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TAK-625

Phase 3

Alagille Syndrome (ALGS) | Small molecule | Other |Takeda Pharmaceutical Company Limited|Last Updated: Jan 29, 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 Enrollment7
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05543174A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS)PHASE3 COMPLETED 7 — — Jan 16, 2023Jul 25, 2025Jan 29, 20268 Japan
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Study Endpoints
Primary Endpoints
Change in Fasting Serum Bile Acid (sBA) Levels From Week 18 to Week 22
From Week 18 to Week 22

Change in fasting sBA levels from Week 18 to 22 was reported. Change was calculated as: observed value at Week 22- observed value at Week 18.

Secondary Endpoints
Change in Fasting sBA Levels From Baseline to Week 18
Baseline to Week 18
Change in Weekly Average Severity of Pruritus Measured by Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) From Baseline to Week 18
Baseline to Week 18
Change in Weekly Average Morning Severity of Pruritus Measured by Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) From Baseline to Week 18
Baseline to Week 18
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
TAK-625EXPERIMENTALTAK-625 200 mcg per kilogram, orally, once daily for 1 week. After that, TAK-625 400 mcg per kilogram, orally, once daily after Week 1.
Interventions
NameTypeDescription
TAK-625DRUGTAK-625 200 mcg or 400 mcg per kilogram, orally, once daily
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Eligibility Criteria
Age Range1 Month — N/A
SexALL
Healthy VolunteersNo
Study Sites8

Inclusion Criteria: 1. The participant is Japanese male or female with a body weight \>=3.0 kilograms (kg) and who is \>=1 month of age at the time of informed consent. 2. The participant is diagnosed with ALGS. 3. The participant has one or more of the following evidences of cholestasis: 1. To...

Countries:Japan
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