Efgartigimod
Also known as PH20
ARGENX SE ARGX · Generalized Myasthenia Gravis; Graves Disease; Primary Immune Thrombocytopenia; gMG; Active Idiopathic Inflammatory Myopathy
Key figures
Next catalyst
None scheduled
Only dates with a source are listed
Phase
Phase 3
Not recorded
Linked trials
14
From ClinicalTrials.gov
Latest FDA action
Approved
BLA761195,
Recalls, shortages, patents and EU status at a glance
Key information
- Sponsor
- ARGENX SE ARGX Source: Company record for ARGENX SE (opens in a new tab)
- Indications
- Generalized Myasthenia Gravis; Graves Disease; Primary Immune Thrombocytopenia; gMG; Active Idiopathic Inflammatory Myopathy Source: Conditions from ClinicalTrials.gov (opens in a new tab)
- Applications
- BLA 761195 · Standard review Source: Drugs@FDA via openFDA (opens in a new tab) · Action letter for BLA 761195 (opens in a new tab)
- BLA 761304 · Priority review Source: Drugs@FDA via openFDA (opens in a new tab) · Action letter for BLA 761304 (opens in a new tab)
- Designations
- None recorded
- Next catalyst
- None scheduled
Development timeline
- Preclinical Completed
- Phase 1 Completed
- Phase 2 CompletedFirst trial start 26 Oct 20214 linked trials
- Phase 3 Current stageFirst trial start 2 Jun 202014 linked trials
- Filed Not yet
- Approved Not yetFirst approval 17 Dec 2021
Overview
- Mechanism
- Not recorded
- Route
- Not recorded
- Modality
- Not recorded
- Specialties
- Not recorded
Overview text is written by HealthTrade editors.
Key dates
No catalysts yet. Editors add each date together with its source.
FDA designations
No FDA designations are on record for Efgartigimod. Editors add each one with its source.
Filed application
No filed application is on record for Efgartigimod. Approvals are listed under FDA actions.
FDA advisory committees
No FDA advisory committee meetings are on record for Efgartigimod.
Clinical trials
| Trial | Title | Phase | Status | Enrollment | Primary completion | Results posted |
|---|---|---|---|---|---|---|
| NCT07570316ClinicalTrials.gov record for NCT07570316 (opens in a new tab)VitaliThy | A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC PFS in Adult Participants With Graves' Disease | Phase 3 | Recruiting | 230 (estimated) | Jun 2028 (estimated) | No |
| NCT07596849ClinicalTrials.gov record for NCT07596849 (opens in a new tab)VitaliThy | A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC PFS in Adult Participants With Graves' Disease. | Phase 3 | Recruiting | 230 (estimated) | Jun 2028 (estimated) | No |
| NCT07194850ClinicalTrials.gov record for NCT07194850 (opens in a new tab)Advance Jr | A Study of Efgartigimod IV in Participants From 12 Years to Less Than 18 Years of Age With Chronic Immune Thrombocytopenia (ITP) | Phase 2/3 | Recruiting | 24 (estimated) | Oct 2028 (estimated) | No |
| NCT06684847ClinicalTrials.gov record for NCT06684847 (opens in a new tab)Unity | A Study of the Efficacy and Safety of Efgartigimod in Patients With Primary Sjögren's Syndrome | Phase 3 | Active, not recruiting | 631 (actual) | Jul 2027 (estimated) | No |
| NCT06544499ClinicalTrials.gov record for NCT06544499 (opens in a new tab)advance NEXT | A Study to Assess the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia | Phase 3 | Recruiting | 69 (estimated) | Jun 2028 (estimated) | No |
| NCT06392386ClinicalTrials.gov record for NCT06392386 (opens in a new tab)ADAPT Jr SC | A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis | Phase 2/3 | Recruiting | 12 (estimated) | Sep 2027 (estimated) | No |
| NCT06298552ClinicalTrials.gov record for NCT06298552 (opens in a new tab)ADAPT SERON | A Phase 3 Study to Evaluate the Efficacy and Safety of Efgartigimod IV in Patients With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis | Phase 3 | Active, not recruiting | 119 (actual) | 30 Jun 2025 (actual) | Yes First posted Results on ClinicalTrials.gov for NCT06298552 (opens in a new tab) |
Posted results on ClinicalTrials.gov for ADAPT SERON Serious adverse events (Available data included for Part A (up to 8 weeks).): Efgartigimod IV 3 of 58; Placebo 1 of 61 Source: ClinicalTrials.gov results for NCT06298552 (opens in a new tab)· imported Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately. | ||||||
| NCT05979441ClinicalTrials.gov record for NCT05979441 (opens in a new tab)ALKIVIA+ | A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Active Idiopathic Inflammatory Myopathy | Phase 3 | Enrolling by invitation | 240 (estimated) | Sep 2028 (estimated) | No |
| NCT05267600ClinicalTrials.gov record for NCT05267600 (opens in a new tab)BALLAD | A Phase 2/3 Study of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid | Phase 2/3 | Completed | 98 (actual) | 13 Sep 2024 (actual) | Yes First posted Results on ClinicalTrials.gov for NCT05267600 (opens in a new tab) |
