Pompe Disease Market - Forecast(2024 - 2030)

Report Code: HCR 0948 Report Format: PDF + Excel
Pompe Disease Market Overview

Pompe Disease Market size is $1,277Bn in 2019, growing at a CAGR of 3.7% during the forecast period 2020-2025. Pompe disease is also known as GAA deficiency or type II glycogen storage disease (GSD). It is a genetic disorder in which complex sugar called glycogen builds up metabolism in the body’s cells. The disease results from the deficiency of an enzyme called Acid Alfa Glucosidase (GAA), which breaks downs complex sugars in the body. This buildup occurs in organs and tissues, especially in muscles, causing them to break down. The additional glycogen builds up in the patient and is stored in skeletal muscle, heart and other tissues, which causes the progressive indications of pompe disease. It causes muscle weakness and trouble breathing affecting the liver, heart (Cardiomegaly), and muscles that can lead to premature death in the new-born.
Report Coverage
The report: “Pompe Disease Market – Forecast (2020-2025)”, by IndustryARC covers an in-depth analysis of the following segments of the Pompe Disease Market. 

By Type: Classic Infantile-Onset, Non-Classic Infantile-Onset and Late Onset
By Therapy Type: Enzyme Replacement Therapy (ERT), Substrate Reduction Therapy (SRT), Gene Therapy and Others
By Form: Liquid and Solid
By End User: Hospitals/Clinics, Pharmaceuticals, Researchers and Others
By Geography: North America, Europe, Asia-Pacific and Rest of the World

Key Takeaways
  • Increased research in the field of Pompe Disease is set to propel the growth of the market. 
  • Increasing developments in the gene therapy and enzyme replacement therapies are driving factors for the growth of Pompe disease market.
  • Europe region is estimated to record the fastest growth rate during the forecast period 2020-2025.

By Therapy Type - Segment Analysis

In 2019, Enzyme Replacement Therapy (ERT) segment dominated the Pompe Disease Market in terms of revenue is estimated to grow at a CAGR of 3.0%. Enzyme Replacement Therapy is used to increase the levels of GAA in the body and reduce the accumulation of glycogen inside cells. ERT is administered into the bloodstream and has been shown to reduce the abnormal thickening of the heart walls that is characteristic of Pompe disease. It also helps maintain muscle function and improve patients’ quality of life. Lumizyme (alglucosidase alfa) is an FDA approved drug for Enzyme Replacement Therapy abetting towards the market’s growth.

Geography - Segment Analysis

In 2019, the North America region dominated Pompe Disease Market in terms of revenue with a market share of 39% owing towards owing to the presence of established payers and an increase in the number of Pompe disease patients in the region. This growth can be attributed towards the increasing research & development initiatives and government support for the improvement of the healthcare sector. Government has taken initiatives to promote the healthcare expenditure, growing awareness among patients regarding rare disorders in this region are key factors in the growth of the Pompe Disease market. Europe is estimated to record the fastest growth rate during the forecast period 2020-2025. 

Pompe Disease Market Drivers

Increase In The Prevalence of Pompe Disease 

According to the National Organization for Rare Disorders, the estimated frequency occurrence of Pompe disease is approximately 1 in 40,000 people in the US. The increase in the prevalence of pompe disease is driving the Pompe Disease Market owing towards the increase of manufacturers focusing on expanding their pipeline to cater to the demand for Pompe disease treatment. Governments have taken initiatives for developing and manufacturing innovative products for the diagnosis & treatment of Pompe disease is the other major factor driving growth of the Pompe Disease market. 

Pompe Disease Market Challenges

Lack Of Cost-Effective Treatment

The lack of cost effective treatments is restraining the growth of the market. The treatment is very expensive owing to the lack of FDA approved drugs which leads to the less affordability rate. The scarcity of low cost treatment hampers the growth of the market owing towards the patients being uncured.

Pompe Disease Industry Outlook 

Product Launches was the key strategy of the players in the Pompe Disease Industry. Pompe Disease top 10 companies include Amicus Therapeutics, Inc., Sanofi S.A., Audentes Therapeutics, Oxyrane , Valerion Therapeutics , AVROBIO, Inc., CENTOGENE AG, Sangamo BioSciences, BioMarin Pharmaceutical and Sanofi Genzyme.

