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常染色体显性多囊性肾病变市场 - 全球及区域 - 分析与预测（2025-2035）
市场调查报告书
商品编码
1789844

常染色体显性多囊性肾病变市场 - 全球及区域 - 分析与预测（2025-2035）

Autosomal Dominant Polycystic Kidney Disease Market - A Global and Regional Analysis: Analysis and Forecast, 2025-2035
出版日期: | 出版商: BIS Research | 英文 100 Pages | 商品交期: 1-5个工作天内

价格

体染色体显性多囊性肾病变是一种遗传性疾病，其特征是肾臟中进行性形成大量充满液体的囊肿。

这些囊肿会随着时间而增长，导致肾臟肿大、肾功能下降，最终导致末期肾功能衰竭(ESRD)。体染色体显性多囊性肾病变 (ADKD) 是最常见的遗传性肾病变之一，全球每 500 到 1,000 人中约有 1 人患有此病。此疾病主要由 PKD1 和 PKD2 基因突变引起，会损害正常的肾细胞生长和功能。症状通常在成年期开始，包括高血压、腰痛、尿道感染和肾结石。常染色体显性多囊性肾病变 (ADKD) 会严重影响患者的生活质量，并给医疗保健带来沉重的负担，因为肾功能障碍进行性，可能需要进行透析或肾臟移植。

常染色体显性多囊性肾病变 (ADKD) 市场正经历强劲增长，这得益于对该疾病病理学理解的不断深入、ADC标靶治疗的出现以及诊断率的不断提高。以往，ADC 的治疗主要着重于支持性治疗，重点在于控制血压和缓解症状。然而，大冢製药核准托伐普坦 (Zinalc) 标誌着一项里程碑，这是首个透过拮抗加压素 V2 受体来减缓囊肿生长和肾衰竭的疾病修正治疗。

常染色体显性多囊性肾病变 (ADKD) 盛行率的不断上升是推动市场的主要因素之一。 ADC 影响全球约 400 万至 600 万人，估计盛行率为 1/500 至 1/1,000。随着人们所认识的提高和诊断工具的进步，例如高解析度成像技术（MRI 和超音波）和基因检测，人们能够更早、更准确地进行诊断。例如，在美国，美国国家糖尿病、消化和肾臟疾病研究所 (NIDDK) 的一份报告指出，早期发现正变得越来越普遍，这使得患者能够及时接受干预，从而减缓病情的进展。全球 ADC 诊断数量的增加正在扩大需要药物治疗的患者群体，从而推动医药市场的成长。

此外，2018年美国食品药物管理局（FDA）核准托伐普坦（由大冢製药以Jynarque为商品名上市）作为首个常染色体显性多囊性肾病变的疾病修正治疗，标誌着一个重要的里程碑。托伐普坦透过阻断加压素V2受体来减缓囊肿生长并维持肾功能。 TEMPO3:4等临床试验表明，接受託伐普坦治疗的患者肾臟体积增长减少了30%，肾功能下降速度也减缓，这使得该药物成为常染色体显性多囊肾病的突破性疗法。这项成功刺激了研发投入的增加，多家製药公司正在寻求疗效和安全性更高的下一代药物。

此外，随着全球人口老化，患有常染色体显性多囊性肾病变 (ADKD) 等慢性疾病的患者数量正在增加。 AKD 患者通常在四、五十岁时出现高血压、心血管併发症和肾衰竭。世界银行预测，到 2050 年，全球 65 岁及以上人口将达到 15 亿，这将增加对慢性病管理的需求。这一人口趋势正在扩大对综合治疗方案的需求，这些方案既能解决肾功能问题，又能治疗相关合併症，从而刺激 AKDD 市场的成长。

然而，高昂的治疗费用和有限的治疗选择阻碍了常染色体显性多囊性肾病变市场的成长。例如，在美国，托伐普坦疗法的年费用可能超过5万美元，这给患者和医疗保健系统带来了经济障碍。高昂的价格限制了治疗的可及性，尤其是在报销和保险覆盖有限的中低收入国家。

