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efgartigimod

Phase 3

Primary Immune Thrombocytopenia | Monoclonal antibody | Hematology |argenx SE|Last Updated: Mar 16, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment232
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04225156A Long-term Study to Assess the Safety and Efficacy of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).PHASE3 COMPLETED 101 — — Jun 2, 2020Mar 11, 2026Mar 16, 202687 United States, Austria +16
NCT04188379A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).PHASE3 COMPLETED 131 — — Dec 16, 2019Feb 3, 2022Mar 13, 2025121 United States, Austria +16
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Study Endpoints
Primary Endpoints
Frequency and severity of Adverse Events
Up to 60 weeks
Frequency and severity of vital signs
Up to 60 weeks
Frequency and severity of laboratory assessments
Up to 60 weeks
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.
From Week 19 up to Week 24

Percentage of participants with chronic ITP with a sustained platelet count response was 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 study.

Secondary Endpoints
Extent of disease control defined as the percentage of weeks in the trial with platelet counts of ≥50×10E9/L.
Over the 52 weeks of treatment
Percentage of patients with overall platelet count response defined as achieving a platelet count of ≥50×10^9/L on at least 4 occasions at any time during the 52-week treatment period.
Over the 52 weeks of treatment
Mean change from baseline in platelet count at each visit.
Up to 60 weeks, at each visit
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
efgartigimodEXPERIMENTALpatients receiving efgartigimod
PlaceboPLACEBO_COMPARATORPatients receiving placebo
Interventions
NameTypeDescription
efgartigimodBIOLOGICALIntravenous infusion of efgartigimod
PlaceboOTHERIntravenous infusion of placebo
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites87

Inclusion criteria: 1. Ability to understand the requirements of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the trial protocol procedures (including required trial visits). 2. Patients enro...

Countries:United StatesAustriaBelgiumBulgariaCzechiaFranceGeorgiaGermanyHungaryItalyJapanNetherlandsPolandRussiaSpainTurkey (Türkiye)UkraineUnited Kingdom
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Recent Changes (Last 90 Days)
MEDIUMMay 21, 2026NCT04225156TRIAL_REMOVED: changed
MEDIUMMay 21, 2026NCT04225156TRIAL_REMOVED: changed