常染色體顯性多囊性腎病變（ADPKD）：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 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，複合年成長率約為 5-6%。
• 目前的治療選擇有限，托沃帕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

體染色體顯性多囊性腎病變（ADPKD）是最常見的遺傳性腎病變，主要由PKD1基因（約佔78-85%）或PKD2基因（約佔15%）的突變引起，這兩個基因編碼多囊蛋白，參與腎小管的結構和訊號傳導。雖然大多數病例為遺傳性，但約10%的病例是由新突變引起的。

這種疾病的特徵是腎臟內進行性出現大量充滿液體的囊腫，導致腎臟肥大、高血壓和腎功能逐漸下降。臨床嚴重程度差異很大，PKD1 基因突變往往進展更快，並導致更早發展為末期腎功能衰竭(ESRD)。

現代疾病管理著重於利用影像學生物標記（例如身高校正總腎體積 (htTKV)）來識別病情快速進展的患者。目前標準的疾病修正治療是托伐普坦，一種血管加壓素 V2 受體拮抗劑，可抑制囊腫生長並延緩腎功能下降。然而，治療方法仍然有限，許多患者最終仍需接受透析或腎臟移植。

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%）。

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療法有何看法？
• 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

• 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

• 目前療法
  • 重點
  • 診斷和治療過程/演算法
  • 主要療法概述及KOL洞察
• 新興療法
  • 重點
  • 值得關注的後期新療法—概述、市場上市預期和KOL洞察
  • 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

• 主要未滿足的需求以及透過新興療法實現的未來可能性
• TPP分析與KOL展望

第7章 監理與報銷環境

第8章附錄

Autosomal Dominant Polycystic Kidney Disease (ADPKD) Market Outlook

Thelansis's "Autosomal Dominant Polycystic Kidney Disease (ADPKD) 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 Autosomal Dominant Polycystic Kidney Disease treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Autosomal Dominant Polycystic Kidney Disease (ADPKD) Overview

Autosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary kidney disorder, primarily caused by mutations in the PKD1 (~78-85%) or PKD2 (~15%) genes, which encode polycystin proteins involved in renal tubular structure and signaling. While largely inherited, ~10% of cases arise from de novo mutations.

The disease is characterized by the progressive development of multiple fluid-filled renal cysts, leading to kidney enlargement (nephromegaly), hypertension, and gradual loss of renal function. Clinical severity varies significantly, with PKD1 mutations associated with a more aggressive course and earlier progression to end-stage renal disease (ESRD).

Modern disease management focuses on identifying rapid progressors using imaging biomarkers such as height-adjusted total kidney volume (htTKV). The current standard disease-modifying therapy is tolvaptan, a vasopressin V2 receptor antagonist that reduces cyst growth and slows renal decline. However, treatment remains limited, and many patients ultimately require dialysis or transplantation.

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

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/report will cost USD 1,750 per country

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)