此外，托伐普坦目前是唯一核准专门用于治疗常染色体显性多囊性肾病变的缓解疾病药物，大多数患者依赖支持性治疗，例如使用血管紧张素转换酶 (ACE) 抑制剂或血管紧张素受体阻断剂 (ARB) 来控制血压。有效药物疗法的匮乏凸显了巨大的未满足医疗需求。这一缺口正在推动新型候选药物的临床试验，包括Sirolimus等 mTOR 抑制剂、生长抑制素类似物，以及旨在更有效控制疾病进展的新型基因治疗药物。

常染色体显性多囊性肾病变市场的主要企业正在采取多项策略性倡议，以提升竞争优势并抓住成长机会。大冢製药等主要企业正在大力投资研发，以扩大其下一代疗法组合，例如托伐普坦，该疗法比现有疗法具有更高的疗效和安全性。例如，大冢製药持续强化托伐普坦的临床应用，探索联合治疗和新型製剂，以提高患者的依从性和耐受性。

此外，为了加速常染色体显性多囊性肾病变的技术创新和成功的临床试验，公司正在与生物技术公司、学术机构和诊断公司建立策略联盟和合作，从而推动市场成长。

本报告研究了全球常染色体显性多囊性肾病变市场，概述了市场以及区域趋势和参与市场的公司概况。

目录

执行摘要

第一章全球常染色体显性多囊性肾病变市场：产业展望

  • 市场概览
  • 常染色体显性多囊性肾病变的流行病学分析
  • 监管状况
  • 主要趋势
  • 临床试验分析
  • 市场动态
    • 影响分析
    • 市场驱动因素
    • 市场限制
    • 市场机会

2. 全球常染色体显性多囊性肾病变市场（按地区，百万美元，2023-2035 年）

  • 北美洲
  • 欧洲
  • 亚太地区

3. 全球常染色体显性多囊性肾病变市场：竞争格局与公司概况

  • 竞争格局
    • 关键策略和发展
  • 公司简介
    • Otsuka Pharmaceutical
    • Reata Pharmaceuticals
    • Janssen Pharmaceuticals (Johnson & Johnson)
    • Vertex Pharmaceuticals Incorporated
    • PKD
    • Centessa Pharmaceuticals
    • Novartis AG (Regulus Therapeutics)
    • XORTX Therapeutics Inc.

第四章调查方法

Product Code: BHL3170SA

Global Autosomal Dominant Polycystic Kidney Disease Market, Analysis and Forecast: 2025-2035

Autosomal dominant polycystic kidney disease is a genetic disorder characterized by the progressive development of numerous fluid-filled cysts in the kidneys. These cysts enlarge over time, leading to kidney enlargement, loss of renal function, and eventually end-stage renal disease (ESRD). Autosomal dominant polycystic kidney disease is one of the most common inherited kidney disorders, affecting approximately 1 in 500 to 1,000 individuals globally. The disease is caused primarily by mutations in the PKD1 and PKD2 genes, which disrupt normal kidney cell growth and function. Symptoms often begin in adulthood and include hypertension, flank pain, urinary tract infections, and kidney stones. Autosomal dominant polycystic kidney disease significantly impacts patient quality of life and imposes a substantial healthcare burden due to the progressive nature of renal impairment and the need for dialysis or kidney transplantation in advanced stages.

Autosomal dominant polycystic kidney disease market is witnessing robust growth fueled by advancements in understanding the disease pathophysiology, the emergence of targeted therapies for autosomal dominant polycystic kidney disease, and increasing diagnosis rates. Historically, management of autosomal dominant polycystic kidney disease was largely supportive, focusing on blood pressure control and symptom relief. However, the approval of tolvaptan (Jynarque) by Otsuka Pharmaceuticals marked a breakthrough as the first disease-modifying therapy that slows cyst growth and renal function decline by antagonizing vasopressin V2 receptors.

The rising prevalence of the autosomal dominant polycystic kidney disease is one of the major factors driving the market. Autosomal dominant polycystic kidney disease affects approximately 4 to 6 million people worldwide, with prevalence estimates ranging from 1 in 500 to 1,000 individuals. Increasing awareness and advancements in diagnostic tools such as high-resolution imaging techniques (MRI and ultrasound) and genetic testing-have enabled earlier and more accurate diagnosis. For instance, in the U.S., the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) reports that earlier detection is becoming more common, allowing patients to receive timely interventions that can delay disease progression. The global rise in autosomal dominant polycystic kidney disease diagnosis is expanding the patient pool requiring pharmacological treatment, which in turn is driving growth in the drug market.

