Global Pompe Disease Treatment market is estimated to reach $1.45 Billion by 2026; growing at a CAGR of 2.81% from 2018 to 2026.

Market Overview

Global Pompe Disease Treatment market is estimated to reach $1.45 Billion by 2026; growing at a CAGR of 2.81% from 2018 to 2026. Pompe disease is a rare inherited disorder characterized by the buildup of an unusual sugar molecule called glycogen in the bodyâ€™s cell. This buildup damages the working of different organs and tissues, especially skeletal muscles, heart, liver, and the respiratory system. The Pompe also is known as GAA deficiency or type II glycogen storage disease (GSD) is inherited in an autosomal recessive pattern. This means the disorder only grows in people who receive two faulty copies of the gene, one from each parent. The disease can cause to both males and females.

Market Determinants

The growing prevalence of Pompe disease, continuous uptake of long-term therapies, and developing healthcare infrastructure are the major factors driving the growth of the global Pompe disease treatment market. For instance, in the U.S., the prevalence of Pompe disease is approximately 1 person in 28,000, which was 1 person in 40,000 earlier. In addition, it can take about 3 months to diagnose Pompe disease in a baby and can take as long as 7 to 9 years for kids and adults. However, the high cost of these treatments and lack of awareness in the emerging economies may act as the hindering events for the market growth. Moreover, rising government initiatives and growth in the R&D activities in the gene therapy and enzyme replacement therapies is expected to generate numerous growth opportunities for the market in the coming years.

Market Segmentation

The global Pompe disease treatment market is mainly classified based on treatment, distribution channel, and route of administration. Treatment is further segmented into chaperone-advanced replacement therapy, substrate reduction therapy, enzyme replacement therapy, and others. Based on the distribution channel, the market is divided into retail pharmacies, hospitals and clinics pharmacies, online pharmacies, and others. By route of administration, the market is further categorized into intravenous, oral, and others.

Regional Segmentation

Based on geography, the global Pompe disease treatment market is divided into North America, Europe, Asia-Pacific, South America, and Middle East & Africa. North America is further divided in the U.S., Canada, and Mexico, whereas Europe consists of the UK, Germany, France, Italy, and Rest of Europe. Asia-Pacific is categorized into India, Japan, China, South Korea, and Rest of APAC. The South America region includes Brazil, Argentina, and the Rest of South America, while the Middle East & Africa is categorized into GCC Countries, Egypt, South Africa, and Rest of Middle East & Africa. Among all the regions, North America accounted for the majority of the share in the global market owing to a rising number of Pompe patients in the region especially in the U.S. Europe stands on the second number owing to the growing number of government initiatives, and a surge in the number of the R&D activities. However, Asia-Pacific is expected to grow with the fastest growth rate (CAGR), owing to the rising healthcare expenditure and improving healthcare infrastructure.

Competitive Players

Key players operating in the Pompe Disease Treatment industry include Amicus Therapeutics, Audentes Therapeutics, AVROBIO, Inc., CENTOGENE AG, EpiVax, Oxyrane, Sangamo BioSciences, Sanofi Genzyme, and Valerion Therapeutics, among others. For instance, April 30, 2019 - Amicus Therapeutics announced initial preclinical data from its new adeno-associated viral (AAV) gene therapy program for Pompe disease in mice. In addition, on March 20, 2019 â€“ Amicus announced that the U.S. Patent and Trademark Office has issued two new patents directed to the composition of the matter and methods of making ATB200. It is a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with enhanced carbohydrate structures, specifically mannose-6 phosphate (M6P), to improve uptake. On Feb 2017 â€“ Sanofi Genzyme announced additional data on avalglucosidase alfa in Pompe disease. The multiple studies of this investigational therapy were presented during the 15th Annual â€˜WORLDSymposiumâ€™ in Orlando, Florida. The study revealed Pompe disease affects an estimated 50,000 people worldwide. Moreover, it can occur at any age from infancy to late adulthood.

Pompe Disease Treatment Market Key Segments:

By Treatment

Chaperone-Advanced Replacement Therapy
Substrate Reduction Therapy
Enzyme Replacement Therapy
Others

By Distribution Channel

Retail Pharmacies
Hospitals and Clinics Pharmacies
Online Pharmacies
Others

By Route of Administration

Intravenous
Oral
Others

By Geography

North America
- The U.S.
- Canada
- Mexico
Europe
- U.K
- Germany
- France
- Italy
- Rest of Europe
Asia Pacific
- India
- China
- Japan
- South Korea
- Rest of Asia Pacific
South America
- Brazil
- Argentina
- Rest of South America
Middle East & Africa
- GCC Countries
- Egypt
- South Africa
- Rest of Middle East & Africa

