Efgartigimod Alfa
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Also known as Argx-113EfgartigimodEfgartigimod alfa fcabEfgartigimod alfa-fcabEfgartigimod alfa-qvfcVyvgart
Summary
Efgartigimod Alfa (CHEMBL4297551) is an approved protein (ATC L04AA58) targeting FCGRT; indicated across 15 conditions including myasthenia gravis and immune system disorder.
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Protein
- ATC class: L04AA58
- Targets: 1 (FCGRT)
- Indications: 15 conditions
- Clinical trials: 60
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL4297551 |
| Name | Efgartigimod Alfa |
| Type | Protein |
| Max phase | 4 |
| ATC | L04AA58 |
Also known as: Argx-113, ARGX-113, Efgartigimod, Efgartigimod alfa, Efgartigimod alfa fcab, Efgartigimod alfa-fcab, Efgartigimod alfa-qvfc, Vyvgart, EFGARTIGIMOD ALFA
Targets
Targets
Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).
| Gene | Target | Action | pAffinity | Cancer dependency | UniProt |
|---|---|---|---|---|---|
| FCGRT | Fc fragment of IgG receptor and transporter | Binding | 0% | P55899 |
Bioactivity
No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).
Target pathways
Aggregated over 1 target gene(s): FCGRT.
Dominant GO biological processes
| GO term | Targets |
|---|---|
| IgG immunoglobulin transcytosis in epithelial cells mediated by FcRn immunoglobulin receptor | 1 |
| immune response | 1 |
Indications & clinical
Indications
15 indications (2 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).
| Indication | Trial phase | MONDO | EFO |
|---|---|---|---|
| myasthenia gravis | 4 | MONDO:0009688 | EFO:0004991 |
| immune system disorder | 4 | MONDO:0005046 | EFO:0000540 |
| autoimmune thrombocytopenic purpura | 3 | MONDO:0008558 | EFO:0007160 |
| bullous pemphigoid | 3 | MONDO:0019082 | EFO:0007187 |
| thrombocytopenia | 3 | MONDO:0002049 | HP:0001873 |
| Guillain-Barre syndrome, familial | 2 | MONDO:0007691 | EFO:0009538 |
| Sjogren syndrome | 2 | MONDO:0010030 | EFO:0000699 |
| kidney disorder | 2 | MONDO:0005240 | EFO:0003086 |
| lupus nephritis | 2 | MONDO:0005556 | EFO:0005761 |
| Guillain-Barre syndrome | 2 | MONDO:0016218 | EFO:0007292 |
| postural orthostatic tachycardia syndrome | 2 | MONDO:0011479 | EFO:1000645 |
| optic neuritis | 2 | MONDO:0005885 | EFO:0007405 |
| autoimmune disease | 1 | MONDO:0007179 | EFO:0005809 |
2 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.
Clinical trials
Total trials: 60.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE3 | 23 |
| PHASE2 | 18 |
| Not specified | 6 |
| PHASE1 | 5 |
| PHASE4 | 4 |
| PHASE2/PHASE3 | 3 |
| PHASE1/PHASE2 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT06860633 | PHASE4 | RECRUITING | Treatment of Myasthenia Gravis Exacerbation or Crisis With Efgartigimod |
| NCT06909214 | PHASE4 | RECRUITING | A Study to Evaluate the Clinical Outcomes of Efgartigimod PH20 SC in Adults With New-onset Generalized Myasthenia Gravis (gMG) |
| NCT07072988 | PHASE4 | RECRUITING | Evaluate the Benefit of Corticoid Sparing in Elderly With Generalized AntiRAch Myasthenia Gravis Treated With IV or SC Efgartigimod |
| NCT06637072 | PHASE4 | COMPLETED | A Study to Assess Adults With CIDP Transitioning From IVIg to Efgartigimod PH20 SC |
| NCT04812925 | PHASE3 | ACTIVE_NOT_RECRUITING | A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia |
| NCT05979441 | PHASE3 | ENROLLING_BY_INVITATION | A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Active Idiopathic Inflammatory Myopathy |
| NCT06392386 | PHASE2/PHASE3 | RECRUITING | A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis |
| NCT06558279 | PHASE3 | ACTIVE_NOT_RECRUITING | A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC in Adults With Ocular Myasthenia Gravis |
| NCT06587867 | PHASE3 | RECRUITING | Seronegative Myasthenia Gravis - Efgartigimod IV |
| NCT06684847 | PHASE3 | ACTIVE_NOT_RECRUITING | A Study of the Efficacy and Safety of Efgartigimod in Patients With Primary Sjögren’s Syndrome |
| NCT06765161 | PHASE3 | RECRUITING | Efgartigimod in IVIG Dependent Myasthenia Gravis Patients |
| NCT06885762 | PHASE2/PHASE3 | NOT_YET_RECRUITING | Efgartigimod for the Treatment of Guillain-Barré Syndrome |
