原發性膽汁性膽管炎：市場展望、流行病學、競爭格局、市場預測報告（2025-2035年）

市場概覽

• 德國 PBC 市場預計將從約 5,400 萬美元成長到 1.45 億美元，顯示市場將大幅擴張。
• 市場成長受以下因素支撐：
• 擴大二線治療和新型標靶治療的應用
• 擴大PPAR促效劑和聯合療法的應用。
• 診斷技術的進步、早期療育以及人們對該疾病認知的提高，都促進了治療的普及。
• 未來市場成長將取決於下一代標靶治療、改善長期疾病管理以及個人化治療方法的擴展。

原發性膽汁性膽管炎（PBC）概述

原發性膽汁性膽管炎（PBC）是一種慢性進行性自體免疫膽汁淤積性肝病，其特徵是免疫介導的肝內膽管破壞，導致膽汁流動受阻、慢性膽汁淤積、纖維化，最終在晚期為肝硬化。此病主要影響中年女性，且與抗粒線體抗體（AMA）密切相關，AMA是大多數患者的特徵性診斷生物標記。常見的臨床症狀包括疲倦、搔癢和膽汁淤積性肝酵素升高，但此疾病可進展為門靜脈高壓、肝功能障礙和肝功能衰竭。

診斷主要依據肝功能檢查結果（包括膽汁淤積）、抗粒線體抗體（AMA）陽性，以及在某些情況下進行的組織學觀察。熊去氧膽酸（UDCA）仍是標準的第一線治療方案，可顯著提高無需肝臟移植的生存率，但相當一部分患者會出現生化反應不足或疾病持續進展的情況。二線治療方案包括奧貝膽酸和貝特類藥物，但近期核准的過氧化物酶體增殖物激活受體（PPAR）促效劑，如依Elafibranor和塞拉德帕，正在拓展治療選擇。

儘管治療方法不斷進步，但對於治療反應不完全、出現進行性纖維化以及持續存在症狀負擔（尤其是嚴重影響生活品質的疲勞和瘙癢）的患者而言，仍然存在未被滿足的需求。不斷發展的治療格局日益側重於新型標靶治療、更完善的風險分層以及旨在延緩疾病進展和減少肝臟移植需求的個人化疾病管理方案。

主要亮點

• 在法國，PBC 的診斷數量預計將從 2025 年的約 18,716 例增加到 2035 年的近 20,069 例，反映出診斷盛行率的逐漸增加。
• PBC 主要影響女性，由於其進行性膽汁淤積性肝損傷，從長遠來看仍然是一種高風險疾病。
• 相當一部分患者對 UDCA 的生化反應不足，這推動了對二線療法和聯合治療的需求。
• 最近核准的PPAR促效劑顯著擴大了持續性疾病活動患者的治療選擇。
• 持續的疲勞和搔癢仍然是影響患者生活品質的主要未滿足需求。

格式化和更新訊息

• 詳細報告（PDF）
• 市場預測模型（基於微軟Excel）
• 流行病學數據（MS Excel，互動式工具）
• 高階主管洞察（PPT簡報）
• 其他功能：定期更新、自訂和顧問支援。
• 根據 Thelansis 的政策，我們確保所有最新更新在發布前都反映在報告內容和市場模型中。

主要問題

• 我們如何最佳化 G8 市場（美國、歐盟 5 國、日本、中國）的藥物開發與生命週期管理策略？
• 從發病率、盛行率、人群組成以及接受藥物治療的患者人數來看，患者數量分別是多少？
• 未來十年市場收入和病患佔有率的預測是多少？
• 哪些因素對市場趨勢影響最大？
• 受訪專家對目前和新興的治療方法有何看法？
• 哪款在研發線產品最有前景？其上市潛力及未來市場定位如何？
• 主要未被滿足的需求是什麼？ KOL 對目標受眾有何期望？
• 為確保藥物核准並順利進入市場，必須滿足哪些關鍵的監管和支付方要求？

目標國家

• G8
  • 美國
  • EU5
    • 法國
    • 德國
    • 義大利
    • 西班牙
    • 英國
  • 日本
  • 中國

大公司

• 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

目錄

第1章：主要調查結果及分析師說明

• 主要趨勢：市場概況、SWOT分析、商業性利益與風險等。

第2章：疾病背景

• 疾病定義、分類、病因和病理生理學、藥物標靶等。

第3章：流行病學

• 重點
• 發病率/盛行率
• 已確診並接受藥物治療的患者人數
• 合併症
• 其他相關患者群

第4章 市場規模及預測

• 重點
• 市場促進因素與限制因素
• 按藥物類別分類的趨勢
• 各國具體趨勢

第5章 競爭情勢

• 目前的治療方法
• 重點
• 診斷和治療過程/演算法
• 主要治療方法概述及KOL洞察
• 新興治療方法
• 重點
• 值得關注的後期新治療方法－概述、市場上市預期及KOL洞察
• 值得關注的早期研發管線

