Progressive Familial Intrahepatic Cholestasis
Gastroenterology
2
Pipeline Programs
2
Companies
3
Clinical Trials
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
1
0
1
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
IpsenChina - Tianjin
2 programs1
A4250Phase 31 trial
OdevixibatN/A1 trial
Active Trials
NCT04483531Approved For Marketing
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
IpsenA4250
Mirum PharmaceuticalsMaralixibat
Clinical Trials (3)
Total enrollment: 143 patients across 3 trials
Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC
Start: Sep 2018Est. completion: Dec 2025116 patients
Phase 3Completed
A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).
Start: Sep 2021Est. completion: Dec 202427 patients
Phase 2Completed
Odevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis
N/AApproved For Marketing
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Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
1 late-stage (Phase 3) programs — potential near-term approvals
2 companies competing in this space