Disease

Cholestasis

Late-stage therapeutic development

Also known as bile occlusion, obstruction of bile duct.

Clinical trials

Associated genes

Related proteins

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate

Key developments

3 clinical trials expected to report results, the earliest in Q3 2026.

Upcoming Milestones

Q3 2026Long-term Safety and Tolerability Study of Linerixibat for the Treatment of Cholestatic Pruritus in Participants With Primary Biliary Cholangitis
Q1 2027The Use of Near-Infrared Fluorescence Cholangiography With Indocyanine Green (ICG) in the Work Up of Neonatal Cholestasis
Q3 2030Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the European Union (LEAP-EU)

Major developments

Major trial resultsHigh impact

An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children With Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2)

Results posted2026-04-30

Upcoming trial readoutHigh impact

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

Results expected Q3 20302026-06-23

Clinical Milestones6View

Results posted 2

Results expected 3

2026-06-23Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the European Union (LEAP-EU)Results expected Q3 2030
2026-06-22Long-term Safety and Tolerability Study of Linerixibat for the Treatment of Cholestatic Pruritus in Participants With Primary Biliary CholangitisResults expected Q3 2026
2026-02-18The Use of Near-Infrared Fluorescence Cholangiography With Indocyanine Green (ICG) in the Work Up of Neonatal CholestasisResults expected Q1 2027

Recently completed 1

2026-08-01A Single-arm Open-label Safety Study of SMOFlipid to Evaluate the Risk of Developing Essential Fatty Acid Deficiency (EFAD) and/or Parenteral Nutrition-associated Cholestasis (PNAC) in Pediatric Patients 1 Month to 17 Years of Age and in Adult Patients, Who Are Anticipated to Need 8 Weeks or Longer of Parenteral Nutrition TreatmentPrimary completion

Activity timeline6

2026-08-01ClinicalA Single-arm Open-label Safety Study of SMOFlipid to Evaluate the Risk of Developing Essential Fatty Acid Deficiency (EFAD) and/or Parenteral Nutrition-associated Cholestasis (PNAC) in Pediatric Patients 1 Month to 17 Years of Age and in Adult Patients, Who Are Anticipated to Need 8 Weeks or Longer of Parenteral Nutrition TreatmentPrimary completion
2026-06-23ClinicalLong-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the European Union (LEAP-EU)Results expected Q3 2030
2026-06-22ClinicalLong-term Safety and Tolerability Study of Linerixibat for the Treatment of Cholestatic Pruritus in Participants With Primary Biliary CholangitisResults expected Q3 2026
2026-04-30ClinicalAn Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children With Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2)Results posted
2026-02-18ClinicalThe Use of Near-Infrared Fluorescence Cholangiography With Indocyanine Green (ICG) in the Work Up of Neonatal CholestasisResults expected Q1 2027
2026-01-28ClinicalAn Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of TAK-625 in the Treatment of Subjects With Progressive Familial Intrahepatic CholestasisResults posted

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies

Maralixibat chlorideapproved

Approval — Livmarli is indicated for the treatment of: Cholestatic pruritus in patients with Alagil… (2022)

Odevixibatapproved

Accelerated approval — Bylvay is indicated for the treatment of progressive familial intrahepatic cholestasis (P… (2021)

Clinical trials

13 sponsors · 2 new · 1 completed in the last 12 months (net +2)

The current development programme across all trial phases.

Clinical programme

All trials

Active

Late-stage

Completed

Late-stage studies

Phase 4 · Recruiting · Mirum Pharmaceuticals, Inc.

Long-term Safety and Tolerability Study of Linerixibat for the Treatment of Cholestatic Pruritus in Participants With Primary Biliary Cholangitis

Phase 3 · Active, not recruiting · GlaxoSmithKline

A Single-arm Open-label Safety Study of SMOFlipid to Evaluate the Risk of Developing Essential Fatty Acid Deficiency (EFAD) and/or Parenteral Nutrition-associated Cholestasis (PNAC) in Pediatric Patients 1 Month to 17 Years of Age and in Adult Patients, Who Are Anticipated to Need 8 Weeks or Longer of Parenteral Nutrition Treatment

Phase 4 · Recruiting · Fresenius Kabi

An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children With Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2)

Phase 3 · Completed · Albireo, an Ipsen Company

An Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of TAK-625 in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis

Phase 3 · Completed · Takeda

Fish Oil Lipid Emulsion for the Treatment of Parenteral-Nutrition-Associated Liver Disease in Infants

Phase 2/3 · Completed · University of Oklahoma

Recruiting

Phase 4 · Recruiting · Mirum Pharmaceuticals, Inc.

Phase 4 · Recruiting · Fresenius Kabi

The Use of Near-Infrared Fluorescence Cholangiography With Indocyanine Green (ICG) in the Work Up of Neonatal Cholestasis

Early Phase 1 · Recruiting · University of Alabama at Birmingham

Recently completed

Multicenter Comparative Randomized Study to Assess Safety and Efficacy and Select the Optimal Dosage Regimen of REMAXA®, Enteric-coated Tablets, in Comparison With REMAXOL®, Solution for Infusions, in Patients With Intrahepatic Cholestasis Caused by Chronic Diffuse Liver Diseases

Phase 2/3 · Completed · POLYSAN Scientific & Technological Pharmaceutical Company

An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children With Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2)

Phase 3 · Completed · Albireo, an Ipsen Company

An Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of TAK-625 in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis

Phase 3 · Completed · Takeda

Comparison of Composite Lipid Emulsion Containing Fish Oil to Soy-based Lipid Reduction for Cholestasis Prevention in Neonates Requiring Abdominal Surgery

Phase 3 · Completed · Indiana University

Effect of Lipid Intake on Direct Hyperbilirubinemia in Late Preterm and Term Infants With Gastrointestinal Surgical Problems.

Phase 3 · Completed · University of Rochester

Fish Oil Lipid Emulsion for the Treatment of Parenteral-Nutrition-Associated Liver Disease in Infants

Phase 2/3 · Completed · University of Oklahoma

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals

2022emaApproval — Maralixibat chloride· Livmarli is indicated for the treatment of: Cholestatic pruritus in patients with Alagille syndrome (ALGS) 2 months of age and older, Progressive familial intrahepatic cholestasis (PFIC) in patients 3 months of age and older. Livmarli tablets is indicated in adults and adolescents 12 years and older for the treatment of: Cholestatic pruritus in patients with Alagille syndrome (ALGS), Progressive familial intrahepatic cholestasis (PFIC). source ↗

2021emaAccelerated approval — Odevixibat· Bylvay is indicated for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older (see sections 4.4 and 5.1). source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Associated genes

12 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

USP530.62 ABCC20.48 CFTR0.47 BAAT0.47 AKR1D10.47 FAH0.46 LIPA0.46 MPV170.38 KIF120.38 DGUOK0.37 SLC51B0.28 MMP150.23

Disease biology

1 match

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Cystic fibrosis transmembrane conductance regulatorvia CFTR0.47

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

Impairment of bile flow due to obstruction in small bile ducts (INTRAHEPATIC CHOLESTASIS) or obstruction in large bile ducts (EXTRAHEPATIC CHOLESTASIS).

Synonyms

bile occlusion, obstruction of bile duct

References & data sources

Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
Related entities are derived from literature co-mention (studied together) — associative, not causal.