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biliary atresia - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

biliary atresia

MONDO:0008867

Also known as:atresia of bile ductatresia of bile ductsbiliary atresia, congenital

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Overview

A rare, biliary tract disease characterized by progressive obliterative cholangiopathy of the intra- and extrahepatic bile ducts, occurring in the embryonic/ perinatal period, leading to severe and persistent neonatal jaundice and acholic stool.

TreatmentsNo approved treatments · 2 in pipeline

Clinical Trials26 registered · 17 recruiting

Clinical Presentation

Built from Human Phenotype Ontology (HPO) phenotype annotations and GeneReviews clinical descriptions. Features represent findings documented across known cases — not an exhaustive list of possible symptoms.

Features include very common findings: Jaundice, Cholestasis, and Failure to thrive; and common findings: Decreased liver function, Severe failure to thrive, Enlarged liver (hepatomegaly), and Fat malabsorption and others. 28 total HPO annotations.

Organ System	Phenotype Count	Example Features
Digestive system	9	Jaundice, Cholestasis, Decreased liver function

Diagnosis

Biomarker and diagnostic research for biliary atresia has been reported in the published literature.

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

No approved treatments are currently available for biliary atresia. An additional 2 compounds hold orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for biliary atresia, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.

The following drugs have received orphan drug designation from the FDA for biliary atresia. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor

Prognosis

Phenotype severity distribution: 3 very common features, 13 common features.

Estimated prevalence: 1-9 in 100,000 (Uncommon).

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

26 clinical trials registered, 17 recruiting. Interventions under study include other interventions, drug therapy, procedural interventions, and biologic therapy. Pipeline includes 2 PHASE3, 5 PHASE1, 1 EARLY_PHASE1. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT06764082](https://clinicaltrials.gov/study/NCT06764082)

Data assembled from 6 of 12 sources · Last updated Sep 19, 2026, 3:29 AM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:30391

GARD

Genetic and Rare Diseases Info Center

GARD:12010

Questions for Your Care Team

→Are any of the 26 active clinical trials a good fit for my situation?
→What is the current development status of maralixibat, and when might it become available?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

← Back to All Diseases

Research Type	Count	% of Total
Research summaries	37	36%
Laboratory research	19	18%
Testing and diagnosis research	17	16%
Disease patterns and progression	12	12%
Other research	8	8%
Patient case studies	5	5%
Clinical study results	4	4%
New treatment approaches	2	2%

Kisho

biliary atresia

Overview

Clinical Presentation

Diagnosis

Treatment

Orphan Drug Designations

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Explore clinical features

Clinical Trials

Recent Publications

Latest News & Developments

Ductular Reaction Underlies Development of Portal Hypertension in Biliary Atresia.

Liver graft as a 'Trojan horse': manifestation of variegate porphyria in an 11-month-old girl with biliary atresia after living-related liver transplantation.

Unveiling Hepatic Protein Alterations in Neonatal and Infant Biliary Atresia.

biliary atresia

Overview

Clinical Presentation

Diagnosis

Treatment

Orphan Drug Designations

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Subtypes

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Explore clinical features

Clinical Trials

Recent Publications

Latest News & Developments

Ductular Reaction Underlies Development of Portal Hypertension in Biliary Atresia.

Liver graft as a 'Trojan horse': manifestation of variegate porphyria in an 11-month-old girl with biliary atresia after living-related liver transplantation.

Unveiling Hepatic Protein Alterations in Neonatal and Infant Biliary Atresia.