Pompe Disease Treatment Market by Treatment (Enzyme Replacement Therapy (ERT), Substrate Reduction Therapy (SRT), Chaperone-Advanced Replacement Therapy (CART), and Others), Route of Administration (Oral, Intravenous, and Others), Distribution Channel (Ho

Pompe Disease Treatment Market by Treatment (Enzyme Replacement Therapy (ERT), Substrate Reduction Therapy (SRT), Chaperone-Advanced Replacement Therapy (CART), and Others), Route of Administration (Oral, Intravenous, and Others), Distribution Channel (Hospital and Clinics Pharmacies, Retail Pharmacies, Online Pharmacies, and Others), Indication Type (Infantile-Onset Pompe Disease (IOPD), Classic Infantile Form, Non-Classic Infantile Form, Late-Onset Pompe Disease (LOPD), and Others), and Region 2024-2032


The global Pompe disease treatment market size reached US$ 1.0 Billion in 2023. Looking forward, IMARC Group expects the market to reach US$ 1.3 Billion by 2032, exhibiting a growth rate (CAGR) of 2.58% during 2024-2032. The increasing number of clinical studies and trials, the rising popularity of ERT, and the growing number of patients suffering from Pompe disease represent some of the key factors driving 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:
IMARC Group provides an analysis of the key trends in each segment of the global Pompe disease treatment market, along with forecasts at the global, regional, and country level from 2024-2032. Our report has categorized the market based on treatment, route of administration, distribution channel, and indication type.

Treatment Insights:

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

The report has provided a detailed breakup and analysis of the Pompe disease treatment market based on the treatment. This includes enzyme replacement therapy (ERT), substrate reduction therapy (SRT), chaperone-advanced replacement therapy (CART), and others. According to the report, enzyme replacement therapy (ERT) represented the largest segment.

Route of Administration Insights:

Oral
Intravenous
Others

A detailed breakup and analysis of the Pompe disease treatment market based on the route of administration has also been provided in the report. This includes oral, intravenous, and others.

Distribution Channel Insights:

Hospital and Clinics Pharmacies
Retail Pharmacies
Online Pharmacies
Others

A detailed breakup and analysis of the Pompe disease treatment market based on the distribution channel has also been provided in the report. This includes hospital and clinics, retail, and online pharmacies, and others. According to the report, hospital and clinics pharmacies accounted for the largest market share.

Indication Type Insights:

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

A detailed breakup and analysis of the Pompe disease treatment market based on the indication type has also been provided in the report. This includes infantile-onset Pompe disease (IOPD), classic infantile form, non-classic infantile form, late-onset Pompe disease (LOPD), and others. According to the report, late-onset Pompe disease (LOPD) accounted for the largest market share.

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:
How has the global Pompe disease treatment market performed so far and how will it perform in the coming years?
What are the drivers, restraints, and opportunities in the global Pompe disease treatment market?
What are the key regional markets?
Which countries represent the most attractive Pompe disease treatment markets?
What is the breakup of the market based on the treatment?
What is the breakup of the market based on the route of administration?
What is the breakup of the market based on the distribution channel?
What is the breakup of the market based on the indication type?
What is the competitive structure of the global Pompe disease treatment market?
Who are the key players/companies in the global Pompe disease treatment market?


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

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