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

Ipsen
IpsenChina - Tianjin
2 programs
1
A4250Phase 31 trial
OdevixibatN/A1 trial
Active Trials
NCT04483531Approved For Marketing
NCT03659916Completed116Est. Dec 2025
Mirum Pharmaceuticals
Mirum PharmaceuticalsFOSTER CITY, CA
1 program
1
MaralixibatPhase 21 trial
Active Trials
NCT04729751Completed27Est. Dec 2024

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

Related Jobs in Gastroenterology

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

1 late-stage (Phase 3) programs — potential near-term approvals
2 companies competing in this space