VivoSeq
Gastroenterology

Biliary Atresia

19

Pipeline Programs↗

4

Active Trials↗

13

Companies Involved↗

Biliary Atresia is an emerging landscape with 19 programs across 13 companies. The most active players are Mirum Pharmaceuticals, Inc., Eastern Cooperative Oncology Group, Validus Pharmaceuticals. 2 therapies are approved with 2 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

46/100

Pipeline Velocity

0/100

Mech. Concentration

17/100

Completion Rate

79/100

Attrition Rate

13/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

M

Mirum Pharmaceuticals, Inc.

1 program

Phase 2E

Eastern Cooperative Oncology Group

1 program

Phase 2V

Validus Pharmaceuticals

1 program

Phase 2Sanofi logo

Sanofi

2 programs

Phase 2I

Intercept Pharmaceuticals

1 program

Phase 3P

Pfizer's Upjohn has merged with Mylan to form Viatris Inc.

1 program

Phase 3

Pipeline

Programs by Phase

Analysis

Modality Breakdown

83%

Small molecule

19 of 23 classified programs — the dominant modality here.

17%ALL OTHERSCell therapy313%Protein / enzyme14%

Small molecule leads; the ring shows all 23 classified programs, each share of the total. Modality classified for 92% of 25 pipeline programs.

Analysis

Trial Status

23

clinical trials

Completed11 · 48%
Unknown status5 · 22%
Terminated3 · 13%
Recruiting2 · 9%
Not yet recruiting1 · 4%
Enrolling by invitation1 · 4%

Clinical Trials

Trial Activity

23 trials

Stem Cell Applications in Biliary Atresia Patients
Recruiting·NNecmi Kadıoğlu Hospital·N/A·NCT06564740·64 enrolled
Evaluation of the Use of Granulocyte Colony Stimulating Factor (GCSF) in Post Kasai Type 3 Biliary Atresia
Recruiting·NNational Liver Institute, Egypt·N/A·NCT06708572·40 enrolled
Clinical Outcomes of Early Kasai Surgery With Umbilical Cord MSCs in Biliary Atresia
Not yet recruiting·UUniversitas Diponegoro·Phase 1·NCT07011199·14 enrolled
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia (BOLD-EXT)
Enrolling by invitation·AAlbireo, an Ipsen Company·Phase 3·NCT05426733·180 enrolled
GCSF Adjunct Therapy for Biliary Atresia
Completed·—·Phase 1·NCT03395028·6 enrolled
Biliary Atresia, Hepatic Buffer Response and Sevoflurane
Completed·—·N/A·NCT02471209·25 enrolled
The Comparison of Desflurane and Sevoflurane on Postoperative Recovery and Hepatic Function of Biliary Atresia Patients During Kasai Operation
Completed·YYonsei University·N/A·NCT01443572·40 enrolled
N-Acetylcysteine in Biliary Atresia After Kasai Portoenterostomy
Completed·—·Phase 2·NCT03499249·13 enrolled
Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai
Completed·MMirum Pharmaceuticals, Inc.·Phase 2·NCT04524390·75 enrolled
Clinical Study About the Role of COX-2 Inhibitor in Liver Cirrhosis With Biliary Atresia
Completed·YYonsei University·Phase 4·NCT02298218·50 enrolled
Use of Probiotics to Prevent Cholangitis in Children With Biliary Atresia After the Kasai Portoenterostomy
Completed·NNational Taiwan University Hospital·N/A·NCT00166868·30 enrolled
Umbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) Transplantation for Children Suffering From Biliary Atresia
Completed·VVinmec Research Institute of Stem Cell and Gene Technology·Phase 1/2·NCT04522869·20 enrolled
Safety Study of Intravenous Immunoglobulin (IVIG) Post-Portoenterostomy in Infants With Biliary Atresia
Completed·National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)·Phase 1/2·NCT01854827·30 enrolled
A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy
Completed·AAlbireo, an Ipsen Company·Phase 3·NCT04336722·254 enrolled
Biliary Atresia Research Network Northeast (BARNN)
Completed·YYale University·N/A·NCT06184971·320 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)