for tSNMG. (PubMed, Front Immunol) - "After admission, the patient showed poor responses to treatments including pyridostigmine bromide, human immunoglobulin, glucocorticoids, and tacrolimus. Ultimately, following therapy with efgartigimod followed by rituximab, significant symptomatic improvement was observed. By analyzing the diagnosis and treatment process of this case alongside relevant literature, this report explores the therapeutic value of efgartigimod followed by rituximab for tSNMG, key points for individualized regimen adjustments, and the challenges in diagnosis and treatment, thereby providing a reference for the clinical management of such refractory cases."

Journal • CNS Disorders • Immunology • Myasthenia Gravis • Solid Tumor • Thymoma • Thymus Cancer

Case Report: A case of anti-mGluR1 antibody-associated encephalitis treated with efgartigimod combined with rituximab. (PubMed, Front Immunol) - "At the 12-month follow-up, the patient's clinical symptoms continued to improve, with no observed relapse. This case report underscores the importance of early diagnosis and therapeutic intervention, highlighting the need to suspect this condition based on its characteristic clinical presentation."

Journal • Ataxia • CNS Disorders • Epilepsy • Immunology • Movement Disorders • Psychiatry

Efgartigimod-alfa therapy in acetylcholine receptor antibody myasthenia gravis: Romanian experience. (PubMed, Front Neurol) - "Among patients on corticosteroids (n = 29), median prednisone dose decreased from 20.0 to 10.0 mg/day (p = 0.004), with 58.6% achieving reduction or discontinuation. This first Eastern European real-world efgartigimod-treated cohort confirms rapid and sustained improvement and corticosteroid sparing, consistent with prior evidence."

Clinical • Journal • CNS Disorders • Immunology • Myasthenia Gravis

Efficacy and Safety Study of Efgartigimod in the Treatment of Acute Episodes of Neuromyelitis Optica Spectrum Disorders (ChiCTR) - P=N/A | N=60 | Not yet recruiting | Sponsor: The Third Affiliated Hospital Sun Yat-sen University; The Third Affiliated Hospital Sun Yat-sen University

New trial • CNS Disorders • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

Pathophysiology and Emerging Therapies in Chronic Inflammation Demyelinating Polyneuropathy. (PubMed, Intern Med) - "Recent advances in targeted therapies, particularly the introduction of neonatal Fc receptor (FcRn) inhibitors, have transformed the therapeutic landscape, while complement-targeted therapies remain under clinical investigation. This review summarizes the current understanding of CIDP immunopathology and discusses evolving therapeutic strategies, including FcRn-targeted therapies, for mechanism-based precision medicine."

Journal • Immunology • Inflammation • Pain • CNTN • CNTN1

ANSWER: Efgartigimod in Anti-NMDA Receptor Encephalitis. (clinicaltrials.gov) - P2 | N=20 | Recruiting | Sponsor: Beijing Tongren Hospital

New P2 trial • CNS Disorders • Immunology

Effectiveness and safety of efgartigimod in patients with anti-NMDAR or anti-LGI1 encephalitis and its 18F-DPA714 PET imaging evaluation: A multicenter, single-arm, prospective, observational real-world study. (PubMed, Neuroprotection) - "This study was registered at the National Clinical Research Center (https://www.medicalresearch.org.cn; No. MR-31-24-053225)."

Journal • Observational data • Real-world evidence • Brain Cancer • CNS Disorders • Glioma • Immunology • Infectious Disease • Inflammation • Oncology • Pneumonia • Respiratory Diseases • Solid Tumor

Case Report: Efficacy of efgartigimod for autonomic dysfunction in patients with autoimmune glial fibrillary acidic protein astrocytopathy: a case series of 3 patients. (PubMed, Front Immunol) - "We observed that after combined treatment with efgartigimod and corticosteroids, the corticosteroid-refractory autonomic dysfunction in GFAP-A patients was significantly alleviated, providing a new clinical treatment option. However, this article is based on a small sample size and requires further validation through larger-scale studies."

Journal • CNS Disorders • Immunology • Inflammation • GFAP

Efgartigimod as adjunctive therapy for immunotherapy-dependent autoimmune diencephalitis: A case report. (PubMed, Medicine (Baltimore)) - "This case demonstrates the favorable effect of efgartigimod as an adjunctive therapy for autoimmune diencephalitis, effectively controlling symptoms and improving patient prognosis."

Journal • CNS Disorders • Epilepsy • Immunology • Pain

Efficacy of efgartigimod in the treatment of autoimmune encephalitis: a systematic review. (PubMed, Neurol Sci) - "Efgartigimod holds potential as a safe and effective adjunctive therapy for AE patients, either as adjunctive treatment after inadequate responses to first-line or second-line therapies or as concomitant add-on therapy combined with first-line treatment."

Journal • Review • Immunology • Infectious Disease • Inflammation

Long-Term Efficacy and Safety of Efgartigimod in Sjögren's Disease: Findings from the RHO+ Open-Label Extension Study (ACR Convergence 2026) - No abstract available

Clinical • Sjogren's Syndrome

Efgartigimod and Steroid Pulse Therapy as an Early Fast-acting Treatment Strategy for Anti-MUSK Myasthenic Crisis. (PubMed, Intern Med) - "Notably, clinical remission was achieved without plasma exchange or intravenous immunoglobulin administration, and the patient was successfully maintained on tacrolimus without the use of oral corticosteroids. This case suggests that efgartigimod may be effective in acute anti-MUSK MG exacerbations, positioning FcRn inhibition as a promising alternative to conventional fast-acting rescue therapies."

