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市場調查報告書
商品編碼
1909037

龐貝氏症治療市場規模、佔有率和成長分析（按類型、診斷、疾病類型、治療方法、給藥途徑、分銷管道、最終用戶和地區分類）—2026-2033年產業預測

Pompe Disease Treatment Market Size, Share, and Growth Analysis, By Type, By Diagnosis, By Disease Type, By Treatment, By Route of Administration, By Distribution Channel, By End User, By Region - Industry Forecast 2026-2033
出版日期: | 出版商: SkyQuest | 英文 191 Pages | 商品交期: 3-5個工作天內

價格
簡介目錄

全球龐貝氏症治療市場規模預計在 2024 年達到 22.4 億美元，從 2025 年的 24 億美元成長到 2033 年的 40.9 億美元，在預測期（2026-2033 年）內複合年成長率為 6.9%。

由於龐貝氏症發病率上升以及對有效治療方法的需求不斷成長，全球龐貝氏症治療市場正經歷強勁成長。診斷能力的提高以及患者和醫療專業人員對該疾病認知的增強，增加了診斷和治療機會，從而擴大了需要治療的患者群體。醫療機構和政府將罕見疾病列為優先事項，推動了醫療服務的可及性和報銷範圍的擴大。相關人員之間圍繞著治療效果、病患留存率和長期臨床結果的競爭，進一步推動了市場動態。包括新一代酵素替代療法和創新基因療法在內的最新技術進步，徹底改變了治療選擇，透過實現卓越的臨床療效和長期糾正酵素缺陷，為永續的醫療保健解決方案提供了可能。

全球龐貝氏症治療市場促進因素

龐貝氏症在全球範圍內的日益普及是推動有效治療方案需求成長的主要因素。人們對該疾病的認知不斷提高，診斷能力也隨之進步，導致確診病例增多，進而增加了對治療性介入的需求。不斷成長的患者群體促使醫療機構和製藥公司開發並實施針對該疾病的長期管理策略。因此，全球龐貝氏症治療市場在各個地區都呈現快速成長的態勢，這反映出各方正齊心協力地透過改善醫療服務可及性和提供創新治療方案來滿足該疾病患者的需求。

限制全球龐貝氏症治療市場的因素

全球龐貝氏症治療市場面臨的主要阻礙因素是現有治療方法（尤其是需要終身治療的酵素替代療法）的高昂費用。在許多地區，尤其是在較不富裕的國家，這些治療的保險覆蓋範圍極小甚至沒有，嚴重阻礙了患者獲得治療。高昂的費用往往導致診斷和治療啟動的延誤，從而限制了整體市場滲透率，並削弱了有效長期疾病管理的潛力。因此，這些經濟挑戰阻礙了患者及時獲得適當的龐貝氏症治療。

全球龐貝氏症治療市場趨勢

全球龐貝氏症治療市場正經歷著向基因療法的重大轉變，基因療法作為一種潛在的變革性長期解決方案，備受關注。與需要持續用藥的傳統酵素替代療法不同，基因療法旨在透過一次性治療恢復功能性GAA酶的產生，從而解決疾病的根本遺傳原因。這種模式轉移吸引了大量投資，並加快了臨床試驗的步伐，顯示醫學界對基因療法的前景日益樂觀。對基因療法的關注代表著一個極具吸引力的市場趨勢，並有望在未來幾年重新定義龐貝氏症的治療策略。

目錄

介紹

  • 調查目標
  • 調查範圍
  • 定義

調查方法

  • 資訊收集
  • 二手資料和一手資料方法
  • 市場規模預測
  • 市場假設與限制

執行摘要

  • 全球市場展望
  • 供需趨勢分析
  • 細分市場機會分析

市場動態與展望

  • 市場規模
  • 市場動態
    • 促進因素和機遇
    • 限制與挑戰
  • 波特分析

關鍵市場考察

  • 關鍵成功因素
  • 競爭程度
  • 關鍵投資機會
  • 市場生態系統
  • 市場吸引力指數（2025）
  • PESTEL 分析
  • 總體經濟指標
  • 價值鏈分析
  • 定價分析