Moreover, the approval of tolvaptan (marketed as Jynarque by Otsuka Pharmaceuticals) in 2018 by the FDA marked a significant milestone as the first disease-modifying therapy for autosomal dominant polycystic kidney disease. Tolvaptan works by blocking vasopressin V2 receptors to slow cyst growth and preserve kidney function. Clinical trials such as the TEMPO 3:4 study demonstrated a 30% reduction in kidney volume growth and a slower decline in kidney function among treated patients, positioning tolvaptan as a breakthrough in autosomal dominant polycystic kidney disease management. This success has encouraged increased R&D investments, with several pharmaceutical companies exploring next-generation agents to improve efficacy and safety profiles.

In addition, as global populations age, the number of patients with chronic conditions like autosomal dominant polycystic kidney disease is increasing. Autosomal dominant polycystic kidney disease patients frequently develop hypertension, cardiovascular complications, and renal failure in their 40s and 50s. The World Bank estimates that the global population aged 65 and above will reach 1.5 billion by 2050, intensifying demand for chronic disease management. This demographic trend expands the need for comprehensive treatment options addressing both kidney function and associated comorbidities, thereby fueling growth in the autosomal dominant polycystic kidney disease market.

However, the high treatment cost and limited treatment options are some of the factors hindering the autosomal dominant polycystic kidney disease market growth. For instance, the annual cost of tolvaptan therapy can exceed $50,000 in the U.S., creating substantial affordability barriers for patients and healthcare systems. This high price limits access, particularly in low- and middle-income countries where reimbursement and insurance coverage are limited.

Moreover, currently, tolvaptan is the only approved disease-modifying drug specifically for autosomal dominant polycystic kidney disease, with most patients relying on supportive care such as blood pressure management using ACE inhibitors or ARBs. The scarcity of effective pharmacotherapies underscores a significant unmet medical need. This gap drives ongoing clinical trials for new candidates, including mTOR inhibitors like sirolimus, somatostatin analogs, and emerging gene therapies aimed at addressing disease progression more effectively.

Key players in the autosomal dominant polycystic kidney disease market are adopting several strategic initiatives to gain a competitive edge and capitalize on growing opportunities. Leading companies such as Otsuka Pharmaceuticals are investing heavily in research and development to expand their portfolios with next-generation therapies that offer improved efficacy and safety over existing treatments, such as tolvaptan. For instance, Otsuka continues to enhance tolvaptan's clinical applications while exploring combination therapies and novel formulations to improve patient adherence and tolerability.

Additionally, companies are forming strategic collaborations and partnerships with biotech firms, academic institutions, and diagnostic companies to accelerate innovation and clinical trial success for autosomal dominant polycystic kidney disease, thereby impelling the market growth.

Table of Contents

Executive Summary

Scope and Definition

Market/Product Definition

Key Questions Answered

Analysis and Forecast Note

1. Global Autosomal Dominant Polycystic Kidney Disease Market: Industry Outlook

  • 1.1 Market Overview
  • 1.2 Epidemiological Analysis of Autosomal Dominant Polycystic Kidney Disease
  • 1.3 Regulatory Landscape
  • 1.4 Key Trends
  • 1.5 Clinical Trial Analysis
  • 1.6 Market Dynamics
    • 1.6.1 Impact Analysis
    • 1.6.2 Market Drivers
    • 1.6.3 Market Restraint
    • 1.6.4 Market Opportunities

