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efgartigimod PH20

Phase 3

Primary Immune Thrombocytopenia | Monoclonal antibody | Hematology |argenx SE|Last Updated: Jan 12, 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 Enrollment380
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04812925A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune ThrombocytopeniaPHASE3 ACTIVE NOT_RECRUITING 173Nov 17, 2021Oct 1, 2026Jan 12, 202683 United States, Argentina +23
NCT04687072A Study to Evaluate the Efficacy and Safety of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune ThrombocytopeniaPHASE3 COMPLETED 207Dec 16, 2020Oct 9, 2023Oct 31, 2024200 United States, Argentina +30
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Study Endpoints
Primary Endpoints
Incidence, frequency, and severity of adverse events (AEs), AEs of special interest (AESIs), and serious AEs (SAEs)
216 weeks
Vital sign measurement: blood pressure in the overall population
216 weeks
ECG: PR, QT and QRS interval in the overall population
216 weeks
Laboratory safety evaluations: CRP analysis in the overall population
216 weeks
Percentage of Participants With Chronic Immune Thrombocytopenia (ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24
Up to 6 weeks (between Weeks 19 and 24)

A participant was considered a responder for this endpoint (i.e., had a sustained platelet count response) if the participant had platelet counts of ≥50 × 10\^9/L for ≥4 of the 6 analysis visits between Weeks 19 and 24.

Secondary Endpoints
Extent of disease control defined as the percentage of weeks in the trial with platelet counts of ≥50×10E9/L
52 weeks
Proportion of patients with overall platelet count response defined as achieving a platelet count of ≥50×10E9/L on at least 4 occasions at any time during the 52-week treatment period
52 weeks
Mean change from baseline in platelet count at each visit
52 weeks
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
efgartigimod PH20 SCEXPERIMENTALPatients receiving efgartigimod PH20 SC treatment
Placebo PH20 SCPLACEBO_COMPARATORPatients receiving placebo PH20 SC treatment
Interventions
NameTypeDescription
efgartigimod PH20 SCBIOLOGICALSubcutaneous injection with efgartigimod PH20 SC
Placebo PH20 SCOTHERSubcutaneous injection with placebo PH20 SC
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites83

Inclusion Criteria: 1. Ability to understand the requirements of the trial and provide written informed consent (including consent for the use and disclosure of research-related health information), willing and able to comply with the trial protocol procedures (including attending the required tria...

Countries:United StatesArgentinaAustraliaBulgariaChileChinaGeorgiaGreeceIrelandItalyJapanJordanMexicoNew ZealandNorwayPolandPortugalRomaniaRussiaSouth AfricaSouth KoreaThailandTunisiaTurkey (Türkiye)United KingdomDenmarkFranceGermanyIsraelSerbiaSpainTaiwan
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT04812925primaryCompletionDate: changed
LOWMay 24, 2026NCT04812925studyFirstPostDate: changed