Alagille Syndrome

9
Pipeline Programs
3
Companies
12
Clinical Trials
3 recruiting
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
5
0
2
2
Early DiscoveryClinical DevelopmentMarket

On Market (1)

Approved therapies currently available

Ipsen
BYLVAYApproved
odevixibat
Ipsen
Ileal Bile Acid Transporter Inhibitor [EPC]oral2021

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Mirum Pharmaceuticals
Mirum PharmaceuticalsFOSTER CITY, CA
9 programs
5
1
Livmarli Oral ProductPhase 41 trial
LUM001Phase 21 trial
LUM001Phase 21 trial
LUM001Phase 21 trial
LUM001Phase 21 trial
+4 more programs
Active Trials
NCT06193928Recruiting70Est. Sep 2030
NCT02131623Completed23Est. Mar 2015
NCT04530994Approved For Marketing
+6 more trials
Ipsen
IpsenChina - Tianjin
2 programs
2
1
OdevixibatPhase 31 trial
OdevixibatPhase 31 trial
Active Trials
NCT05035030Recruiting70Est. Dec 2026
NCT04674761Completed52Est. Sep 2022
Alliance Pharmaceuticals
1 program
Assessment for bleeding disorder with tailored post-operative careN/A1 trial
Active Trials
NCT05846854Unknown40Est. Nov 2024

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
Mirum PharmaceuticalsLivmarli Oral Product
IpsenOdevixibat
IpsenOdevixibat
Mirum PharmaceuticalsLUM001
Mirum PharmaceuticalsLUM001
Mirum PharmaceuticalsLUM001
Mirum PharmaceuticalsLUM001
Mirum PharmaceuticalsLUM001
Mirum PharmaceuticalsLivmarli
Alliance PharmaceuticalsAssessment for bleeding disorder with tailored post-operative care
Mirum PharmaceuticalsValidation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver Disease

Clinical Trials (12)

Total enrollment: 496 patients across 12 trials

Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome in the European Union (LEAP-EU)

Start: Sep 2025Est. completion: Dec 2030100 patients
Phase 4Recruiting

Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome

Start: Sep 2021Est. completion: Dec 202670 patients
Phase 3Recruiting

Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome

Start: Mar 2021Est. completion: Sep 202252 patients
Phase 3Completed

An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome

Start: Mar 2015Est. completion: Jun 202034 patients
Phase 2Completed

Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome

Start: Nov 2014Est. completion: Nov 201637 patients
Phase 2Completed

Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS)

Start: Oct 2014Est. completion: May 202031 patients
Phase 2Completed

An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS)

Start: Dec 2013Est. completion: Jun 202019 patients
Phase 2Completed

Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome

Start: Aug 2013Est. completion: Mar 201520 patients
Phase 2Completed

A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)

N/AApproved For Marketing

Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)

Start: Sep 2023Est. completion: Sep 203070 patients
N/ARecruiting
NCT05846854Alliance PharmaceuticalsAssessment for bleeding disorder with tailored post-operative care

Decreasing Hemorrhage Risk in Children With Alagille Syndrome

Start: Apr 2023Est. completion: Nov 202440 patients
N/AUnknown
NCT02131623Mirum PharmaceuticalsValidation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver Disease

Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver Disease

Start: Jul 2014Est. completion: Mar 201523 patients
N/ACompleted

Phase Legend

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

Key Insights

2 late-stage (Phase 3) programs — potential near-term approvals
3 actively recruiting trials targeting 496 patients
3 companies competing in this space