Disease

Primary biliary cholangitis

Late-stage therapeutic development

Also known as Hanot syndrome, PBC, biliary liver cirrhosis, cholestatic cirrhosis+6 more

Hanot syndrome, PBC, biliary liver cirrhosis, cholestatic cirrhosis, chronic non-suppurative destructive cholangitis, chronic nonsuppurative destructive cholangitis, primary Bilary cirrhosis (PBC), primary biliary cirrhosis, biliary cirrhosis, primary, familial primary biliary cirrhosis.

Clinical trials

Associated genes

Related proteins

Current focus

Bile acid biologyTherapeutic developmentInflammation & immunity

Latest activity

beta

Recent clinical, regulatory, research and industry developments relating to this disease.

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Volixibat in the Treatment of Cholestatic Pruritus in Patients With Primary Biliary Cholangitis

Clinical trial2026-08-25Status changed to Active, not recruiting · ClinicalTrials.gov

An Open Label Long-Term Study to Evaluate the Safety and Tolerability of Fenofibrate in Subjects With Primary Biliary Cholangitis (PBC)

Clinical trial2026-07-29Status changed to Active, not recruiting · ClinicalTrials.gov

Conditional marketing authorisation: Iqirvo (EMA)

Regulatory2024-09-19EMA

Market withdrawal: Ocaliva (EMA)

Regulatory2024-08-30EMA

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

6 clinical trials expected to report results, the earliest in Q4 2026.
1 industry development reported.

Upcoming Milestones

Major developments

Upcoming trial readoutHigh impact

AFFIRM: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Effect of Seladelpar on Clinical Outcomes in Patients With Primary Biliary Cholangitis (PBC) and Compensated Cirrhosis

Results expected Q3 20302026-10-06

Upcoming trial readoutHigh impact

A Phase III, Open-label, Single Arm Study to Investigate the Efficacy and Safety of Elafibranor 80 mg in Adult Japanese Participants With Primary Biliary Cholangitis (PBC)

Results expected Q4 20262026-10-01

Clinical Milestones8View

Results expected 5

2026-10-06AFFIRM: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Effect of Seladelpar on Clinical Outcomes in Patients With Primary Biliary Cholangitis (PBC) and Compensated CirrhosisResults expected Q3 2030
2026-10-01A Phase III, Open-label, Single Arm Study to Investigate the Efficacy and Safety of Elafibranor 80 mg in Adult Japanese Participants With Primary Biliary Cholangitis (PBC)Results expected Q4 2026
2026-08-25A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Volixibat in the Treatment of Cholestatic Pruritus in Patients With Primary Biliary CholangitisResults expected Q1 2027
2026-08-03A Phase III Randomised, Parallel-Group, Double-Blind, Placebo-Controlled, Two-Arm Study to Evaluate the Efficacy and Safety of Elafibranor 80 mg on Long-Term Clinical Outcomes in Adult Participants With Primary Biliary Cholangitis (PBC)Results expected Q2 2029
2026-01-08An Open Label Long-Term Study to Evaluate the Safety and Tolerability of Fenofibrate in Combination With Ursodeoxycholic Acid in Subjects With Primary Biliary Cholangitis (PBC)Results expected Q4 2035

Recently completed 2

Industry & Market1View

2026-10-22'Constipation Cookies'; Antibiotics and IBS; Primary Biliary Cholangitis GuidelinesIndustry · MedPage Today — Gastroenterology

Activity timeline9

2026-10-22Industry'Constipation Cookies'; Antibiotics and IBS; Primary Biliary Cholangitis GuidelinesIndustry · MedPage Today — Gastroenterology
2026-10-06ClinicalAFFIRM: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Effect of Seladelpar on Clinical Outcomes in Patients With Primary Biliary Cholangitis (PBC) and Compensated CirrhosisResults expected Q3 2030
2026-10-01ClinicalA Phase III, Open-label, Single Arm Study to Investigate the Efficacy and Safety of Elafibranor 80 mg in Adult Japanese Participants With Primary Biliary Cholangitis (PBC)Results expected Q4 2026
2026-08-25ClinicalA Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Volixibat in the Treatment of Cholestatic Pruritus in Patients With Primary Biliary CholangitisResults expected Q1 2027
2026-08-03ClinicalA Phase III Randomised, Parallel-Group, Double-Blind, Placebo-Controlled, Two-Arm Study to Evaluate the Efficacy and Safety of Elafibranor 80 mg on Long-Term Clinical Outcomes in Adult Participants With Primary Biliary Cholangitis (PBC)Results expected Q2 2029
2026-06-16ClinicalA Phase IIIb Randomised, Parallel-Group, Double-Blind, Placebo-Controlled, Two-Arm Study to Evaluate the Effect of Elafibranor 80 mg on Normalisation of Alkaline Phosphatase in Adult Participants With Primary Biliary Cholangitis (PBC) and Inadequate Response or Intolerance to Ursodeoxycholic Acid.Completed
2026-05-06ClinicalIDEAL: A 52-week, Double-blind, Placebo-controlled, Randomized, Phase 3 Study Intended to Determine the Effects of Seladelpar on Normalization of Alkaline Phosphatase Levels in Subjects With Primary Biliary Cholangitis (PBC) and an Incomplete Response or Intolerance to Ursodeoxycholic Acid (UDCA)Completed
2026-01-08ClinicalAn Open Label Long-Term Study to Evaluate the Safety and Tolerability of Fenofibrate in Combination With Ursodeoxycholic Acid in Subjects With Primary Biliary Cholangitis (PBC)Results expected Q4 2035
2026-01-08ClinicalAn Open Label Long-Term Study to Evaluate the Safety and Tolerability of Fenofibrate in Combination With Ursodeoxycholic Acid in Subjects With Primary Biliary Cholangitis (PBC)Results expected Q4 2035

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

Elafibranorapproved

Conditional marketing authorisation — Iqirvo is indicated for the treatment of primary biliary cholangitis (PBC) in combination… (2024)

Obeticholic acidapproved

Product authorisation ended — Ocaliva is indicated for the treatment of primary biliary cholangitis (also known as prim… (2024)

Clinical trials

10 sponsors · 0 new · 2 completed in the last 12 months (net +0)

The current development programme across all trial phases.

