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

Sustained improvements in patient-reported outcomes after intravenous efgartigimod in adults with primary immune thrombocytopenia in a Phase 3 clinical trial (ADVANCE IV) and the first 52 weeks in its open-label extension study (ADVANCE IV+) (ASH 2025) - P3 | "Participants who received longer-term treatment withefgartigimod (EFG-EFG) continued to demonstrate improvements in HRQoL during TP1 of ADVANCE IV+that were initially observed during the RCT. Ongoing evaluation of EFG's benefit on HRQoL and PROs arebeing evaluated in the ADVANCE NEXT trial (NCT06544499).ClinicalTrials.gov ID: NCT04188379, NCT04225156"

Clinical • P3 data • Patient reported outcomes • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura

Targeted Therapy for Idiopathic Inflammatory Myopathy. (PubMed, J Cachexia Sarcopenia Muscle) - "The targeted therapy landscape in IIM is rapidly expanding, enabling more precise and effective management. These strategies show significant promise in improving outcomes for patients with refractory disease. Future efforts should focus on optimizing treatment selection through biomarker discovery, conducting larger randomized trials to strengthen the evidence base, and managing long-term safety."

IO biomarker • Journal • Review • Immunology • Infectious Disease • Inflammation • Interstitial Lung Disease • Myositis • Pulmonary Disease • Respiratory Diseases

Steroid-sparing in myasthenia gravis: How much is efgartigimod, and How much is carryover? (PubMed, J Neurol Sci) - No abstract available

Journal • CNS Disorders • Myasthenia Gravis

Successful Treatment of Evans Syndrome With efgartigimod: A Case Report. (PubMed, Am J Ther) - No abstract available

Journal

Zai Lab Limited…announced that the…medicines and indications have been renewed in the 2025 National Reimbursement Drug List (NRDL) released by China’s National Healthcare Security Administration (NHSA) (FinancialContent) - "VYVGART (efgartigimod alfa injection) is renewed for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive."

Reimbursement • Myasthenia Gravis

EPIC: Comparative Efficacy of Nipocalimab and Efgartigimod in Participants With Generalized Myasthenia Gravis (clinicaltrials.gov) - P3 | N=115 | Recruiting | Sponsor: Janssen Research & Development, LLC | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Myasthenia Gravis

Real-World Efgartigimod Effectiveness in CIDP: A Prospective Study (clinicaltrials.gov) - P=N/A | N=200 | Recruiting | Sponsor: argenx

New trial • Real-world evidence • Pain

Case Report: A Patient with Lambert-Eaton Myasthenic Syndrome Successfully Treated with Efgartigimod. (PubMed, Int Med Case Rep J) - "The patient demonstrated substantial clinical improvement following treatment. Nevertheless, more clinical studies are required to verify whether efgartigimod offers a novel treatment approach for patients with LEMS."

Journal • Lung Cancer • Oncology • Small Cell Lung Cancer • Solid Tumor

Case Report: Efgartigimod as an additional therapy for MOG antibody-associated disease overlapping GFAP-IgG. (PubMed, Front Immunol) - "We report a case of MOGAD-overlapping GFAP IgG treated with combination therapy of steroids and efgartigimod. This case enhances our understanding of the clinical manifestations of overlapping syndromes and expands the treatment options for this disorder."

Journal • Achromatopsia • CNS Disorders • Immunology • Pain • Solid Tumor • GFAP

What is in the Neuromuscular Junction Literature? (PubMed, J Clin Neuromuscul Dis) - "The role of thymectomy in older patients with MG is considered. The medical treatment section addresses corticosteroid regimens, intravenous immunoglobulin as maintenance therapy, a phase 3 study of the recently approved neonatal Fc receptor (FcRN) blocker nipocalimab, use of complement inhibitors and FcRN blockers in general, regimens for efgartigimod, and positive studies on the interleukin-16 receptor monoclonal antibody (Ab) satralizumab and the CD19 B-cell-depleting monoclonal Ab inebilizumab."