Table of Content 1. Market Introduction 1.1. Executive Summary 1.2. Market Definition 1.3. Market Scope 2. Research Methodology 2.1. Primary Research 2.2. Research Methodology 2.3. Assumptions & Exclusions 2.4. Secondary data sources 3. Pompe Disease Treatment Market Overview 3.1. Report Segmentation & Scope 3.2. Key Market Trend 3.3. Drivers 3.3.1. Growing prevalence of Pompe disease 3.3.2. Continuous uptake of long-term therapies 3.3.3. Developing healthcare infrastructure 3.4. Restraints 3.4.1. High cost of these treatments 3.4.2. Lack of awareness in the emerging economies 3.5. Opportunities 3.5.1. Rising government initiatives 3.5.2. Growth in the R&D activities in the gene therapy and enzyme replacement therapies 3.6. Porter’s Five Forces Analysis 3.6.1. Porter’s Five Forces Analysis 3.7. Market Share Analysis 4. Treatment Overview 4.1. Introduction 4.1.1. Market Size & Forecast 4.2. Chaperone-Advanced Replacement Therapy 4.2.1. Market Size & Forecast 4.3. Substrate Reduction Therapy 4.3.1. Market Size & Forecast 4.4. Enzyme Replacement Therapy 4.4.1. Market Size & Forecast 4.5. Others 4.5.1. Market Size & Forecast 5. Distribution Channel Overview 5.1. Introduction 5.1.1. Market Size & Forecast 5.2. Retail Pharmacies 5.2.1. Market Size & Forecast 5.3. Hospitals and Clinics Pharmacies 5.3.1. Market Size & Forecast 5.4. Online Pharmacies 5.4.1. Market Size & Forecast 5.5. Others 5.5.1. Market Size & Forecast 6. Route of Administration Overview 6.1. Introduction 6.1.1. Market Size & Forecast 6.2. Intravenous 6.2.1. Market Size & Forecast 6.3. Oral 6.3.1. Market Size & Forecast 6.4. Others 6.4.1. Market Size & Forecast 7. Pompe Disease Treatment Market Regional Overview 7.1. Introduction 7.1.1. Market Size & Forecast 7.2. North America Pompe Disease Treatment Market 7.2.1. North America Market Size & Forecast, By Country 7.2.2. North America Market Size & Forecast, By Treatment 7.2.3. North America Market Size & Forecast, By Distribution Channel 7.2.4. North America Market Size & Forecast, By Route of Administration 7.2.5. U.S. 7.2.5.1. Market Size and Forecast 7.2.6. Canada 7.2.6.1. Market Size and Forecast 7.2.7. Mexico 7.2.7.1. Market Size and Forecast 7.3. Europe Pompe Disease Treatment Market 7.3.1. Europe Market Size & Forecast, By Country 7.3.2. Europe Market Size & Forecast, By Treatment 7.3.3. Europe Market Size & Forecast, By Distribution Channel 7.3.4. Europe Market Size & Forecast, By Route of Administration 7.3.5. Germany 7.3.5.1. Market Size and Forecast 7.3.6. France 7.3.6.1. Market Size and Forecast 7.3.7. UK 7.3.7.1. Market Size and Forecast 7.3.8. Italy 7.3.8.1. Market Size and Forecast 7.3.9. Spain 7.3.9.1. Market Size and Forecast 7.3.10. Rest of Europe 7.3.10.1. Market Size and Forecast 7.4. Asia-Pacific Pompe Disease Treatment Market 7.4.1. Asia-Pacific Market Size & Forecast, By Country 7.4.2. Asia-Pacific Market Size & Forecast, By Treatment 7.4.3. Asia-Pacific Market Size & Forecast, By Distribution Channel 7.4.4. Asia-Pacific Market Size & Forecast, By Route of Administration 7.4.5. Japan 7.4.5.1. Market Size and Forecast 7.4.6. China 7.4.6.1. Market Size and Forecast 7.4.7. Australia 7.4.7.1. Market Size and Forecast 7.4.8. India 7.4.8.1. Market Size and Forecast 7.4.9. South Korea 7.4.9.1. Market Size and Forecast 7.4.10. Rest of Asia-Pacific 7.4.10.1. Market Size and Forecast 7.5. South America Pompe Disease Treatment Market 7.5.1. South America Market Size & Forecast, By Country 7.5.2. South America Market Size & Forecast, By Treatment 7.5.3. South America Market Size & Forecast, By Distribution Channel 7.5.4. South America Market Size & Forecast, By Route of Administration 7.5.5. Brazil 7.5.5.1. Market Size and Forecast 7.5.6. Argentina 7.5.6.1. Market Size and Forecast 7.5.7. Rest of South America 7.5.7.1. Market Size and Forecast 7.6. Middle East & Africa Pompe Disease Treatment Market 7.6.1. Middle East & Africa Market Size & Forecast, By Country 7.6.2. Middle East & Africa Market Size & Forecast, By Treatment 7.6.3. Middle East & Africa Market Size & Forecast, By Distribution Channel 7.6.4. Middle East & Africa Market Size & Forecast, By Route of Administration 7.6.5. GCC Countries 7.6.5.1. Market Size and Forecast 7.6.6. Egypt 7.6.6.1. Market Size and Forecast 7.6.7. South Africa 7.6.7.1. Market Size and Forecast 7.6.8. Rest of Middle East & Africa 7.6.8.1. Market Size and Forecast 8. Company Profile 8.1. Amicus Therapeutics 8.1.1. Company Basic Information, Manufacturing Base, Sales Area and Its Competitors 8.1.2. Amicus Therapeutics Product Category, Application, and Specification 8.1.3. Amicus Therapeutics Financial Performance (2016-2018) 8.1.4. Main Business/Business Overview 8.2. Audentes Therapeutics 8.3. AVROBIO, Inc. 8.4. CENTOGENE AG 8.5. EpiVax 8.6. Oxyrane 8.7. Sangamo BioSciences 8.8. Sanofi Genzyme 8.9. Valerion Therapeutics

Key Global Pompe Disease Treatment Industry Players

Amicus Therapeutics
Audentes Therapeutics
AVROBIO, Inc.
CENTOGENE AG
EpiVax
Oxyrane
Sangamo BioSciences
Sanofi Genzyme
Valerion Therapeutics

Market Overview

Market Determinants

Market Segmentation

Regional Segmentation

Competitive Players

Pompe Disease Treatment Market Key Segments:

Report Highlights:

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