Acquisitions/Product Launches

  • In January 2020, Sanofi S.A. acquired Synthorx, Inc. This acquisition helped the company in increasing the production for genetic disorders, oncology and immunological therapy research.
  • In April 2019, Audentes Therapeutics collaborated AAV Technology Platform and Pipeline with New Development Programs for Duchenne Muscular Dystrophy and Myotonic Dystrophy. 
1. Pompe Disease Market Overview
    1.1 Definitions and Scope
2. Pompe Disease Market - Executive Summary
    2.1 Market Revenue and Key Trends by Company
    2.2 Key trends by Type
    2.3 Key trends by Therapy Type
    2.4 Key trends by Geography
3. Pompe Disease Market – Comparative Analysis
    3.1 Product Benchmarking – Key Companies
    3.2 Financial Analysis – Key Companies
    3.3 Market Value Split by Key Companies
    3.4 Patent Analysis – Key Companies
    3.5 Pricing Analysis
4. Pompe Disease Market - Startup Companies Scenario (Premium)
    4.1 Key Startup Company Analysis by
        4.1.1 Investment
        4.1.2 Revenue
        4.1.3 Venture Capital and Funding Scenario
5. Pompe Disease Market – Industry Market Entry Scenario Premium (Premium)
    5.1 Regulatory Framework Overview
    5.2 New Business and Ease of Doing business index
    5.3 Case Studies of Successful Ventures
6. Pompe Disease Market Forces
    6.1 Market Drivers
    6.2 Market Constraints/Challenges
    6.3 Porters Five Force Model
        6.3.1 Bargaining power of suppliers
        6.3.2 Bargaining powers of customers
        6.3.3 Threat of new entrants
        6.3.4 Rivalry among existing players
        6.3.5 Threat of substitutes 
7. Pompe Disease Market – Strategic Analysis
    7.1 Value Chain Analysis
    7.2 Opportunities Analysis
    7.3 Market Life Cycle
8. Pompe Disease Market – By Type (Market Size –$Million/$Billion)
    8.1 Classic Infantile-Onset 
    8.2 Non-Classic Infantile-Onset 
    8.3 Late-Onset
9. Pompe Disease Market – By Therapy Type (Market Size –$Million/$Billion)
    9.1 Enzyme Replacement Therapy(ERT)
    9.2 Substrate Reduction Therapy(SRT)
    9.3 Gene Therapy
    9.4 Others
10. Pompe Disease Market – By Form (Market Size –$Million/$Billion)
    10.1 Liquid
    10.2 Solid
11. Pompe Disease Market – By End User (Market Size –$Million/$Billion)
    11.1 Hospitals/Clinics
    11.2 Pharmaceuticals 
    11.3 Researchers
    11.4 Others
12. Pompe Disease Market - By Geography (Market Size –$Million/$Billion)
    12.1 North America
        12.1.1 U.S.
        12.1.2 Canada
        12.1.3 Mexico
    12.2 Europe
        12.2.1 U.K.
        12.2.2 Germany
        12.2.3 France
        12.2.4 Italy
        12.2.5 Spain
        12.2.6 Rest of Europe
    12.3 Asia-Pacific
        12.3.1 China
        12.3.2 India
        12.3.3 Japan
        12.3.4 South Korea
        12.3.5 Australia & New Zealand
        12.3.6 Rest of Asia-Pacific
    12.4 Rest of the World
        12.4.1 Middle East
        12.4.2 Africa
        12.4.3 South America
13. Pompe Disease Market - Entropy
14. Pompe Disease Market – Industry/Segment Competition Landscape  (Premium) 
    14.1 Market Share Analysis
        14.1.1 Global Market Share – Key Companies
        14.1.2 Market Share by Region – Key companies
        14.1.3 Market Share by Countries – Key Companies
        14.1.4 Best Practices for Companies
15. Pompe Disease Market – Key Company List by Country Premium (Premium)
16. Pompe Disease Market Company Analysis
    16.1 Company 1
    16.2 Company 2
    16.3 Company 3
    16.4 Company 4
    16.5 Company 5
    16.6 Company 6
    16.7 Company 7
    16.8 Company 8
    16.9 Company 9
    16.10 Company 10 and more
"*Financials for private companies would be provided on a best efforts basis”.