原發性膽汁性膽管炎（PBC）：全球市場展望、流行病學、競爭格局和市場預測報告（32個主要市場），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疾病認知的提高，都促使更多人接受這種治療方法。
• 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，由自身反應性 T 細胞攻擊粒線體抗原（主要是丙酮酸去氫酶複合物 E2次單元）觸發，主要發生於具有遺傳易感性的個體。抗線粒體抗體 (AMA) 是此疾病的特徵性診斷生物標記物，在超過 90% 的患者中均可檢測到。 PBC 主要影響中年女性，患者可能在出現明顯的膽汁淤積之前逐漸出現疲乏和瘙癢症狀，也可能在常規生物化學檢驗中偶然發現鹼性磷酸酶和 γ-谷氨醯轉移酶 (GGT) 水平升高。診斷是根據 AMA 陽性、與膽汁淤積相關的肝功能檢查結果以及（在 AMA 陰性病例中）肝臟切片檢查觀察（提示存在顯著膽管病變）的綜合評估。檢驗的評分系統，例如 GLOBE 和 UK-PBC 評分，用於風險分層，以識別生化反應不足且肝硬化進展風險最高的患者。熊去氧膽酸 (UDCA) 仍然是一線治療藥物，可改善膽汁流量並延長存活期，無需進行肝臟移植。對於 UDCA 療效不佳的患者，FXR促效劑，例如奧貝膽酸和苯扎貝特，可作為二線治療藥物。新型 PPAR促效劑，Elafibranor和塞拉德帕，是近期獲準的治療進展，拓展了治療選擇。早期治療和良好的生化反應預後良好。肝臟移植仍然是末期疾病的根治性治療方法，但以患者為中心的症狀管理——特別是緩解搔癢和疲勞——對於最佳化生活品質 (QOL) 至關重要。

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，反映出該疾病的盛行率逐漸增加。
• PBC 主要影響女性，由於進行性膽汁淤積性肝損傷，仍與嚴重的長期發病率有關。
• 相當一部分患者對 UDCA 的生化反應不足，這增加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。

市場定義：

• 北美洲（美國、加拿大）
• 歐洲（奧地利、比利時、捷克、丹麥、芬蘭、法國、德國、希臘、義大利、荷蘭、挪威、波蘭、葡萄牙、俄羅斯、西班牙、瑞典、瑞士、英國）
• 中東（沙烏地阿拉伯、阿拉伯聯合大公國、科威特、土耳其）
• 亞太地區（澳洲、中國、香港、印度、印尼、日本、馬來西亞、紐西蘭、菲律賓、新加坡、韓國、台灣、泰國、越南）
• 非洲（埃及、奈及利亞、南非、摩洛哥）
• 南美洲/拉丁美洲（阿根廷、巴西、智利、哥倫比亞、墨西哥、秘魯）

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

• 我們如何最佳化在 32 個市場（北美、歐洲、中東、亞太、非洲和南美/拉丁美洲）的藥物開發和生命週期管理策略？
• 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？
• 為確保藥物獲得批准並順利進入市場，必須滿足哪些關鍵的監管和支付方要求？

目標國家

• 北美洲（美國、加拿大）
• 歐洲（奧地利、比利時、捷克、丹麥、芬蘭、法國、德國、希臘、義大利、荷蘭、挪威、波蘭、葡萄牙、俄羅斯、西班牙、瑞典、瑞士、英國）
• 中東（沙烏地阿拉伯、阿拉伯聯合大公國、科威特、土耳其）
• 亞太地區（澳洲、中國、香港、印度、印尼、日本、馬來西亞、紐西蘭、菲律賓、新加坡、韓國、台灣、泰國、越南）
• 非洲（埃及、奈及利亞、南非、摩洛哥）
• 南美洲/拉丁美洲（阿根廷、巴西、智利、哥倫比亞、墨西哥、秘魯）

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
• 已確診並正在接受藥物治療的患者族群。
• 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
• 目前主要治療方法－概述及關鍵意見領袖見解
• 新興治療方法
• 重點
• 值得關注的後期新興治療方法—概述、市場上市預期和關鍵意見領袖見解
• 值得關注的早期管道

第6章：未滿足的需求與目標產品分析

• 尚未滿足的主要需求及其透過新興治療方法。
• TPP分析與KOL展望

第7章 監理與報銷環境

第8章附錄

Global Primary Biliary Cholangitis (PBC) Market Outlook

Thelansis's "Global Primary Biliary Cholangitis (PBC) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report (Top 32 Markets) - 2025 To 2035" covers disease overview, epidemiology, kidney biopsy cases, 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 the 32 markets (North America, Europe, Middle East, Asia Pacific, Africa, South / Latin America).

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.

Market Definition:

• North America (United States, Canada)
• Europe (Austria, Belgium, Czech Republic, Denmark, Finland, France, Germany, Greece, Italy, Netherlands, Norway, Poland, Portugal, Russia, Spain, Sweden, Switzerland, United Kingdom)
• Middle East (Saudi Arabia, UAE, Kuwait, Turkey)
• Asia Pacific (Australia, China, Hong Kong, India, Indonesia, Japan, Malaysia, New Zealand, Philippines, Singapore, South Korea, Taiwan, Thailand, Vietnam)
• Africa (Egypt, Nigeria, South Africa, Morocco)
• South / Latin America (Argentina, Brazil, Chile, Colombia, Mexico, Peru)

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 the 32 markets (North America, Europe, Middle East, Asia Pacific, Africa, South / Latin America)?
• 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

• North America (United States, Canada)
• Europe (Austria, Belgium, Czech Republic, Denmark, Finland, France, Germany, Greece, Italy, Netherlands, Norway, Poland, Portugal, Russia, Spain, Sweden, Switzerland, United Kingdom)
• Middle East (Saudi Arabia, UAE, Kuwait, Turkey)
• Asia Pacific (Australia, China, Hong Kong, India, Indonesia, Japan, Malaysia, New Zealand, Philippines, Singapore, South Korea, Taiwan, Thailand, Vietnam)
• Africa (Egypt, Nigeria, South Africa, Morocco)
• South / Latin America (Argentina, Brazil, Chile, Colombia, Mexico, Peru)

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)