세계의 폼페병 치료 시장 : 치료별, 투여 경로별, 유통 채널별, 적응증 유형별, 지역별(2025-2033년)

The global Pompe disease treatment market size reached USD 1.07 Billion in 2024. Looking forward, IMARC Group expects the market to reach USD 1.33 Billion by 2033, exhibiting a growth rate (CAGR) of 2.47% during 2025-2033. The increasing number of clinical research and the growing popularity of enzyme replacement therapy (ERT) are stimulating the market.

Pompe disease is a genetic disorder caused by the buildup of a complex sugar known as glycogen in the cells of the body, which affects organs, tissues, and muscles. It is an autosomal recessive glycogen storage syndrome caused by the deficiency of lysosomal enzyme acid a-glucosidase (GAA). It is associated with weak muscles, enlarged liver, trouble breathing, hearing problem, lung infection, irregular heartbeat, and losing weight. It is diagnosed using various tools and tests, including electromyography, X-rays, and magnetic resonance imaging (MRI). Pompe disease can be treated using medications, enzyme replacement therapy (ERT), substrate reduction therapy (SRT), chaperone-advanced replacement therapy (CART), and injections, which aid in improving the quality of life.

Pompe Disease Treatment Market Trends:

There is a considerable rise in the prevalence of genetic abnormalities among individuals and an increase in the number of patients suffering from Pompe disease. This, coupled with the growing Pompe disease among infants and increasing concerns among parents about the health of their newborns, represents one of the key factors bolstering the growth of the market. Moreover, the growing geriatric population that is susceptible to developing these medical disorders is resulting in an increasing demand for Pompe disease treatment. In addition, the expanding number of clinical studies and trials to develop new treatments for Pompe disease is influencing the market positively. Apart from this, the rising awareness among people about ERT for curing Pompe disease is favoring the market growth. Furthermore, there is an increase in awareness about various treatments, including stem cell transplant, bone marrow transplantation, chemotherapy, and immunotherapy, on account of their numerous advantages. This, along with the awareness about the easy availability of ERT treatment alternatives like lumizyme, is creating a positive outlook for the market. Besides this, there is a rise in the adoption of health insurance policies that provide financial assistance and minimize healthcare expenditure for Pompe disease treatment. Additionally, governments and non-governmental organizations (NGOs) of numerous countries are undertaking measures to offer quality healthcare facilities that are accessible to all. This, along with significant improvements in the diagnostic technologies, is anticipated to provide a favorable outlook to the market.

Key Market Segmentation:

Treatment Insights:

• Enzyme Replacement Therapy (ERT)
• Substrate Reduction Therapy (SRT)
• Chaperone-Advanced Replacement Therapy (CART)
• Others

Route of Administration Insights:

• Oral
• Intravenous
• Others

Distribution Channel Insights:

• Hospital and Clinics Pharmacies
• Retail Pharmacies
• Online Pharmacies
• Others

Indication Type Insights:

• Infantile-Onset Pompe Disease (IOPD)
• Classic Infantile Form
• Non-Classic Infantile Form
• Late-Onset Pompe Disease (LOPD)
• Others

Regional Insights:

• North America
• United States
• Canada
• Asia Pacific
• China
• Japan
• India
• South Korea
• Australia
• Indonesia
• Others
• Europe
• Germany
• France
• United Kingdom
• Italy
• Spain
• Russia
• Others
• Latin America
• Brazil
• Mexico
• Others
• Middle East and Africa
• The report has also provided a comprehensive analysis of all the major regional markets that include North America (the United States and Canada), Asia Pacific (China, Japan, India, South Korea, Australia, Indonesia, and others), Europe (Germany, France, the United Kingdom, Italy, Spain, Russia, and others), Latin America (Brazil, Mexico, and others), and the Middle East and Africa. According to the report, North America was the largest market for Pompe disease treatment. Some of the factors driving the North America Pompe disease treatment market included the growing awareness about various alternative treatments, increasing number of clinical studies and trials, and well-established healthcare facilities.