全球龐貝氏症治療市場規模（按類型和複合年成長率分類）（2026-2033 年）

  • 經典嬰兒型
  • 非典型嬰兒型
  • 晚髮型
  • 其他

全球龐貝氏症治療市場規模（按診斷和複合年成長率分類）（2026-2033 年）

  • 血液檢查
  • 基因檢測
  • 產前檢查
  • 其他

全球龐貝氏症治療市場規模（按疾病類型和複合年成長率分類）（2026-2033 年）

  • 嬰兒型龐貝氏症
  • 晚髮型龐貝氏症
  • 經典型龐貝氏症

全球龐貝氏症治療市場規模（依治療方法分類）及複合年成長率（2026-2033 年）

  • 酵素替代療法
  • 支持性護理
  • 其他

全球龐貝氏症治療市場規模（依給藥途徑及複合年成長率分類）（2026-2033 年）

  • 口服
  • 腸外
  • 其他

全球龐貝氏症治療市場規模（依通路分類）及複合年成長率（2026-2033 年）

  • 醫院藥房
  • 網路藥房
  • 零售藥房

全球龐貝氏症治療市場規模（按最終用戶分類）及複合年成長率（2026-2033 年）

  • 醫院
  • 居家醫療
  • 專業醫療中心
  • 其他

全球龐貝氏症治療市場規模（按地區分類）及複合年成長率（2026-2033 年）

  • 北美洲
    • 美國
    • 加拿大
  • 歐洲
    • 德國
    • 西班牙
    • 法國
    • 英國
    • 義大利
    • 其他歐洲地區
  • 亞太地區
    • 中國
    • 印度
    • 日本
    • 韓國
    • 亞太其他地區
  • 拉丁美洲
    • 巴西
    • 其他拉丁美洲地區
  • 中東和非洲
    • 海灣合作理事會國家
    • 南非
    • 其他中東和非洲地區

競爭資訊

  • 前五大公司對比
  • 主要企業的市場定位（2025 年）
  • 主要市場參與者所採取的策略
  • 近期市場趨勢
  • 公司市佔率分析（2025 年）
  • 主要企業公司簡介
    • 公司詳情
    • 產品系列分析
    • 依業務板塊進行公司股票分析
    • 2023-2025年營收年比比較

主要企業簡介

  • Sanofi（France）
  • Amicus Therapeutics, Inc.（USA）
  • Astellas Pharma（Japan）
  • Roche（Switzerland）
  • Shionogi & Co., Ltd.（Japan）
  • Maze Therapeutics, Inc.（USA）
  • Spark Therapeutics（USA）
  • AVROBIO, Inc.（USA）
  • Asklepios Biopharmaceutical（USA）
  • Takeda Pharmaceutical Company Limited（Japan）
  • BioMarin Pharmaceutical Inc.（USA）
  • Genethon（France）
  • Entrada Therapeutics（USA）
  • Oxyrane（Belgium）
  • Aro Biotherapeutics（USA）
  • Valerion Therapeutics（USA）
  • Actus Therapeutics（USA）
  • M6P Therapeutics（USA）
  • GeneCradle Pharmaceutical（China）
  • Centogene AG（Germany）

結論與建議

簡介目錄
Product Code: SQMIG35D2353

Global Pompe Disease Treatment Market size was valued at USD 2.24 Billion in 2024 and is poised to grow from USD 2.4 Billion in 2025 to USD 4.09 Billion by 2033, growing at a CAGR of 6.9% during the forecast period (2026-2033).

The global Pompe disease treatment market is experiencing robust growth due to a rising incidence of Pompe disease and an increasing demand for effective therapies. Enhanced diagnostic capabilities and greater awareness among patients and healthcare professionals are leading to more diagnoses and treatment opportunities, expanding the patient population needing care. Health organizations and governments are prioritizing rare diseases, facilitating improved access and reimbursement options. Market dynamics are further intensified by competition among stakeholders focused on treatment efficacy, patient adherence, and long-term clinical outcomes. Recent advancements in technology, including next-generation enzyme replacement therapies and innovative gene therapy approaches, are revolutionizing treatment options, delivering superior clinical responses and targeting long-term correction of the enzymatic deficiency, thus enabling sustainable healthcare solutions.

Top-down and bottom-up approaches were used to estimate and validate the size of the Global Pompe Disease Treatment market and to estimate the size of various other dependent submarkets. The research methodology used to estimate the market size includes the following details: The key players in the market were identified through secondary research, and their market shares in the respective regions were determined through primary and secondary research. This entire procedure includes the study of the annual and financial reports of the top market players and extensive interviews for key insights from industry leaders such as CEOs, VPs, directors, and marketing executives. All percentage shares split, and breakdowns were determined using secondary sources and verified through Primary sources. All possible parameters that affect the markets covered in this research study have been accounted for, viewed in extensive detail, verified through primary research, and analyzed to get the final quantitative and qualitative data.