第6章：未滿足的需求與TPP分析

• 主要未滿足的需求及其未來透過新興治療方法實現的可能性
• TPP分析與KOL展望

第7章 監理與報銷環境

第8章附錄

Primary Biliary Cholangitis (PBC) Market Outlook

Thelansis's "Primary Biliary Cholangitis (PBC) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report - 2025 To 2035" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Primary Biliary Cholangitis treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Primary Biliary Cholangitis (PBC) Overview

Primary biliary cholangitis (PBC) is a chronic, progressive autoimmune cholestatic liver disease characterized by immune-mediated destruction of small intrahepatic bile ducts, leading to impaired bile flow, chronic cholestasis, fibrosis, and eventual cirrhosis in advanced stages. The disease predominantly affects middle-aged women and is strongly associated with antimitochondrial antibodies (AMA), which serve as the hallmark diagnostic biomarker in the majority of patients. Common clinical manifestations include fatigue, pruritus, and elevated cholestatic liver enzymes, while advanced disease may lead to portal hypertension, hepatic insufficiency, and liver failure.

Diagnosis is based on cholestatic liver biochemistry, AMA positivity, and histological findings in select cases. Ursodeoxycholic acid (UDCA) remains the standard first-line therapy and significantly improves transplant-free survival; however, a substantial proportion of patients demonstrate inadequate biochemical response or persistent disease progression. Second-line treatment options include obeticholic acid and fibrates, while recently approved PPAR agonists such as elafibranor and seladelpar are expanding the therapeutic landscape.

Despite therapeutic advances, unmet needs persist in patients with incomplete treatment response, progressive fibrosis, and persistent symptom burden, particularly fatigue and pruritus, which significantly impact quality of life. The evolving treatment landscape is increasingly focused on novel targeted therapies, improved risk stratification, and personalized disease management approaches aimed at delaying progression and reducing the need for liver transplantation.

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 robust research, including:

• In-depth interviews with leading KOLs and payers
• Physician surveys
• RWE analysis for claims and EHR datasets
• Secondary research (e.g., peer-reviewed journal articles, third-party research databases)

Deliverables format and updates*:

• Detailed Report (PDF)
• Market Forecast Model (MS Excel-based automated dashboard)
• Epidemiology (MS Excel; interactive tool)
• Executive Insights (PowerPoint presentation)
• Others: regular updates, customizations, consultant support
• As per Thelansis's policy, we ensure that we include all the recent updates before releasing the report content and market model.

Salient features of Market Forecast model:

• 10-year market forecast (2025-2035)
• Bottom-up patient-based market forecasts validated through the top-down sales methodology
• Covers clinically and commercially-relevant patient populations/ line of therapies
• Annualized drug-level sales and patient share projections
• Utilizes our proprietary Epilansis and Analog tool (e.g., drug uptake and erosion) datasets and conjoint analysis approach
• Detailed methodology/sources & assumptions
• Graphical and tabular outputs
• Users can customize the model based on requirements

Key business questions answered:

• How can drug development and lifecycle management strategies be optimized across G8 markets (US, EU5, Japan, and China)?
• How large is the patient population in terms of incidence, prevalence, segments, and those receiving drug treatments?
• What is the 10-year market outlook for sales and patient share?
• Which events will have the greatest impact on the market's trajectory?
• What insights do interviewed experts provide on current and emerging treatments?
• Which pipeline products show the most promise, and what is their potential for launch and future positioning?
• What are the key unmet needs and KOL expectations for target profiles?
• What key regulatory and payer requirements must be met to secure drug approval and favorable market access?

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 risks, etc.

2. Disease Context

• Disease definition, classification, etiology and pathophysiology, drug targets, etc.

3. Epidemiology

• Key takeaways
• Incidence / Prevalence
• Diagnosed and Drug-Treated populations
• Comorbidities
• Other relevant patient segments

4. Market Size and Forecast

• Key takeaways
• Market drivers and constraints
• Drug-class specific trends
• Country-specific trends

5. Competitive Landscape

• Current therapies
• Key takeaways
• Dx and Tx journey/algorithm
• Key current therapies - profiles and KOL insights
• Emerging therapies
• Key takeaways
• Notable late-phase emerging therapies - profiles, launch expectations, KOL insights
• Notable early-phase pipeline

6. Unmet Need and TPP Analysis

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

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

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