Clinical programme

All trials

Active

Late-stage

Completed

Late-stage studies

An Open Label Long-Term Study to Evaluate the Safety and Tolerability of Fenofibrate in Combination With Ursodeoxycholic Acid in Subjects With Primary Biliary Cholangitis (PBC)

Phase 3 · Recruiting · Xijing Hospital of Digestive Diseases

An Open Label Long-Term Study to Evaluate the Safety and Tolerability of Fenofibrate in Combination With Ursodeoxycholic Acid in Subjects With Primary Biliary Cholangitis (PBC)

Phase 3 · Recruiting · Xijing Hospital of Digestive Diseases

A Phase III, Open-label, Single Arm Study to Investigate the Efficacy and Safety of Elafibranor 80 mg in Adult Japanese Participants With Primary Biliary Cholangitis (PBC)

Phase 3 · Active, not recruiting · Ipsen

AFFIRM: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Effect of Seladelpar on Clinical Outcomes in Patients With Primary Biliary Cholangitis (PBC) and Compensated Cirrhosis

Phase 3 · Recruiting · Gilead Sciences

A Phase III Randomised, Parallel-Group, Double-Blind, Placebo-Controlled, Two-Arm Study to Evaluate the Efficacy and Safety of Elafibranor 80 mg on Long-Term Clinical Outcomes in Adult Participants With Primary Biliary Cholangitis (PBC)

Phase 3 · Recruiting · Ipsen

A Phase IIIb Randomised, Parallel-Group, Double-Blind, Placebo-Controlled, Two-Arm Study to Evaluate the Effect of Elafibranor 80 mg on Normalisation of Alkaline Phosphatase in Adult Participants With Primary Biliary Cholangitis (PBC) and Inadequate Response or Intolerance to Ursodeoxycholic Acid.

Phase 3 · Completed · Ipsen

Recruiting

An Open Label Long-Term Study to Evaluate the Safety and Tolerability of Fenofibrate in Combination With Ursodeoxycholic Acid in Subjects With Primary Biliary Cholangitis (PBC)

Phase 3 · Recruiting · Xijing Hospital of Digestive Diseases

An Open Label Long-Term Study to Evaluate the Safety and Tolerability of Fenofibrate in Combination With Ursodeoxycholic Acid in Subjects With Primary Biliary Cholangitis (PBC)

Phase 3 · Recruiting · Xijing Hospital of Digestive Diseases

AFFIRM: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Effect of Seladelpar on Clinical Outcomes in Patients With Primary Biliary Cholangitis (PBC) and Compensated Cirrhosis

Phase 3 · Recruiting · Gilead Sciences

Phase 3 · Recruiting · Ipsen

Recently completed

Phase 3 · Completed · Ipsen

IDEAL: A 52-week, Double-blind, Placebo-controlled, Randomized, Phase 3 Study Intended to Determine the Effects of Seladelpar on Normalization of Alkaline Phosphatase Levels in Subjects With Primary Biliary Cholangitis (PBC) and an Incomplete Response or Intolerance to Ursodeoxycholic Acid (UDCA)

Phase 3 · Completed · Gilead Sciences

A Phase 2a, Double-Blind, Randomized, Active Controlled, Parallel Group Study Evaluating the Efficacy, Safety, and Tolerability of Bezafibrate Administered in Combination With Obeticholic Acid in Subjects With Primary Biliary Cholangitis

Phase 2 · Completed · Intercept Pharmaceuticals

A Multicenter, Randomized, Double-blind, Placebo Controlled, Phase 2b/3 Study to Evaluate the Efficacy and Safety of Saroglitazar Magnesium in Subjects With Primary Biliary Cholangitis

Phase 2/3 · Completed · Zydus Therapeutics Inc.

A Randomized, Double-blind, Multicenter, Placebo-controlled Phase III Clinical Trial to Evaluate the Efficacy and Safety of Obeticholic Acid in Patients With Primary Biliary Cholangitis

Phase 3 · Completed · Nanjing Chia-tai Tianqing Pharmaceutical

A 52-week, Placebo-controlled, Randomized, Phase 3 Study to Evaluate the Safety and Efficacy of Seladelpar in Subjects With Primary Biliary Cholangitis (PBC) and an Inadequate Response to or an Intolerance to Ursodeoxycholic Acid (UDCA)

Phase 3 · Completed · Gilead Sciences

Regulatory timeline

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

First approvals

2024emaConditional marketing authorisation — Elafibranor· Iqirvo is indicated for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA. source ↗

Other regulatory activity

2024emaProduct authorisation ended — Obeticholic acid· Ocaliva is indicated for the treatment of primary biliary cholangitis (also known as primary biliary cirrhosis) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA. 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.

NR1H40.61 IL7R0.55 NFKB10.53 TNFRSF1A0.52 MANBA0.51 IL12A0.50 IL12RB20.46 CD2260.45 CD580.44 IL21R0.44 PPARA0.44 TNFSF150.44

Disease biology

4 matches

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

Bile acid receptorvia NR1H40.61 Peroxisome proliferator-activated receptor alphavia PPARA0.44 Peroxisome proliferator-activated receptor deltavia PPARD0.42 Pro-interleukin-16via IL160.32

Reference

Authoritative identity, definition & identifiers.

Synonyms

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.