Global Pompe Disease Treatment Market Segments Analysis

Global Pompe Disease Treatment Market is segmented by Type, Diagnosis, Disease Type, Treatment, Route of Administration, Distribution Channel, End User and region. Based on Type, the market is segmented into Classic Infantile Form, Non-Classic Infantile Form, Late-Onset Form and Others. Based on Diagnosis, the market is segmented into Blood Test, Genetic Test, Prenatal Test and Others. Based on Disease Type, the market is segmented into Infantile Pompe Disease, Late-Onset Pompe Disease and Classical Pompe Disease. Based on Treatment, the market is segmented into Enzyme Replacement Therapy, Supportive Therapies and Others. Based on Route of Administration, the market is segmented into Oral, Parenteral and Others. Based on Distribution Channel, the market is segmented into Hospital Pharmacy, Online Pharmacy and Retail Pharmacy. Based on End User, the market is segmented into Hospitals, Homecare, Specialty Centers and Others. Based on region, the market is segmented into North America, Europe, Asia Pacific, Latin America and Middle East & Africa.

Driver of the Global Pompe Disease Treatment Market

The rising prevalence of Pompe disease worldwide serves as a significant catalyst for the demand for effective treatment solutions. Heightened awareness and advancements in diagnostic capabilities have led to an increase in diagnoses, which, in turn, fuels the need for therapeutic interventions. This growing population of patients motivates healthcare providers and pharmaceutical companies to develop and implement long-term management strategies for the condition. Consequently, the global landscape of the Pompe disease treatment market is experiencing swift growth across various regions, reflecting a concerted effort to address the needs of those affected by this condition through improved access to care and innovative treatment options.

Restraints in the Global Pompe Disease Treatment Market

A major constraint on the global Pompe disease treatment market is the exorbitant cost of available therapies, notably enzyme replacement therapy, which is a lifelong commitment. In many regions, particularly those outside of affluent countries, reimbursement for such treatments is minimal or nonexistent, creating significant barriers to access for patients. This high expense often leads to delays in diagnosis and initiation of treatment, which in turn restricts overall market penetration and diminishes the potential for effective long-term disease management. Consequently, these financial challenges hinder the ability of patients to receive timely and adequate care for Pompe disease.

Market Trends of the Global Pompe Disease Treatment Market

The global Pompe disease treatment market is witnessing a significant shift towards gene therapy, which is garnering considerable attention as a potentially transformative long-term solution. Unlike conventional enzyme replacement therapies that require ongoing administration, gene therapy aims to address the underlying genetic cause of the disease by restoring the production of the functional GAA enzyme through a one-time treatment. This paradigm shift is attracting substantial investments and accelerating the pace of clinical trials, highlighting an increasing optimism within the medical community. The focus on gene therapy signifies a compelling market trend that could redefine treatment strategies for Pompe disease in the coming years.

Table of Contents

Introduction

  • Objectives of the Study
  • Scope of the Report
  • Definitions

Research Methodology

  • Information Procurement
  • Secondary & Primary Data Methods
  • Market Size Estimation
  • Market Assumptions & Limitations

Executive Summary

  • Global Market Outlook
  • Supply & Demand Trend Analysis
  • Segmental Opportunity Analysis

Market Dynamics & Outlook

  • Market Overview
  • Market Size
  • Market Dynamics
    • Drivers & Opportunities
    • Restraints & Challenges
  • Porters Analysis
    • Competitive rivalry
    • Threat of substitute
    • Bargaining power of buyers
    • Threat of new entrants
    • Bargaining power of suppliers

Key Market Insights

  • Key Success Factors
  • Degree of Competition
  • Top Investment Pockets
  • Market Ecosystem
  • Market Attractiveness Index, 2025
  • PESTEL Analysis
  • Macro-Economic Indicators
  • Value Chain Analysis
  • Pricing Analysis

Global Pompe Disease Treatment Market Size by Type & CAGR (2026-2033)

  • Market Overview
  • Classic Infantile Form
  • Non-Classic Infantile Form
  • Late-Onset Form
  • Others

Global Pompe Disease Treatment Market Size by Diagnosis & CAGR (2026-2033)

  • Market Overview
  • Blood Test
  • Genetic Test
  • Prenatal Test
  • Others

Global Pompe Disease Treatment Market Size by Disease Type & CAGR (2026-2033)

  • Market Overview
  • Infantile Pompe Disease
  • Late-Onset Pompe Disease
  • Classical Pompe Disease

Global Pompe Disease Treatment Market Size by Treatment & CAGR (2026-2033)

  • Market Overview
  • Enzyme Replacement Therapy
  • Supportive Therapies
  • Others

Global Pompe Disease Treatment Market Size by Route of Administration & CAGR (2026-2033)

