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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.

20
Clinical trials
12
Associated genes
4
Related proteins
Current focus
Bile acid biologyTherapeutic developmentInflammation & immunity
Latest activity
beta

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

Approval: 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.
Upcoming Milestones
Clinical Milestones11View all 11
Results expected 5
Recently completed 3
Other updates 2
+3 more in the activity timeline below
Activity timeline11

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

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

Clinical trials

9 sponsors · 1 new · 3 completed in the last 12 months (net -1)

The current development programme across all trial phases.

Clinical programme
20
All trials
6
Active
12
Late-stage
6
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 Multicenter, Randomized, Double-Blind, Double-Dummy, Active-Controlled Clinical Trial of Fenofibrate in Treatment-Naïve Patients With Primary Biliary Cholangitis

Phase 3 · Recruiting · Xijing Hospital of Digestive Diseases
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

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 Multicenter, Randomized, Double-Blind, Double-Dummy, Active-Controlled Clinical Trial of Fenofibrate in Treatment-Naïve Patients With Primary Biliary Cholangitis

Phase 3 · Recruiting · Xijing Hospital of Digestive Diseases
Recently completed

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

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
2024emaApproval — 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 ↗
Safety updates
2024emaMarket withdrawal — 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.

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

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

Reference

Authoritative identity, definition & identifiers.

Synonyms

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

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.