常染色體顯性多囊性腎病變（ADPKD）：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 ADPKD 市場預計將從2025年的約 1.3億美元成長到2035年的約 2.25億美元，年複合成長率約為 5-6%。
• 目前的治療選擇有限，托伐普坦是唯一廣泛使用的疾病修正治療。

常染色體顯性多囊性腎病變（ADPKD）概述

常染色體顯性多囊性腎病變（ADPKD）是最常見的遺傳性腎病，主要由PKD1基因（約佔78-85%）或PKD2基因（約佔15%）的突變引起，這兩個基因分別編碼機械感應蛋白多囊蛋白-1和多囊蛋白-2。雖然ADPKD通常為遺傳性，但約10%的病例是由新發突變引起的。此病的特徵是腎實質內散在分佈多個充滿液體的囊腫，這些囊腫不斷形成並增大，導致顯著的腎肥大、早發性高血壓和進行性性結構損傷。即使在同一家族內，臨床嚴重程度也存在顯著差異，但PKD1突變通常會導致病情快速進展，患者往往在50歲末期發展至末期腎功能衰竭（ESRD），而末期腎功能衰竭突變患者則大約在20年後發展至ESRD。現代預後管理強調透過磁振造影（MRI）測量身高校正後的總腎體積（htTKV），以識別疾病快速進展的患者。針對這些高風險患者的標準疾病修正治療是tolvaptan，一種血管加壓素V2受體拮抗劑。其作用機轉是透過抑制細胞內cAMP，減緩囊腫生長，進而保護腎功能並延緩腎臟替代治療的需求。

主要亮點

• ADPKD 是一種罕見的慢性進行性疾病，會對腎臟和心血管系統造成嚴重的長期負擔。
• 在英國，預計新增病例數將從2025年的約 4,400 例增加到2035年的約 4,900 例，反映出較低的成長率（年複合成長率約為 1%）。

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MicrosoftExcel）
• 流行病學資料（MS Excel，互動式工具）
• 高層洞察（PPT簡報）
• 其他功能：定期更新、自訂和顧問支援。
• 根據 Thelansis 的政策，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

• 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）

主要企業

• Regulus Therapeutics Inc.
• AbbVie
• AstraZeneca
• Jemincare
• Rege Nephro Co., Ltd.
• PYC Therapeutics
• Vertex Pharmaceuticals
• Otsuka Pharmaceutical
• XORTX Therapeutics
• Poxel SA
• Calico Life Sciences
• AceLink Therapeutics
• PYC Therapeutics

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
• 依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
• 值得關注的早期研發管線

第6章 未滿足的需求與目標產品分析

• 主要未滿足需求及新興療法的未來成就
• TPP分析與KOL展望

第7章 監理與償付環境

第8章 附錄

Global Autosomal Dominant Polycystic Kidney Disease (ADPKD) Market Outlook

Thelansis's "Global Autosomal Dominant Polycystic Kidney Disease (ADPKD) 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 ADPKD treatment modalities options for the 32 markets (North America, Europe, Middle East, Asia Pacific, Africa, South / Latin America).

Autosomal Dominant Polycystic Kidney Disease (ADPKD) Overview

Autosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary kidney disorder, driven primarily by mutations in the PKD1 (~78-85%) or PKD2 (~15%) genes, which encode the mechanosensory proteins polycystin-1 and polycystin-2. While typically inherited, approximately 10% of cases arise from de novo mutations. The disease is characterized by the inexorable development and expansion of multiple fluid-filled cysts scattered throughout the renal parenchyma, leading to massive nephromegaly, early-onset hypertension, and progressive structural damage. Clinical severity is highly variable even within families, though PKD1 mutations uniformly dictate a more aggressive course, often driving patients to end-stage renal disease (ESRD) in their late 50s, whereas PKD2 patients typically reach ESRD about two decades later. Modern prognostic management heavily relies on measuring height-adjusted total kidney volume (htTKV) via MRI to identify rapid progressors. For these high-risk patients, the standard disease-modifying therapy is the vasopressin V2 receptor antagonist tolvaptan, which suppresses intracellular cAMP to slow cyst proliferation, thereby preserving renal function and delaying the need for renal replacement therapy.

Key Highlights

• ADPKD is a chronic, progressive rare disease with significant long-term renal and cardiovascular burden.
• In the UK, incident cases are expected to increase from ~4.4K in 2025 to ~4.9K by 2035, reflecting a low growth rate (~1% CAGR).

Market Overview

• The ADPKD market in Germany is projected to grow from ~$130M in 2025 to ~$225M by 2035, reflecting a CAGR of ~5-6%.
• Current treatment landscape is limited, with Tolvaptan as the only widely adopted disease-modifying therapy

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

• Regulus Therapeutics Inc.
• AbbVie
• AstraZeneca
• Jemincare
• Rege Nephro Co., Ltd.
• PYC Therapeutics
• Vertex Pharmaceuticals
• Otsuka Pharmaceutical
• XORTX Therapeutics
• Poxel SA
• Calico Life Sciences
• AceLink Therapeutics
• PYC Therapeutics

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)