Competitive Landscape:

• The report has also provided a comprehensive analysis of the competitive landscape in the global Pompe disease treatment market. Detailed profiles of all major companies have also been provided. Some of the companies covered include Amicus Therapeutics Inc., Audentes Therapeutics Inc. (Astellas US Holding Inc.), Oxyrane UK Limited, Sanofi S.A., Spark Therapeutics Inc, etc.

Key Questions Answered in This Report

• 1.How big is the pompe disease treatment market?
• 2.What is the future outlook of pompe disease treatment market?
• 3.What are the key factors driving the pompe disease treatment market?
• 4.Which region accounts for the largest pompe disease treatment market share?
• 5.Which are the leading companies in the global pompe disease treatment market?

Table of Contents

1 Preface

2 Scope and Methodology

• 2.1 Objectives of the Study
• 2.2 Stakeholders
• 2.3 Data Sources
  • 2.3.1 Primary Sources
  • 2.3.2 Secondary Sources
• 2.4 Market Estimation
  • 2.4.1 Bottom-Up Approach
  • 2.4.2 Top-Down Approach
• 2.5 Forecasting Methodology

3 Executive Summary

4 Introduction

• 4.1 Overview
• 4.2 Key Industry Trends

5 Global Pompe Disease Treatment Market

• 5.1 Market Overview
• 5.2 Market Performance
• 5.3 Impact of COVID-19
• 5.4 Market Forecast

6 Market Breakup by Treatment

• 6.1 Enzyme Replacement Therapy (ERT)
  • 6.1.1 Market Trends
  • 6.1.2 Market Forecast
• 6.2 Substrate Reduction Therapy (SRT)
  • 6.2.1 Market Trends
  • 6.2.2 Market Forecast
• 6.3 Chaperone-Advanced Replacement Therapy (CART)
  • 6.3.1 Market Trends
  • 6.3.2 Market Forecast
• 6.4 Others
  • 6.4.1 Market Trends
  • 6.4.2 Market Forecast

7 Market Breakup by Route of Administration

• 7.1 Oral
  • 7.1.1 Market Trends
  • 7.1.2 Market Forecast
• 7.2 Intravenous
  • 7.2.1 Market Trends
  • 7.2.2 Market Forecast
• 7.3 Others
  • 7.3.1 Market Trends
  • 7.3.2 Market Forecast

8 Market Breakup by Distribution Channel

• 8.1 Hospital & Clinics Pharmacies
  • 8.1.1 Market Trends
  • 8.1.2 Market Forecast
• 8.2 Retail Pharmacies
  • 8.2.1 Market Trends
  • 8.2.2 Market Forecast
• 8.3 Online Pharmacies
  • 8.3.1 Market Trends
  • 8.3.2 Market Forecast
• 8.4 Others
  • 8.4.1 Market Trends
  • 8.4.2 Market Forecast

9 Market Breakup by Indication Type

• 9.1 Infantile-Onset Pompe Disease (IOPD)
  • 9.1.1 Market Trends
  • 9.1.2 Market Forecast
• 9.2 Classic Infantile Form
  • 9.2.1 Market Trends
  • 9.2.2 Market Forecast
• 9.3 Non-Classic Infantile Form
  • 9.3.1 Market Trends
  • 9.3.2 Market Forecast
• 9.4 Late-Onset Pompe Disease (LOPD)
  • 9.4.1 Market Trends
  • 9.4.2 Market Forecast
• 9.5 Others
  • 9.5.1 Market Trends
  • 9.5.2 Market Forecast

