The clinical evidence base for Efgartigimod PH20 SC comprises 40 peer-reviewed publications across 15 journals, 10 pivotal-trial primary-outcome rows reported to ClinicalTrials.gov, spanning indications including Myasthenia Gravis, Purpura, Thrombocytopenic, Idiopathic, Post-Acute COVID-19 Syndrome, and Polyradiculoneuropathy, Chronic Inflammatory Demyelinating. Most recent publication: Efgartigimod in Sjögren's disease: a phase 2, randomised, placebo-controlled, parallel-group, double-blinded, proof-of-concept study (RHO)., Ann Rheum Dis, 2026.
Top peer-reviewed publications
Curated set of pivotal-trial result papers and recent publications in high-tier journals.
Perioperative Safety and Efficacy of Efgartigimod for Thymoma-Associated Myasthenia Gravis: A Prospective, Multicenter, Phase II Clinical Trial.
Wang S, Zhu M, Dong J, et al. · J Thorac Oncol · 2025
The efficacy, safety, and pharmacokinetics/pharmacodynamics of telitacicept following efgartigimod in generalized myasthenia gravis: protocol of a randomized controlled trial.
Li J, Zhang Y, Deng Y, et al. · Front Immunol · 2025
Efficacy and Safety of Subcutaneous Efgartigimod PH20 in Adults With Primary Immune Thrombocytopenia (ADVANCE SC): A Multicenter, Randomized, Double-Blinded, Placebo-Controlled, Phase 3 Trial.
Cooper N, Broome CM, Miyakawa Y, et al. · Am J Hematol · 2026
Efficacy and safety of the neonatal Fc receptor inhibitor efgartigimod in adults with primary immune thrombocytopenia (ADVANCE IV): a multicentre, randomised, placebo-controlled, phase 3 trial.
Broome CM, McDonald V, Miyakawa Y, et al. · Lancet · 2023
Safety, tolerability, and efficacy of subcutaneous efgartigimod in patients with chronic inflammatory demyelinating polyradiculoneuropathy (ADHERE): a multicentre, randomised-withdrawal, double-blind, placebo-controlled, phase 2 trial.
Allen JA, Lin J, Basta I, et al. · Lancet Neurol · 2024
Percentage of Participants With Chronic ITP With a Sustained Platelet Count Response Defined as Achieving Platelet Counts of at Least 50×10^9/L for at Least 4 of the 6 Visits Between Week 19 and 24 of the Trial.
The efficacy, safety, and pharmacokinetics/pharmacodynamics of telitacicept following efgartigimod in generalized myasthenia gravis: protocol of a randomized controlled trial.
Long-term safety, tolerability, and efficacy of efgartigimod (ADAPT+): interim results from a phase 3 open-label extension study in participants with generalized myasthenia gravis.
Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial.
Efficacy and Safety of Subcutaneous Efgartigimod PH20 in Adults With Primary Immune Thrombocytopenia (ADVANCE SC): A Multicenter, Randomized, Double-Blinded, Placebo-Controlled, Phase 3 Trial.
Efficacy and safety of the neonatal Fc receptor inhibitor efgartigimod in adults with primary immune thrombocytopenia (ADVANCE IV): a multicentre, randomised, placebo-controlled, phase 3 trial.
Safety, tolerability, and efficacy of subcutaneous efgartigimod in patients with chronic inflammatory demyelinating polyradiculoneuropathy (ADHERE): a multicentre, randomised-withdrawal, double-blind, placebo-controlled, phase 2 trial.
In studies for chronic idiopathic thrombocytopenic purpura (ITP), the ADVANCE trial (NCT04188379) reported that **21.8%** of participants receiving **efgartigimod** achieved a sustained platelet count response between Week 19 and 24, compared to 5.0% on placebo. In the ADVANCE SC trial (NCT04687072), 13.7% of participants on efgartigimod PH20 SC achieved a sustained platelet count response between Weeks 19 and 24, versus 16.2% on placebo PH20 SC. For pemphigus, the ADDRESS trial (NCT04598451) showed that **44 participants** receiving efgartigimod PH20 SC achieved complete clinical remission on minimal prednisone therapy, compared to 20 participants on placebo PH20 SC. The ADDRESS+ trial (NCT04598477) monitored safety outcomes up to 60 weeks.
For the **efgartigimod-efgartigimod PH20 SC
All Efgartigimod PH20 SC publications (40)
2026 (3 papers)
Efgartigimod in Sjögren's disease: a phase 2, randomised, placebo-controlled, parallel-group, double-blinded, proof-of-concept study (RHO).
Peene I, Verstappen GM, Arends S, et al. · Ann Rheum Dis · 2026 · Derived
Efficacy and Safety of Subcutaneous Efgartigimod PH20 in Adults With Primary Immune Thrombocytopenia (ADVANCE SC): A Multicenter, Randomized, Double-Blinded, Placebo-Controlled, Phase 3 Trial.
Cooper N, Broome CM, Miyakawa Y, et al. · Am J Hematol · 2026 · Derived
The efficacy, safety, and pharmacokinetics/pharmacodynamics of telitacicept following efgartigimod in generalized myasthenia gravis: protocol of a randomized controlled trial.
Li J, Zhang Y, Deng Y, et al. · Front Immunol · 2025 · Derived
Safety, tolerability, and efficacy of subcutaneous efgartigimod in patients with chronic inflammatory demyelinating polyradiculoneuropathy (ADHERE): a multicentre, randomised-withdrawal, double-blind, placebo-controlled, phase 2 trial.
Allen JA, Lin J, Basta I, et al. · Lancet Neurol · 2024 · Derived
Long-term safety, tolerability, and efficacy of efgartigimod (ADAPT+): interim results from a phase 3 open-label extension study in participants with generalized myasthenia gravis.
Howard JF, Bril V, Vu T, et al. · Front Neurol · 2024 · Background
Efficacy and safety of the neonatal Fc receptor inhibitor efgartigimod in adults with primary immune thrombocytopenia (ADVANCE IV): a multicentre, randomised, placebo-controlled, phase 3 trial.
Broome CM, McDonald V, Miyakawa Y, et al. · Lancet · 2023 · Derived
Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study.
Bril V, Drużdż A, Grosskreutz J, et al. · Lancet Neurol · 2023 · Background
Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial.
Howard JF, Bril V, Vu T, et al. · Lancet Neurol · 2021 · Background
Publications: ctgov.study_references (PubMed PMIDs auto-attached by ClinicalTrials.gov to each trial), reference types RESULT, DERIVED, and BACKGROUND.
Per-arm outcome values: ctgov.outcome_measurements joined to ctgov.design_outcomes where outcome_type = 'PRIMARY', restricted to phase PHASE3 and PHASE2/PHASE3.
This page summarizes published evidence for general reference and does not constitute medical advice. For clinical decisions, consult the linked primary publications and your healthcare provider. Data sourced from PubMed and the ClinicalTrials.gov / AACT database maintained by the Clinical Trials Transformation Initiative (CTTI).