원발성 담즙성 담관염(PBC) : 신규 요법, 미충족 수요 및 TPP 인사이트 리포트(2026년)

Primary Biliary Cholangitis (PBC) Emerging Therapy and TPP Insights

Thelansis's "Primary Biliary Cholangitis (PBC) Emerging Therapy, with Unmet Needs and TPP Insights Report - 2026" provides a comprehensive analysis of the emerging competitive landscape, unmet needs, target product profiles (TPPs), trial designs, and KOL insights on key emerging therapies and key drug development opportunities in the indication.

Primary Biliary Cholangitis (PBC) Overview

Primary biliary cholangitis (PBC) is a chronic, progressive autoimmune cholestatic liver disease characterised by immune-mediated destruction of small intrahepatic bile ducts, driven by autoreactive T-cell targeting of mitochondrial antigens - predominantly the pyruvate dehydrogenase complex E2 subunit - in genetically susceptible individuals, with antimitochondrial antibodies (AMA) serving as the hallmark diagnostic biomarker present in over 90% of patients. Predominantly affecting middle-aged women, patients may present insidiously with fatigue and pruritus - often preceding overt cholestasis - or be identified incidentally through elevated alkaline phosphatase and GGT on routine biochemistry. Diagnosis integrates AMA positivity, cholestatic liver biochemistry, and - in AMA-negative cases - liver biopsy demonstrating florid bile duct lesions. Risk stratification employs validated scores including GLOBE and UK-PBC to identify inadequate biochemical responders at greatest cirrhosis progression risk. Ursodeoxycholic acid (UDCA) remains first-line therapy, improving bile flow and transplant-free survival; obeticholic acid, an FXR agonist, and bezafibrate provide second-line options for inadequate UDCA responders. Elafibranor and seladelpar, novel PPAR agonists, represent recently approved therapeutic advances expanding the management landscape. Prognosis is favourable with early treatment and biochemical response; liver transplantation remains definitive therapy for end-stage disease, with patient-centred symptom management - particularly pruritus and fatigue - integral to quality-of-life optimisation.

Key Highlights

• In France, diagnosed PBC cases are projected to increase from approximately 18,716 in 2025 to nearly 20,069 by 2035, reflecting a gradual rise in diagnosed prevalence.
• PBC predominantly affects women and remains associated with substantial long-term morbidity due to progressive cholestatic liver damage.
• A considerable proportion of patients exhibit inadequate biochemical response to UDCA, driving demand for second-line and combination therapies.
• Recently approved PPAR agonists are significantly expanding treatment options for patients with persistent disease activity.
• Persistent fatigue and pruritus continue to represent major unmet clinical needs impacting patient quality of life.

Market Overview

• The Germany PBC market is projected to grow from approximately $54 MN to $145 MN, reflecting significant market expansion.
• Market growth is supported by:
• Increasing adoption of second-line and novel targeted therapies
• Expanding use of PPAR agonists and combination treatment strategies
• Improved diagnosis, earlier intervention, and enhanced disease awareness are contributing to increased treatment uptake.
• Future market growth will depend on next-generation targeted therapies, improved long-term disease control, and expanded personalized treatment approaches.

Insights driven by surveys with physician / key opinion leaders:

• Survey findings are corroborated and enriched by insights from interviews with leading KOLs
• Survey is customized based on client requirements

Deliverables format:

• PowerPoint presentation
• MS Excel

Key business questions answered:

• Detailed emerging competitive landscape
• Pipeline analysis
• Target patients for emerging therapies
• Key companies
• Key mechanism of actions
• Launch date estimates, etc.
• Clinical trial landscape analysis
• Target patient segments
• Trial endpoints
• Trial design
• Recruitment criteria, etc.
• Unmet Needs and Opportunities
• Performance of key current therapies
• Top areas of unmet needs
• Opportunity sizing for key unmet needs
• Target Product Profiles
• Attributes and levels
• Physician likelihood of prescribing
• Expected patient shares
• KOL insights on key emerging therapies
• Level of awareness
• Expected use / line of therapy
• Extent to fulfil key unmet needs
• KOL quotes

Countries Covered

• G8
  • United States
  • EU5
    • France
    • Germany
    • Italy
    • Spain
    • U.K.
  • Japan
  • China

Apart from the G8 Market, adding any additional country data to the dashboard will cost USD 1,750 per country

Companies Mentioned

• Zydus Therapeutics Inc.
• Gilead Sciences
• Ipsen
• Intercept Pharmaceuticals
• COUR Pharmaceutical Development Company, Inc.
• Tharimmune Inc.
• Mirum Pharmaceuticals, Inc.
• Umecrine Cognition AB
• Parvus Therapeutics, Inc.
• Calliditas Therapeutics Suisse SA

Table of Contents

1. Key Findings and Analyst Commentary

• Key trends: market snapshots, SWOT analysis, commercial benefits and risk, etc.

2. Competitive Landscape

• Current therapies
• Key takeaways
• Dx and Tx journey/algorithm
• Key current therapies - profiles and KOL insights
• Emerging therapies
• Key takeaways
• Dx and Tx journey/algorithm
• Key emerging therapies - profiles and KOL insights

3. Product Attribute Analysis

• Key takeaways
• Scientific attributes
• Commercial attributes
• Product positioning

4. Primary Market Research

• Current treatment landscape
• Key therapies vs. focused patient segment
• Key attributes and benefits
• Futures treatment landscape
• Current challenges
• Unmet needs
• Emerging therapies
• Key therapies vs. focused patient segment
• Key attributes and benefits
• Futures treatment landscape
• Unmet needs and KOL expectations

5. Unmet Need and TPP Analysis

• Top unmet needs and future attainment by emerging therapies
• TPP analysis and KOL expectations

6. Regulatory and Reimbursement Environments (by country and payer insights)

7. Appendix (e.g., bibliography, methodology)