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Eculizumab

Phase 3

Atypical Hemolytic Uremic | Small molecule | Hematology |AstraZeneca PLC|Last Updated: Dec 8, 2025

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
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment25
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05876351Eculizumab in Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in ChinaPHASE3 COMPLETED 25Jul 14, 2023May 7, 2025Dec 8, 20256 China
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Study Endpoints
Primary Endpoints
Percentage of Participants With a Complete Thrombotic Microangiopathy (TMA) Response
Up to Week 26

The criteria for complete TMA response were: 1. Normalization of platelet count (defined as platelet count ≥ 150000/microliter (ul). 2. Normalization of lactate dehydrogenase (LDH, defined as LDH ≤ upper limit of normal \[ULN\]). 3. ≥ 25% improvement in serum creatinine from baseline.

Secondary Endpoints
Number of Participants With an Adverse Event (AE)
Up to Week 34
Mean Serum Concentration of Eculizumab
Pre-dose and post-dose at Days 1, 8, 29, 85, and 141; Pre-dose at Day 183
Change From Baseline in Serum Free Complement 5 (C5)
Baseline (Day 1 pre-dose) to Days 1, 8, 29, 85 and 141 (pre-dose and post-dose) and pre-dose at Day 183
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
EculizumabEXPERIMENTALParticipants will receive Eculizumab in a single dose vial.
Interventions
NameTypeDescription
EculizumabDRUGWeight-based doses of Eculizumab will be administered intravenously as an induction dose followed by maintenance dose at Day 8, 15, or 29 depending on weight; then every 2 or 3 weeks, depending upon weight.
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Eligibility Criteria
Age Range0 Years — 99 Years
SexALL
Healthy VolunteersNo
Study Sites6

Inclusion Criteria: 1. Any age weighing ≥ 5 kg 2. Complement treatment naïve with evidence of TMA. 3. History of aHUS prior to kidney transplant,or persistent evidence of TMA at least 4 days after modifying the immunosuppressive regimen. 4. Among participants with onset of TMA postpartum, persisten...

Countries:China
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