常染色體顯性多囊性腎病變市場 - 全球及區域 - 分析與預測（2025-2035）

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

出版日期: 2025年08月14日 | 出版商:

BIS Research | 英文 100 Pages | 商品交期: 1-5個工作天內

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簡介目錄圖表

體染色體顯性多囊性腎病變是一種遺傳性疾病，其特徵是腎臟中進行性形成大量充滿液體的囊腫。

這些囊腫會隨著時間而成長，導致腎臟腫大、腎功能下降，最終導致末期腎功能衰竭(ESRD)。體染色體顯性多囊性腎病變 (ADKD) 是最常見的遺傳性腎病變之一，全球每 500 到 1,000 人中約有 1 人患有此病。此疾病主要由 PKD1 和 PKD2 基因突變引起，會損害正常的腎細胞生長和功能。症狀通常在成年期開始，包括高血壓、腰痛、尿道感染和腎結石。常染色體顯性多囊性腎病變 (ADKD) 會嚴重影響患者的生活品質，並給醫療保健帶來沉重的負擔，因為腎功能障礙進行性，可能需要進行透析或腎臟移植。

常染色體顯性多囊性腎病變 (ADKD) 市場正經歷強勁成長，這得益於對該疾病病理學理解的不斷深入、ADC標靶治療的出現以及診斷率的不斷提高。以往，ADC 的治療主要著重於支持性治療，重點在於控制血壓和緩解症狀。然而，大塚製藥核准托伐普坦 (Zinalc) 標誌著一項里程碑，這是首個透過拮抗加壓素 V2 受體來減緩囊腫生長和腎衰竭的疾病修正治療。

常染色體顯性多囊性腎病變 (ADKD) 盛行率的不斷上升是推動市場的主要因素之一。 ADC 影響全球約 400 萬至 600 萬人，估計盛行率為 1/500 至 1/1,000。隨著人們所認知的提高和診斷工具的進步，例如高解析度成像技術（MRI 和超音波）和基因檢測，人們能夠更早、更準確地進行診斷。例如，在美國，美國國家糖尿病、消化和腎臟疾病研究所 (NIDDK) 的一份報告指出，早期發現正變得越來越普遍，這使得患者能夠及時接受干涉，從而減緩病情的進展。全球 ADC 診斷數量的增加正在擴大需要藥物治療的患者群體，從而推動醫藥市場的成長。

此外，2018年美國食品藥物管理局（FDA）核准托伐普坦（由大塚製藥以Jynarque為商品名上市）作為首個常染色體顯性多囊性腎病變的疾病修正治療，標誌著一個重要的里程碑。托伐普坦透過阻斷加壓素V2受體來減緩囊腫生長並維持腎功能。 TEMPO3:4等臨床試驗表明，接受託伐普坦治療的患者腎臟體積成長減少了30%，腎功能下降速度也減緩，這使得該藥物成為常染色體顯性多囊腎病的突破性療法。這項成功刺激了研發投入的增加，多家製藥公司正在尋求療效和安全性更高的下一代藥物。

此外，隨著全球人口老化，患有常染色體顯性多囊性腎病變 (ADKD) 等慢性疾病的患者數量正在增加。 AKD 患者通常在四、五十歲時出現高血壓、心血管併發症和腎衰竭。世界銀行預測，到 2050 年，全球 65 歲及以上人口將達到 15 億，這將增加對慢性病管理的需求。這一人口趨勢正在擴大對綜合治療方案的需求，這些方案既能解決腎功能問題，又能治療相關合併症，從而刺激 AKDD 市場的成長。

然而，高昂的治療費用和有限的治療選擇阻礙了常染色體顯性多囊性腎病變市場的成長。例如，在美國，托伐普坦療法的年費用可能超過5萬美元，這給患者和醫療保健系統帶來了經濟障礙。高昂的價格限制了治療的可及性，尤其是在報銷和保險覆蓋有限的中低收入國家。

此外，托伐普坦目前是唯一核准專門用於治療常染色體顯性多囊性腎病變的緩解疾病藥物，大多數患者依賴支持性治療，例如使用血管緊張素轉換酶 (ACE) 抑制劑或血管緊張素受體阻斷劑 (ARB) 來控制血壓。有效藥物療法的匱乏凸顯了巨大的未滿足醫療需求。這一缺口正在推動新型候選藥物的臨床試驗，包括Sirolimus等 mTOR 抑制劑、生長抑制素類似物，以及旨在更有效控制疾病進展的新型基因治療藥物。

常染色體顯性多囊性腎病變市場的主要企業正在採取多項策略性舉措，以提升競爭優勢並抓住成長機會。大塚製藥等主要企業正在大力投資研發，以擴大其下一代療法組合，例如托伐普坦，該療法比現有療法具有更高的療效和安全性。例如，大塚製藥持續強化托伐普坦的臨床應用，探索聯合治療和新型製劑，以提高患者的依從性和耐受性。

此外，為了加速常染色體顯性多囊性腎病變的技術創新和成功的臨床試驗，公司正在與生物技術公司、學術機構和診斷公司建立策略聯盟和合作，從而推動市場成長。

本報告研究了全球常染色體顯性多囊性腎病變市場，概述了市場以及區域趨勢和參與市場的公司概況。

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.

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

簡介目錄圖表

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

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