常染色体显性多囊性肾病变（ADPKD）：市场展望、流行病学、竞争格局、市场预测报告（2025-2035年）

市场概览

• 德国 ADPKD 市场预计将从 2025 年的约 1.3 亿美元成长到 2035 年的约 2.25 亿美元，复合年增长率约为 5-6%。
• 目前的治疗选择有限，托沃帕坦是唯一广泛使用的疾病修正治疗。

常染色体显性多囊性肾病变（ADPKD）概述

体染色体显性多囊性肾病变（ADPKD）是最常见的遗传性肾病变，主要由PKD1基因（约占78-85%）或PKD2基因（约占15%）的突变引起，这两个基因编码多囊蛋白，参与肾小管的结构和讯号传导。虽然大多数病例为遗传性，但约10%的病例是由新突变引起的。

这种疾病的特征是肾臟内进行性出现大量充满液体的囊肿，导致肾臟肥大、高血压和肾功能逐渐下降。临床严重程度差异很大，PKD1 基因突变往往进展更快，并导致更早发展为末期肾功能衰竭(ESRD)。

现代疾病管理着重于利用影像学生物标记（例如身高校正总肾体积 (htTKV)）来识别病情快速进展的患者。目前标准的疾病修正治疗是托伐普坦，一种血管加压素 V2 受体拮抗剂，可抑制囊肿生长并延缓肾功能下降。然而，治疗方法仍然有限，许多患者最终仍需接受透析或肾臟移植。

主要亮点

• ADPKD 是一种罕见的慢性进行性疾病，会对肾臟和心血管系统造成严重的长期负担。
• 在英国，预计新增病例数将从 2025 年的约 4,400 例增加到 2035 年的约 4,900 例，反映出较低的成长率（年复合成长率约为 1%）。

格式化和更新讯息

• 详细报告（PDF）
• 市场预测模型（基于微软Excel）
• 流行病学数据（MS Excel，互动式工具）
• 高阶主管洞察（PPT简报）
• 其他功能：定期更新、自订和顾问支援。
• 根据 Thelansis 的政策，我们确保所有最新更新在发布前都反映在报告内容和市场模型中。

主要问题

• 我们如何优化 G8 市场（美国、欧盟 5 国、日本、中国）的药物开发与生命週期管理策略？
• 从发病率、盛行率、人群组成以及接受药物治疗的患者人数来看，患者数量分别是多少？
• 未来十年市场收入和病患份额的预测是多少？
• 哪些因素对市场趋势影响最大？
• 受访专家对目前和新兴疗法有何看法？
• 哪款在研发线产品最有前景？其上市潜力及未来市场定位如何？
• 主要未被满足的需求是什么？ KOL 对目标受众有何期望？
• 为确保药物核准并顺利进入市场，必须满足哪些关键的监管和支付方要求？

目标国家

• G8
  • 我们
  • EU5
    • 法国
    • 德国
    • 义大利
    • 西班牙
    • 英国
  • 日本
  • 中国

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

目录

第一章：主要调查结果及分析师说明

• 主要趋势：市场概况、SWOT分析、商业性利益与风险等。

第二章：疾病背景

• 疾病定义、分类、病因和病理生理学、药物标靶等。

第三章：流行病学

• 重点
• 发病率/盛行率
• 已确诊并接受药物治疗的患者人数
• 合併症
• 其他相关患者群

第四章 市场规模及预测

• 重点
• 市场驱动因素与限制因素
• 按药物类别分類的趋势
• 各国具体趋势

第五章 竞争情势

• 目前疗法
  • 重点
  • 诊断和治疗过程/演算法
  • 主要疗法概述及KOL洞察
• 新兴疗法
  • 重点
  • 值得关注的后期新疗法—概述、市场上市预期和KOL洞察
  • 值得关注的早期管道

第六章：未满足的需求与目标产品分析

• 主要未满足的需求以及透过新兴疗法实现的未来可能性
• TPP分析与KOL展望

第七章 监理与报销环境

第八章附录

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)