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Iptcaopan

Phase 3

Atypical Hemolytic Uremic Syndrome | Small molecule | Hematology |Novartis AG|Last Updated: Jun 4, 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
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment65
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05795140Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUSPHASE3 RECRUITING 65 — — May 8, 2024Jul 27, 2032Jun 4, 20269 Brazil, China +4
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Study Endpoints
Primary Endpoints
Number of participants with adverse events and serious adverse events
Throughout the study duration, up to 4 years

Number of participants with adverse events and serious adverse events, including abnormal safety laboratory parameters, vital signs and ECGs

Secondary Endpoints
Number of participants with absence of TMA manifestation without the use of anti-C5 antibody
Throughout the study duration, up to 4 years
Number of participants with complete TMA response status without the use of anti-C5 antibody therapy
Throughout the study duration, up to 4 years
Estimated glomerular filtration rate (eGFR)
Throughout study duration, up to 4 years
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Iptacopan 200 mgEXPERIMENTALOpen label , single arm
Interventions
NameTypeDescription
Iptcaopan 200 mgDRUGOpen label, participant specific kits, hard gelatin capsules to be taken twice a day
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Eligibility Criteria
Age Range18 Years — 100 Years
SexALL
Healthy VolunteersNo
Study Sites9

Inclusion Criteria: 1. Signed informed consent must be obtained prior to participation in the open label extension study 2. Willing and able to comply with the study Schedule of Activities 3. Participants who have completed the full study treatment period of any prior "Novartis sponsored" iptacopan...

Countries:BrazilChinaCzechiaIndiaJapanTurkey (Türkiye)
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Recent Changes (Last 90 Days)
LOWJun 4, 2026NCT05795140lastUpdatePostDate: changed
LOWJun 4, 2026NCT05795140lastUpdatePostDate: changed
LOWJun 4, 2026NCT05795140lastUpdatePostDate: changed
LOWJun 4, 2026NCT05795140lastUpdatePostDate: changed
HIGHMay 26, 2026NCT05795140Enrollment: 125 → 65
LOWMay 24, 2026NCT05795140studyFirstPostDate: changed