2. Global Autosomal Dominant Polycystic Kidney Disease Market, by Region, $Million, 2023-2035

  • 2.1 North America
    • 2.1.1 Market Dynamics
    • 2.1.2 Market Sizing and Forecast
      • 2.1.2.1 North America Autosomal Dominant Polycystic Kidney Disease Market, by Country
        • 2.1.2.1.1 U.S.
  • 2.2 Europe
    • 2.2.1 Market Dynamics
    • 2.2.2 Market Sizing and Forecast
      • 2.2.2.1 Europe Autosomal Dominant Polycystic Kidney Disease Market, by Country
        • 2.2.2.1.1 Germany
        • 2.2.2.1.2 U.K.
        • 2.2.2.1.3 France
        • 2.2.2.1.4 Italy
        • 2.2.2.1.5 Spain
  • 2.3 Asia-Pacific
    • 2.3.1 Market Dynamics
    • 2.3.2 Market Sizing and Forecast
      • 2.3.2.1 Asia-Pacific Autosomal Dominant Polycystic Kidney Disease Market, by Country
        • 2.3.2.1.1 Japan

3. Global Autosomal Dominant Polycystic Kidney Disease Market: Competitive Landscape and Company Profiles

  • 3.1 Competitive Landscape
    • 3.1.1 Key Strategies and Developments
      • 3.1.1.1 Funding Activities
      • 3.1.1.2 Mergers and Acquisitions
      • 3.1.1.3 Regulatory Approvals
      • 3.1.1.4 Partnerships, Collaborations and Business Expansions
  • 3.2 Company Profiles
    • 3.2.1 Otsuka Pharmaceutical
      • 3.2.1.1 Overview
      • 3.2.1.2 Product Portfolio
      • 3.2.1.3 Target Customers
      • 3.2.1.4 Key Personnel
      • 3.2.1.5 Analyst View
    • 3.2.2 Reata Pharmaceuticals
      • 3.2.2.1 Overview
      • 3.2.2.2 Product Portfolio
      • 3.2.2.3 Target Customers
      • 3.2.2.4 Key Personnel
      • 3.2.2.5 Analyst View
    • 3.2.3 Janssen Pharmaceuticals (Johnson & Johnson)
      • 3.2.3.1 Overview
      • 3.2.3.2 Product Portfolio
      • 3.2.3.3 Target Customers
      • 3.2.3.4 Key Personnel
      • 3.2.3.5 Analyst View
    • 3.2.4 Vertex Pharmaceuticals Incorporated
      • 3.2.4.1 Overview
      • 3.2.4.2 Product Portfolio
      • 3.2.4.3 Target Customers
      • 3.2.4.4 Key Personnel
      • 3.2.4.5 Analyst View
    • 3.2.5 PKD
      • 3.2.5.1 Overview
      • 3.2.5.2 Product Portfolio
      • 3.2.5.3 Target Customers
      • 3.2.5.4 Key Personnel
      • 3.2.5.5 Analyst View
    • 3.2.6 Centessa Pharmaceuticals
      • 3.2.6.1 Overview
      • 3.2.6.2 Product Portfolio
      • 3.2.6.3 Target Customers
      • 3.2.6.4 Key Personnel
      • 3.2.6.5 Analyst View
    • 3.2.7 Novartis AG (Regulus Therapeutics)
      • 3.2.7.1 Overview
      • 3.2.7.2 Product Portfolio
      • 3.2.7.3 Target Customers
      • 3.2.7.4 Key Personnel
      • 3.2.7.5 Analyst View
    • 3.2.8 XORTX Therapeutics Inc.
      • 3.2.8.1 Overview
      • 3.2.8.2 Product Portfolio
      • 3.2.8.3 Target Customers
      • 3.2.8.4 Key Personnel
      • 3.2.8.5 Analyst View

4. Research Methodology

List of Figures

  • Figure: Global Autosomal Dominant Polycystic Kidney Disease Market, Market Overview
  • Figure: Global Autosomal Dominant Polycystic Kidney Disease Market, Epidemiological Analysis
  • Figure: Global Autosomal Dominant Polycystic Kidney Disease Market Key Trends, Impact Analysis, 2023-2035
  • Figure: Global Autosomal Dominant Polycystic Kidney Disease Market, Competitive Landscape, January 2022-April 2025

List of Tables

  • Table: Global Autosomal Dominant Polycystic Kidney Disease Market, Regulatory Scenario
  • Table: Global Autosomal Dominant Polycystic Kidney Disease Market Dynamics, Impact Analysis