| NCT07217587 | PHASE3 | RECRUITING | Comparative Efficacy of Nipocalimab and Efgartigimod in Participants With Generalized Myasthenia Gravis |
| NCT07231523 | PHASE3 | NOT_YET_RECRUITING | EFG vs IVIG in TAMG |
| NCT07570316 | PHASE3 | NOT_YET_RECRUITING | A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC PFS in Adult Participants With Graves’ Disease |
| NCT07596849 | PHASE3 | NOT_YET_RECRUITING | A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC PFS in Adult Participants With Graves’ Disease. |
| NCT03669588 | PHASE3 | COMPLETED | An Efficacy and Safety Study of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalized Muscle Weakness |
| NCT03770403 | PHASE3 | COMPLETED | A Safety and Tolerability Study of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalized Muscle Weakness. |
| NCT04188379 | PHASE3 | COMPLETED | A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP). |
| NCT04225156 | PHASE3 | COMPLETED | A Long-term Study to Assess the Safety and Efficacy of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP). |
| NCT04274452 | PHASE3 | WITHDRAWN | A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP) |
| NCT04598451 | PHASE3 | COMPLETED | A Study to Assess the Efficacy and Safety of a Subcutaneous Formulation of Efgartigimod PH20 SC in Adults With Pemphigus (Vulgaris or Foliaceus) |
| NCT04598477 | PHASE3 | TERMINATED | A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod PH20 SC in Adults With Pemphigus (Vulgaris or Foliaceus) |
| NCT04687072 | PHASE3 | COMPLETED | A Study to Evaluate the Efficacy and Safety of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia |
| NCT04735432 | PHASE3 | COMPLETED | Evaluating the Pharmacodynamic Noninferiority of Efgartigimod PH20 SC Administered Subcutaneously as Compared to Efgartigimod Administered Intravenously in Patients With Generalized Myasthenia Gravis |
| NCT04818671 | PHASE3 | COMPLETED | Evaluating the Long-Term Safety and Tolerability of Efgartigimod PH20 SC Administered Subcutaneously in Patients With Generalized Myasthenia Gravis |
| NCT05267600 | PHASE2/PHASE3 | COMPLETED | A Phase 2/3 Study of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid |
| NCT05681481 | PHASE3 | TERMINATED | A Phase 3 Study to Evaluate the Long-term Safety, Tolerability and Efficacy of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid |
| NCT06307613 | PHASE3 | TERMINATED | A Study of Efgartigimod PH20 SC Given by Prefilled Syringe in Adults With Thyroid Eye Disease |
| NCT06307626 | PHASE3 | TERMINATED | A Study of Efgartigimod PH20 SC Given by Prefilled Syringe in Adults With Thyroid Eye Disease. |
| NCT04280718 | PHASE2 | ACTIVE_NOT_RECRUITING | A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP, an Autoimmune Disorder That Affects the Peripheral Nerves) |
| NCT06221501 | PHASE2 | NOT_YET_RECRUITING | Perioperative Efgartigimod for Thymoma and Myasthenia Gravis |
| NCT06453694 | PHASE2 | RECRUITING | Efgartigimod for the Treatment of Acute Optic Neuritis |
| NCT06503731 | PHASE2 | RECRUITING | A Study to Evaluate the Safety and Tolerability of Efgartigimod PH20 SC Given by Prefilled Syringe in Kidney Transplant Recipients With Antibody-Mediated Rejection (AMR) |
| NCT06528392 | PHASE2 | NOT_YET_RECRUITING | Efgartigimod for Stiff Person Syndrome (ESPS) |
| NCT06655155 | PHASE2 | RECRUITING | A Study to Assess the Efficacy and Safety of Efgartigimod PH20 SC in Adults With Systemic Sclerosis |
| NCT06831058 | PHASE2 | RECRUITING | A Pilot Study of Efgartigimod for Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP) |
| NCT07011589 | PHASE1/PHASE2 | NOT_YET_RECRUITING | Targeting Collagen VII Antibodies in Bullous Diseases Using Efgartigimod IV (VYVGART) |
| NCT07025330 | PHASE2 | RECRUITING | A Study of Efgartigimod in Patients With IgG4-Related Disease |
| NCT07583641 | PHASE2 | NOT_YET_RECRUITING | A Study to Learn About How Well the Medicine Efgartigimod Works to Treat Autoimmune Encephalitis In Children 12 Years or Older and Adults |
Clinical evidence (CIViC)
No CIViC predictive evidence (expected for non-precision-medicine drugs).
Pharmacology
Pharmacogenomics
No PharmGKB pharmacogenomic data curated for this drug.
Related molecules
Related molecules
No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).