Posted results on ClinicalTrials.gov for BALLAD Serious adverse events (up to 46 weeks): Efgartigimod PH20 SC 12 of 50; Placebo PH20 SC 12 of 48 Source: ClinicalTrials.gov results for NCT05267600 (opens in a new tab)· imported Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately. | ||||||
| NCT04980495ClinicalTrials.gov record for NCT04980495 (opens in a new tab)ADAPT NXT | An Open-label Study to Investigate the Clinical Efficacy of Different Dosing Regimens of Efgartigimod IV in Patients With Generalized Myasthenia Gravis | Phase 3 | Completed | 69 (actual) | 24 Aug 2023 (actual) | Yes First posted Results on ClinicalTrials.gov for NCT04980495 (opens in a new tab) |
Posted results on ClinicalTrials.gov for ADAPT NXT Serious adverse events (Part A, up to week 21): Efgartigimod IV - Cyclic Regimen 1 of 17; Efgartigimod IV - Continuous Regimen 7 of 52 Source: ClinicalTrials.gov results for NCT04980495 (opens in a new tab)· imported Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately. | ||||||
| NCT04812925ClinicalTrials.gov record for NCT04812925 (opens in a new tab)ADVANCE SC+ | A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia | Phase 3 | Active, not recruiting | 173 (actual) | Oct 2027 (estimated) | No |
| NCT04833894ClinicalTrials.gov record for NCT04833894 (opens in a new tab)ADAPT Jr | Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis | Phase 2/3 | Active, not recruiting | 15 (actual) | Mar 2027 (estimated) | No |
| NCT04818671ClinicalTrials.gov record for NCT04818671 (opens in a new tab)ADAPTSC+ | Evaluating the Long-Term Safety and Tolerability of Efgartigimod PH20 SC Administered Subcutaneously in Patients With Generalized Myasthenia Gravis | Phase 3 | Completed | 184 (actual) | 31 Dec 2024 (actual) | Yes First posted Results on ClinicalTrials.gov for NCT04818671 (opens in a new tab) |
Posted results on ClinicalTrials.gov for ADAPTSC+ Serious adverse events (3.5 years): Efgartigimod PH20 SC 55 of 180 Source: ClinicalTrials.gov results for NCT04818671 (opens in a new tab)· imported Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately. Publications listing ADAPTSC+ (2), newest first
Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies. | ||||||
| NCT04225156ClinicalTrials.gov record for NCT04225156 (opens in a new tab)ADVANCE+ | A Long-term Study to Assess the Safety and Efficacy of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP). | Phase 3 | Completed | 101 (actual) | 11 Mar 2026 (actual) | No |
Source: ClinicalTrials.gov, U.S. National Library of Medicine. Posted results are shown as ClinicalTrials.gov prints them; results from company and journal sources are entered by editors and listed separately. Full result tables are on ClinicalTrials.gov.
Trial quality
About CT-VCALoading trial quality…
FDA actions
Data provided by the U.S. Food and Drug Administration.
FDA recalls load here.
FDA drug shortages load here.
Orange Book patents load here.
FAERS adverse event counts load here.
Papers and preprints load here.
Community discussion loads here.
EU status (EMA)
EMA's medicines data lists no human medicine with the same active substance as Efgartigimod.
Source: European Medicines Agency (EMA), medicines data download.
Related news
No recent headlines mention this drug.
Sources
- ClinicalTrials.gov NCT07194850 (opens in a new tab) Licence: public-domain. Official registry record this listing came from
- ClinicalTrials.gov NCT05979441 (opens in a new tab) Licence: public-domain. Official registry record this listing came from
- ClinicalTrials.gov NCT06544499 (opens in a new tab) Licence: public-domain. Official registry record this listing came from
- ClinicalTrials.gov NCT07570316 (opens in a new tab) Licence: public-domain. Official registry record this listing came from
- ClinicalTrials.gov NCT07596849 (opens in a new tab) Licence: public-domain. Official registry record this listing came from
- ClinicalTrials.gov NCT04812925 (opens in a new tab) Licence: public-domain. Official registry record this listing came from
- ClinicalTrials.gov NCT06392386 (opens in a new tab) Licence: public-domain. Official registry record this listing came from
- ClinicalTrials.gov NCT06684847 (opens in a new tab) Licence: public-domain. Official registry record this listing came from
- ClinicalTrials.gov NCT04833894 (opens in a new tab) Licence: public-domain. Official registry record this listing came from
- ClinicalTrials.gov NCT04225156 (opens in a new tab) Licence: public-domain. Official registry record this listing came from
- ClinicalTrials.gov NCT06298552 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT05267600 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT04980495 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT04818671 (opens in a new tab) Licence: public-domain. Fetched
- Drugs@FDA via openFDA BLA761195 (opens in a new tab) Licence: cc0. Fetched
- Drugs@FDA via openFDA BLA761304 (opens in a new tab) Licence: cc0. Fetched
Record updated .
Dates come from company and FDA disclosures and can change. Nothing on this page is investment advice.