  • Market Overview
  • Oral
  • Parenteral
  • Others

Global Pompe Disease Treatment Market Size by Distribution Channel & CAGR (2026-2033)

  • Market Overview
  • Hospital Pharmacy
  • Online Pharmacy
  • Retail Pharmacy

Global Pompe Disease Treatment Market Size by End User & CAGR (2026-2033)

  • Market Overview
  • Hospitals
  • Homecare
  • Specialty Centers
  • Others

Global Pompe Disease Treatment Market Size & CAGR (2026-2033)

  • North America (Type, Diagnosis, Disease Type, Treatment, Route of Administration, Distribution Channel, End User)
    • US
    • Canada
  • Europe (Type, Diagnosis, Disease Type, Treatment, Route of Administration, Distribution Channel, End User)
    • Germany
    • Spain
    • France
    • UK
    • Italy
    • Rest of Europe
  • Asia Pacific (Type, Diagnosis, Disease Type, Treatment, Route of Administration, Distribution Channel, End User)
    • China
    • India
    • Japan
    • South Korea
    • Rest of Asia-Pacific
  • Latin America (Type, Diagnosis, Disease Type, Treatment, Route of Administration, Distribution Channel, End User)
    • Brazil
    • Rest of Latin America
  • Middle East & Africa (Type, Diagnosis, Disease Type, Treatment, Route of Administration, Distribution Channel, End User)
    • GCC Countries
    • South Africa
    • Rest of Middle East & Africa

Competitive Intelligence

  • Top 5 Player Comparison
  • Market Positioning of Key Players, 2025
  • Strategies Adopted by Key Market Players
  • Recent Developments in the Market
  • Company Market Share Analysis, 2025
  • Company Profiles of All Key Players
    • Company Details
    • Product Portfolio Analysis
    • Company's Segmental Share Analysis
    • Revenue Y-O-Y Comparison (2023-2025)

Key Company Profiles

  • Sanofi (France)
    • Company Overview
    • Business Segment Overview
    • Financial Updates
    • Key Developments
  • Amicus Therapeutics, Inc. (USA)
    • Company Overview
    • Business Segment Overview
    • Financial Updates
    • Key Developments
  • Astellas Pharma (Japan)
    • Company Overview
    • Business Segment Overview
    • Financial Updates
    • Key Developments
  • Roche (Switzerland)
    • Company Overview
    • Business Segment Overview
    • Financial Updates
    • Key Developments
  • Shionogi & Co., Ltd. (Japan)
    • Company Overview
    • Business Segment Overview
    • Financial Updates
    • Key Developments
  • Maze Therapeutics, Inc. (USA)
    • Company Overview
    • Business Segment Overview
    • Financial Updates
    • Key Developments
  • Spark Therapeutics (USA)
    • Company Overview
    • Business Segment Overview
    • Financial Updates
    • Key Developments
  • AVROBIO, Inc. (USA)
    • Company Overview
    • Business Segment Overview
    • Financial Updates
    • Key Developments
  • Asklepios Biopharmaceutical (USA)
    • Company Overview
    • Business Segment Overview
    • Financial Updates
    • Key Developments
  • Takeda Pharmaceutical Company Limited (Japan)
    • Company Overview
    • Business Segment Overview
    • Financial Updates
    • Key Developments
  • BioMarin Pharmaceutical Inc. (USA)
    • Company Overview
    • Business Segment Overview
    • Financial Updates
    • Key Developments
  • Genethon (France)
    • Company Overview
    • Business Segment Overview
    • Financial Updates
    • Key Developments
  • Entrada Therapeutics (USA)
    • Company Overview
    • Business Segment Overview
    • Financial Updates
    • Key Developments
  • Oxyrane (Belgium)
    • Company Overview
    • Business Segment Overview
    • Financial Updates
    • Key Developments
  • Aro Biotherapeutics (USA)
    • Company Overview
    • Business Segment Overview
    • Financial Updates
    • Key Developments
  • Valerion Therapeutics (USA)
    • Company Overview
    • Business Segment Overview
    • Financial Updates
    • Key Developments
  • Actus Therapeutics (USA)
    • Company Overview
    • Business Segment Overview
    • Financial Updates
    • Key Developments
  • M6P Therapeutics (USA)
    • Company Overview
    • Business Segment Overview
    • Financial Updates
    • Key Developments
  • GeneCradle Pharmaceutical (China)
    • Company Overview
    • Business Segment Overview
    • Financial Updates
    • Key Developments
  • Centogene AG (Germany)
    • Company Overview
    • Business Segment Overview
    • Financial Updates
    • Key Developments

Conclusion & Recommendations