Biliary Atresia
6
Pipeline Programs
3
Companies
5
Clinical Trials
1
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
0
2
0
2
1
Early DiscoveryClinical DevelopmentMarket
On Market (1)
Approved therapies currently available
Competitive Landscape
3 companies ranked by most advanced pipeline stage
T-TherapeuticsUK - Cambridge
2 programs1
1
FilgrastimPhase 21 trial
Granulocyte Colony-Stimulating FactorPhase 11 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
2028
IpsenOdevixibat
IpsenOdevixibat
T-TherapeuticsFilgrastim
Mirum PharmaceuticalsMaralixibat
T-TherapeuticsGranulocyte Colony-Stimulating Factor
Clinical Trials (5)
Total enrollment: 515 patients across 5 trials
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia
Start: Jul 2022Est. completion: Aug 2028180 patients
Phase 3Enrolling By Invitation
Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)
Start: Jul 2020Est. completion: Jun 2026254 patients
Phase 3Active Not Recruiting
Part II: Granulocyte-Colony Stimulating Factor Adjunct Therapy for Biliary Atresia
Start: Sep 2023Est. completion: Oct 2025
Phase 2Unknown
Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai
Start: Jul 2021Est. completion: Feb 202475 patients
Phase 2Completed
GCSF Adjunct Therapy for Biliary Atresia
Start: Jan 2018Est. completion: Jan 20206 patients
Phase 1Completed
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
2 late-stage (Phase 3) programs — potential near-term approvals
3 companies competing in this space