Journal • CNS Disorders • Immunology • Myasthenia Gravis

FcRn Blocker Efgartigimod: Unique Fc Fragment Allowing Rapid, Sustained IgG Reduction Without Reducing Albumin or Increasing Cholesterol (ETA 2026) - "Unlike certain FcRn blockers in clinical development, no reductions in albumin levels, increases in cholesterol, or notable clinical manifestations of these laboratory changes were observed across efgartigimod studies, regardless of cyclic or weekly dosing, suggesting that differences in molecular interactions with FcRn are responsible for the impact on albumin. Efgartigimods unique molecular structure and interactions with FcRn clinically avoid reductions of albumin levels, while effectively targeting IgG autoantibodies across dosing regimens."

Immunology

Efficacy and Safety of Efgartigimod in the Treatment of Guillain-Barré Syndrome(GREAT) (clinicaltrials.gov) - P2 | N=110 | Not yet recruiting | Sponsor: Beijing Tiantan Hospital

New P2 trial

Comparative efficacy of single-cycle intravenous efgartigimod versus intravenous immunoglobulin in generalized myasthenia gravis patients receiving concomitant immunosuppressive therapy. (PubMed, Neuromuscul Disord) - "In conclusion, single-cycle intravenous efgartigimod was associated with faster early clinical improvement compared with IVIg, while outcomes were comparable at months 3 and 6 in the context of real-world immunosuppressive therapies. These findings suggest that efgartigimod may represent an alternative therapeutic option for patients with gMG requiring rapid symptom control."

IO biomarker • Journal • CNS Disorders • Immunology • Myasthenia Gravis

Modulation of Autoantibody-Driven Immune Pathways by Efgartigimod in Sjögren's Disease: Translational Results from RHO (ACR Convergence 2026) - No abstract available

Sjogren's Syndrome

The Drug Reimbursement Evaluation Committee…also recognized the appropriateness for coverage of treatments for myasthenia gravis… (Chosun Biz) - "Handok's 'VYVGART (ingredient name efgartigimod alfa)' and Korea UCB's 'ZILBRYSQ (ingredient name zilucoplan sodium)' were recognized as appropriate for coverage as add-on therapies to standard treatment for adult myasthenia gravis patients who are positive for anti-acetylcholine receptor (AChR) antibodies."

Reimbursement • Immunology • Myasthenia Gravis

Chronic Inflammatory Demyelinating Polyneuropathy: Update on Diagnosis and Treatment. (PubMed, J Clin Neuromuscul Dis) - "The newly FDA-approved FcRn antagonist, efgartigimod alfa, provides a targeted option for adults with refractory CIDP. Accurate recognition of atypical and antibody-associated forms is essential for optimizing individualized management and improving outcomes."

Journal • Review • Pain

Fatal Myocarditis Relapse in a Patient with Myasthenia Gravis Treated with Efgartigimod Alfa: A Case Report. (PubMed, J Clin Neuromuscul Dis) - "Although efgartigimod effectively reduces pathogenic IgG, it may not adequately suppress cellular or cytokine-mediated inflammation driving MG-associated myocarditis. Clinicians should exercise caution and maintain cardiac monitoring when prescribing FcRn blockers to patients with a history of myocardial involvement."

Journal • Cardiovascular • CNS Disorders • Congestive Heart Failure • Heart Failure • Immunology • Inflammation • Myasthenia Gravis • Vascular Neurology

Efgartigimod as a salvage therapy for anti-NMDAR encephalitis patients after first-line treatment failure: a case series. (PubMed, Front Neurol) - "Meanwhile, no serious adverse effects were identified. The EFG treatment mechanism might be associated with the elimination of IgG, which contains anti-NMDAR antibodies."

Journal • Retrospective data • CNS Disorders • Hematological Disorders • Immunology

Treatment of anti-GAD65 antibody-positive epilepsy and cerebellar ataxia with efgartigimod: a case report and literature review. (PubMed, Front Immunol) - "The sequential regimen of efgartigimod induction combined with mycophenolate mofetil maintenance appears effective and safe. This therapeutic strategy may provide a valuable reference for the clinical management of such rare autoimmune neurological disorders."

Journal • Review • Ataxia • CNS Disorders • Epilepsy • Immunology • Movement Disorders

Polaris: A Study to Learn About How Well the Medicine Efgartigimod Works to Treat Autoimmune Encephalitis In Children 12 Years or Older and Adults (clinicaltrials.gov) - P2 | N=170 | Recruiting | Sponsor: argenx | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Immunology

Development and Validation of a Nomogram to Predict Minimal Symptom Expression in AChR+ gMG Patients Undergoing Efgartigimod Therapy. (PubMed, Curr Neuropharmacol) - "By integrating new-onset MG, TAMG, baseline QMG score, and ΔIgG, this nomogram provides an individualized prediction of MSE in AChR+ gMG patients treated with efgartigimod, offering a practical clinical tool to help tailor treatment strategies."

Journal • CNS Disorders • Myasthenia Gravis • Solid Tumor • Thymoma • Thymus Cancer

Effect of efgartigimod IV on immature platelet fraction in primary immune thrombocytopenia in the ADVANCE IV trial. (PubMed, Br J Haematol) - "In immune thrombocytopenia, low platelet counts may result from reduced production or increased degradation, both driven by IgG autoantibodies. By blocking FcRn, efgartigimod decreases immature platelet fraction, likely by reducing platelet degradation through decreased IgG autoantibody levels."

Journal • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Neonatal fc receptor (fcrn) inhibition and the implications for inflammatory bowel disease and co-existing immune-mediated diseases. (PubMed, Expert Rev Gastroenterol Hepatol) - No abstract available

Journal • Gastroenterology • Gastrointestinal Disorder • Immunology • Inflammation • Inflammatory Bowel Disease