Global Pompe Disease Treatment Market Report and Forecast 2023-2031

Global Pompe Disease Treatment Market Report and Forecast 2023-2031

Global Pompe Disease Treatment Market Outlook

The global Pompe disease treatment market size was valued at USD 1.14 billion in 2022 and is projected to grow at a CAGR of 4.8% during the forecast period of 2023-2031 to reach a value of USD 1.74 billion by 2031. The market growth can be attributed to the increasing prevalence of Pompe disease, advancements in treatment methods, and rising awareness about the condition.

Global Pompe Disease Treatment Market: Introduction

Pompe disease, also known as glycogen storage disease type II, is a rare genetic disorder caused by the deficiency of the acid alpha-glucosidase (GAA) enzyme. This deficiency leads to the accumulation of glycogen in various organs, particularly muscles, causing progressive muscle weakness and respiratory issues. The disease has two forms: infantile-onset and late-onset Pompe disease. The increasing prevalence of Pompe disease and the growing demand for innovative treatment options are expected to drive market growth.

Advancements in treatment methods, such as enzyme replacement therapy (ERT) and gene therapy, have played a significant role in the growth of the Pompe disease treatment market. Improved diagnostic techniques and the development of newborn screening programs have also contributed to the market's growth by increasing the identification and early intervention of Pompe disease cases. Furthermore, increasing investments in research and development, along with collaborations between pharmaceutical companies and research institutes, are expected to boost the market in the coming years.

Pompe Disease Epidemiology

Pompe disease is a rare genetic disorder, affecting approximately 1 in every 40,000 live births. The prevalence of the disease varies across different regions and ethnic groups, with the highest prevalence reported in African American and Chinese populations. The increasing awareness and understanding of the disease, along with advancements in diagnostic techniques, have led to better identification and management of the condition.

Global Pompe Disease Treatment Market Segmentations

The market can be segmented based on indication type, diagnosis methods, therapy type, route of administration, end-user, and region:

Market Breakup by Indication Type

  • Infantile-Onset Pompe Disease (IOPD)
  • Classic Infantile Form
  • Non-Classic Infantile Form
  • Late-Onset Pompe Disease (LOPD)
  • Others
Market Breakup by Diagnosis Methods
  • Blood Tests
  • Genetic Tests
  • Prenatal Tests
  • Others
Market Breakup by Therapy Type
  • Enzyme Replacement Therapy (ERT)
  • Chaperone-Advanced Replacement Therapy (CART)
  • Substrate Replacement Therapy (SRT)
  • Supportive Care
  • Others
Market Breakup by Route of Administration
  • Oral
  • Intravenous
  • Others
Market Breakup by End User
  • Hospitals
  • Diagnostic Centres
  • Ambulatory Surgical Centres
  • Others
Market Breakup by Region
  • North America
  • United States of America
  • Canada
  • Europe
  • United Kingdom
  • Germany
  • France
  • Italy
  • Others
  • Asia Pacific
  • China
  • Japan
  • India
  • ASEAN
  • Australia
  • Others
  • Latin America
  • Brazil
  • Argentina
  • Mexico
  • Others
  • Middle East and Africa
  • Saudi Arabia
  • United Arab Emirates
  • Nigeria
  • South Africa
  • Others
Pompe Disease Treatment Market Analysis

The global Pompe disease treatment market has experienced significant growth over the past few years, driven by factors such as increasing prevalence of the disease, advancements in treatment methods, and rising awareness about the condition. North America is currently the largest market for Pompe disease treatment, accounting for a significant share of the global market. The region's large market share can be attributed to factors such as a well-established healthcare infrastructure, strong investment in research and development, and the presence of key market players. The region's market growth is driven by factors such as a well-established healthcare system, increased awareness of Pompe disease, and government initiatives to support research and development for rare diseases.

Additionally, the European Medicines Agency (EMA) has implemented several regulatory incentives to encourage the development of orphan drugs for rare diseases, further fueling the growth of the Pompe disease treatment market in the region.

Asia Pacific is another region that is experiencing significant growth in the Pompe disease treatment market. The region's market growth is driven by factors such as a large population base, increasing awareness of rare diseases, and the growing adoption of advanced diagnostic and treatment technologies. In addition, government initiatives to address rare diseases and improve access to treatment are helping to drive the growth of the market in the region.