Journal • Review • CNS Disorders • Myasthenia Gravis • Ophthalmology • Thyroid Eye Disease • IL16

ARGX-113-2409: A Study of Efgartigimod IV in Participants From 12 Years to Less Than 18 Years of Age With Chronic Immune Thrombocytopenia (ITP) (clinicaltrials.gov) - P2/3 | N=24 | Recruiting | Sponsor: argenx | Not yet recruiting ➔ Recruiting | Trial completion date: Mar 2030 ➔ Oct 2030 | Trial primary completion date: Mar 2028 ➔ Oct 2028

Enrollment open • Trial completion date • Trial primary completion date • Hematological Disorders • Immune Thrombocytopenic Purpura • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura

Efgartigimod non-responders after the first treatment cycle in generalized myasthenia gravis: a retrospective analysis of predictive factors. (PubMed, Front Neurol) - "Responders were defined by a reduction of >2 points in MG-ADL or >3 points in QMG score after one cycle, whereas non-responders showed improvement below these thresholds and subsequently responded to eculizumab. Comorbidities including thymoma, other tumors, thyroid disease, and additional autoimmune disorders may predict reduced response to efgartigimod in gMG patients. Systematic evaluation of these factors could help optimize treatment selection."

Biomarker • Journal • Retrospective data • CNS Disorders • Endocrine Disorders • Immunology • Myasthenia Gravis • Oncology • Solid Tumor • Thyroid Gland Carcinoma

Case Report: KLHL11 encephalitis in a female patient with dual primary malignancies of breast and lung cancer: response to FcRn inhibitor therapy. (PubMed, Front Immunol) - "Following immunotherapy with a neonatal Fc receptor (FcRn) inhibitor (efgartigimod) and resection of tumors, the patient achieved complete symptomatic remission with no recurrence...This is the first report of FcRn inhibitor in the treatment of KLHL11 encephalitis. This case demonstrated a rare association of KLHL11 encephalitis with breast and lung cancer, and expanded on the clinical manifestations, associated tumor types, and treatment options of KLHL11 encephalitis in women."

Journal • Ataxia • Breast Cancer • CNS Disorders • Epilepsy • Germ Cell Tumors • Immunology • Lung Adenocarcinoma • Lung Cancer • Movement Disorders • Oncology • Otorhinolaryngology • Solid Tumor • Testicular Cancer • Vertigo

Olink Proteomics Analysis Reveals Heterogeneous Responses to FcRn Blockade in Anti-AChR Antibody-Positive Myasthenia Gravis: FGF-19 as a Novel Biomarker. (PubMed, Ann Clin Transl Neurol) - "EFG treatment can alleviate the clinical symptoms and decrease IgG levels in patients with MG. The downregulated expression following EFG treatment, along with the findings of in vitro experiments, indicates that FGF-19 may not only act as a biomarker for the efficacy of EFG, but also play a role in promoting the differentiation of B cells into plasma cells, thereby offering a novel target for therapeutic interventions."

Biomarker • Heterogeneity • Journal • CNS Disorders • Inflammation • Myasthenia Gravis • FGF • FGF19

Mohs Surgery as a Precipitant of Bullous Disease: A Review of Reported Cases and Translational Implications (ISDS 2025) - "Five received systemic corticosteroids, often with azathioprine, doxycycline, or niacinamide; one improved with topical and intralesional therapy...Agents such as IL-1β inhibitors (e.g., anakinra), complement antagonists (e.g., avacopan), and FcRn blockers (e.g., efgartigimod) target relevant immune pathways and warrant exploration in refractory or rapidly progressive cases. While uncommon, PV, PF, and BP may emerge after MMS and mimic typical postoperative complications. Prompt biopsy and immunofluorescence facilitate early diagnosis and immunosuppressive therapy, minimizing progression and promoting wound healing."

Clinical • Review • Surgery • Bullous Pemphigoid • Contact Dermatitis • Dermatitis • Dermatology • Dermatopathology • Immunology • Infectious Disease • Pemphigus Vulgaris • IL1B

Efgartigimod for generalized myasthenia gravis in the extreme elderly (≥80 years): a multicenter retrospective real-world study. (PubMed, Front Immunol) - "This multicenter study demonstrated that efgartigimod was efficacious and safe in the elderly MG over 80 years of age. Elderly patients with MGAE presented to benefit the most from efgartigimod treatment."

Clinical • IO biomarker • Journal • Real-world evidence • Retrospective data • CNS Disorders • Infectious Disease • Myasthenia Gravis • Pain • Respiratory Diseases

Intravenous Efgartigimod or Intravenous Immunoglobulin in Guillain-Barre Syndrome: An Observational Multicenter Study. (PubMed, J Peripher Nerv Syst) - "Based on our preliminary findings, intravenous efgartigimod may be potentially tolerated in GBS over the short term. However, this study cannot establish comparative efficacy given the methodological limitations, and large-scale well-controlled head-to-head trials remain imperative."