10 Market Breakup by Region

• 10.1 North America
  • 10.1.1 United States
    • 10.1.1.1 Market Trends
    • 10.1.1.2 Market Forecast
  • 10.1.2 Canada
    • 10.1.2.1 Market Trends
    • 10.1.2.2 Market Forecast
• 10.2 Asia-Pacific
  • 10.2.1 China
    • 10.2.1.1 Market Trends
    • 10.2.1.2 Market Forecast
  • 10.2.2 Japan
    • 10.2.2.1 Market Trends
    • 10.2.2.2 Market Forecast
  • 10.2.3 India
    • 10.2.3.1 Market Trends
    • 10.2.3.2 Market Forecast
  • 10.2.4 South Korea
    • 10.2.4.1 Market Trends
    • 10.2.4.2 Market Forecast
  • 10.2.5 Australia
    • 10.2.5.1 Market Trends
    • 10.2.5.2 Market Forecast
  • 10.2.6 Indonesia
    • 10.2.6.1 Market Trends
    • 10.2.6.2 Market Forecast
  • 10.2.7 Others
    • 10.2.7.1 Market Trends
    • 10.2.7.2 Market Forecast
• 10.3 Europe
  • 10.3.1 Germany
    • 10.3.1.1 Market Trends
    • 10.3.1.2 Market Forecast
  • 10.3.2 France
    • 10.3.2.1 Market Trends
    • 10.3.2.2 Market Forecast
  • 10.3.3 United Kingdom
    • 10.3.3.1 Market Trends
    • 10.3.3.2 Market Forecast
  • 10.3.4 Italy
    • 10.3.4.1 Market Trends
    • 10.3.4.2 Market Forecast
  • 10.3.5 Spain
    • 10.3.5.1 Market Trends
    • 10.3.5.2 Market Forecast
  • 10.3.6 Russia
    • 10.3.6.1 Market Trends
    • 10.3.6.2 Market Forecast
  • 10.3.7 Others
    • 10.3.7.1 Market Trends
    • 10.3.7.2 Market Forecast
• 10.4 Latin America
  • 10.4.1 Brazil
    • 10.4.1.1 Market Trends
    • 10.4.1.2 Market Forecast
  • 10.4.2 Mexico
    • 10.4.2.1 Market Trends
    • 10.4.2.2 Market Forecast
  • 10.4.3 Others
    • 10.4.3.1 Market Trends
    • 10.4.3.2 Market Forecast
• 10.5 Middle East and Africa
  • 10.5.1 Market Trends
  • 10.5.2 Market Breakup by Country
  • 10.5.3 Market Forecast

11 Drivers, Restraints, and Opportunities

• 11.1 Overview
• 11.2 Drivers
• 11.3 Restraints
• 11.4 Opportunities

12 Value Chain Analysis

13 Porters Five Forces Analysis

• 13.1 Overview
• 13.2 Bargaining Power of Buyers
• 13.3 Bargaining Power of Suppliers
• 13.4 Degree of Competition
• 13.5 Threat of New Entrants
• 13.6 Threat of Substitutes

14 Price Analysis

15 Competitive Landscape

• 15.1 Market Structure
• 15.2 Key Players
• 15.3 Profiles of Key Players
  • 15.3.1 Amicus Therapeutics Inc.
    • 15.3.1.1 Company Overview
    • 15.3.1.2 Product Portfolio
    • 15.3.1.3 Financials
    • 15.3.1.4 SWOT Analysis
  • 15.3.2 Audentes Therapeutics Inc. (Astellas US Holding Inc.)
    • 15.3.2.1 Company Overview
    • 15.3.2.2 Product Portfolio
  • 15.3.3 Oxyrane UK Limited
    • 15.3.3.1 Company Overview
    • 15.3.3.2 Product Portfolio
  • 15.3.4 Sanofi S.A.
    • 15.3.4.1 Company Overview
    • 15.3.4.2 Product Portfolio
    • 15.3.4.3 Financials
    • 15.3.4.4 SWOT Analysis
  • 15.3.5 Spark Therapeutics Inc.
    • 15.3.5.1 Company Overview
    • 15.3.5.2 Product Portfolio

Kindly, note that this only represents a partial list of companies, and the complete list has been provided in the report