Progressive Familial Intrahepatic Cholestasis (PFIC)

Gastroenterology
5
Pipeline Programs
2
Companies
5
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
4
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

2 companies ranked by most advanced pipeline stage

Mirum Pharmaceuticals
Mirum PharmaceuticalsFOSTER CITY, CA
4 programs
1
3
MaralixibatPhase 31 trial
MaralixibatPhase 31 trial
MaralixibatPhase 31 trial
LUM001Phase 21 trial
Active Trials
NCT02057718Completed33Est. May 2020
NCT04185363Completed90Est. Apr 2025
NCT03905330Completed93Est. Sep 2022
+1 more trials
Takeda
TakedaTOKYO, Japan
1 program
1
TAK-625Phase 31 trial
Active Trials
NCT05543187Completed5Est. Jul 2025

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
TakedaTAK-625
Mirum PharmaceuticalsMaralixibat
Mirum PharmaceuticalsMaralixibat
Mirum PharmaceuticalsMaralixibat
Mirum PharmaceuticalsLUM001

Clinical Trials (5)

Total enrollment: 221 patients across 5 trials

NCT05543187TakedaTAK-625

A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC)

Start: Jan 2023Est. completion: Jul 20255 patients
Phase 3Completed
NCT04185363Mirum PharmaceuticalsMaralixibat

An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)

Start: Jan 2020Est. completion: Apr 202590 patients
Phase 3Completed
NCT03905330Mirum PharmaceuticalsMaralixibat

A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)

Start: Jul 2019Est. completion: Sep 202293 patients
Phase 3Completed
NCT03353454Mirum PharmaceuticalsMaralixibat

A Placebo-controlled Study of Maralixibat (SHP625) in Pediatric Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)

Start: Oct 2018Est. completion: Jun 20200
Phase 3Withdrawn
NCT02057718Mirum PharmaceuticalsLUM001

Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 (Maralixibat) in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic Cholestasis

Start: Mar 2014Est. completion: May 202033 patients
Phase 2Completed

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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

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

Related Indications

Hepatic Impairment(83)Gastroesophageal Reflux Disease(40)Irritable Bowel Syndrome(31)Gastroesophageal Reflux(27)Celiac Disease(25)Liver Transplantation(24)Liver Cirrhosis(24)Hepatic Insufficiency(23)Liver Diseases(15)Gastroesophageal Reflux Disease (GERD)(14)