Clinical • Journal • Observational data • Complement-mediated Rare Disorders • Immunology • Pain

Efficacy and safety of intravenous efgartigimod for chronic inflammatory demyelinating polyneuropathy: A case series real-world study. (PubMed, J Neuroimmunol) - "This case series real-world study support the efficacy and safety of efgartigimod treatment in CIDP patients, benefit on partially reducing the usage of glucocorticoids and immunosuppressants. It was well tolerated across age groups. Larger prospective studies are needed to refine dosing and establish guidelines for CIDP management."

Journal • Real-world evidence • Dermatology • Pain • Pruritus

Efgartigimod Combined with Glucocorticoids in the Treatment of Severe Generalized Myasthenia Gravis: A Single-Center, Retrospective Cohort Study. (PubMed, Neurol Ther) - P4 | "Efgartigimod combined with 250 mg IVMP is associated with improvement of symptoms, reduction of transient exacerbations, and adverse event rates compared with higher IVMP doses or IVMP monotherapy, offering a promising regimen for severe gMG."

Journal • Retrospective data • CNS Disorders • Myasthenia Gravis

Efgartigimod as Fast-Acting Rescue Therapy in Very Late-Onset Myasthenia Gravis. (PubMed, Neurol Ther) - "Intravenous efgartigimod was administered as rescue therapy with rapid and sustained clinical improvement. This case supports the utility of efgartigimod in MC unresponsive to conventional therapies, including in older patients with significant comorbidities."

Journal • CNS Disorders • Critical care • Myasthenia Gravis

EFG vs IVIG in TAMG (clinicaltrials.gov) - P3 | N=64 | Not yet recruiting | Sponsor: Shanghai Zhongshan Hospital

New P3 trial • CNS Disorders • Myasthenia Gravis • Solid Tumor • Thymoma • Thymus Cancer

Time to Achieve Platelet Count Response after Intravenous Efgartigimod in Adults with Primary Immune Thrombocytopenia: A Phase 3, Multicenter, Double-Blinded, Placebo-Controlled, Randomized Clinical Trial (ADVANCE IV) (ASH 2023) - P3 | "Concurrent oral corticosteroids, immunosuppressants, dapsone, danazol, fostamatinib, and oral thrombopoietin receptor agonists were permitted but required to remain at the entry dosage and frequency. The response rate, regardless of definition, in participants who received EFG continued to increase over time suggesting that some participants may have delayed responses to EFG. These data further demonstrate early and sustained responses with EFG in ITP."

Clinical • P3 data • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Pharmacovigilance analysis of FcRn antagonists in the treatment of myasthenia gravis: A disproportionality analysis based on the FAERS database. (PubMed, Hum Vaccin Immunother) - "This study aimed to evaluate the safety profiles of FcRn antagonists, efgartigimod alfa and rozanolixizumab, in the treatment of myasthenia gravis using real-world adverse event data from the FAERS database. The study identifies novel safety signals and highlights clinically relevant risk patterns, supporting enhanced monitoring in clinical use. It is crucial to emphasize that this disproportionality analysis is exploratory in nature and identifies potential associations, which do not establish causality and require confirmation through further dedicated studies."

Adverse events • Journal • Atrial Fibrillation • Cardiovascular • CNS Disorders • Genito-urinary Cancer • Infectious Disease • Myasthenia Gravis • Nephrology • Oncology • Pain • Prostate Cancer • Sleep Disorder • Solid Tumor

A Study of Efgartigimod in Patients With IgG4-Related Disease (clinicaltrials.gov) - P2 | N=5 | Recruiting | Sponsor: Stanford University | Not yet recruiting ➔ Recruiting

Enrollment open • Inflammation

BioDigit MG-03: Quantification of Change in MG Disease Activity in Individuals With Generalized Myasthenia Gravis (gMG) After Administration of VYVGART® or VYVGART Hytrulo® Using BioDigit MG (clinicaltrials.gov) - P=N/A | N=25 | Not yet recruiting | Sponsor: BioSensics

New trial • CNS Disorders • Myasthenia Gravis