Key Players in the Global Pompe Disease Treatment Market

The report provides a detailed analysis of the key players involved in the Pompe disease treatment market, including their business overview, product portfolio, recent developments, and financial analysis. Some of the major players operating in the market include:
  • Amicus Therapeutics, Inc.
  • BioMarin
  • Genzyme (Sanofi)
  • Audentes Therapeutics (Astellas Pharma Inc.)
  • EpiVax, Inc
  • Oxyrane
  • Sangamo Therapeutics
  • Bayer AG
  • AVROBIO, Inc.
  • Nova Biomedical
  • Trinity Biotech

*Please Note:* The report will be completed in 7 business days after order confirmation


1 Preface
1.1 Objectives of the Study
1.2 Key Assumptions
1.3 Report Coverage – Key Segmentation and Scope
2 Research Methodology
3 Executive Summary
4 Pompe Disease Overview
4.1 Guidelines and Stages
4.2 Pathophysiology
4.3 Screening and Diagnosis
4.4 Treatment Pathway
5 Patient Profile
5.1 Patient Profile Overview
5.2 Patient Psychology and Emotional Impact Factors
5.3 Risk Assessment and Treatment Success Rate
6 Current Scenario Evaluation and Regulatory Framework
6.1 Emerging Therapies and Clinical Trials
6.2 Patent Landscape
6.2.1 Patent Overview
6.2.1.1 Patent Status and Expiry
6.2.1.2 Timelines from Drug Development to Commercial Launch
6.2.1.3 New Drug Application
6.2.1.3.1 Documentation and Approval Process
6.3 Cost of Treatment
6.4 Regulatory Framework
6.4.1 Regulatory Overview
6.4.1.1 US FDA
6.4.1.2 EU EMA
6.4.1.3 INDIA CDSCO
6.4.1.4 JAPAN PMDA
6.4.1.5 Others
7 Challenges and Unmet Needs
7.1 Treatment Pathway Challenges
7.2 Compliance and Drop-Out Analysis
7.3 Awareness and Prevention Gaps
8 Global Pompe Disease Treatment Market
8.1 Global Pompe Disease Treatment Market Overview
8.2 Global Pompe Disease Treatment Market Analysis
8.2.1 Market Overview
8.2.1.1 Global Pompe Disease Treatment Market Historical Value (2016-2022)
8.2.1.2 Global Pompe Disease Treatment Market Forecast Value (2023-2031)
8.2.2 Global Pompe Disease Treatment Market by Indication Type
8.2.2.1 Market Overview
8.2.2.1.1 Infantile-Onset Pompe Disease (IOPD)
8.2.2.1.1.1 Classic Infantile Form
8.2.2.1.1.2 Non-Classic Infantile Form
8.2.2.1.2 Late-Onset Pompe Disease (LOPD)
8.2.2.1.3 Others
8.2.3 Global Pompe Disease Treatment Market by Diagnosis Type
8.2.3.1 Market Overview
8.2.3.1.1 Blood Tests
8.2.3.1.2 Genetic Tests
8.2.3.1.3 Prenatal Tests
8.2.3.1.4 Others
8.2.4 Global Pompe Disease Treatment Market by Therapy Type
8.2.4.1 Market Overview
8.2.4.1.1 Enzyme Replacement Therapy (ERT)
8.2.4.1.2 Chaperone-Advanced Replacement Therapy (CART)
8.2.4.1.3 Substrate Replacement Therapy (SRT)
8.2.4.1.4 Supportive Care
8.2.4.1.5 Others
8.2.5 Global Pompe Disease Treatment Market by Route of Administration
8.2.5.1 Market Overview
8.2.5.1.1 Oral
8.2.5.1.2 Intravenous
8.2.5.1.3 Others
8.2.6 Global Pompe Disease Treatment Market by End User
8.2.6.1 Market Overview
8.2.6.1.1 Hospitals
8.2.6.1.2 Diagnostic Centres
8.2.6.1.3 Ambulatory Surgical Centres
8.2.6.1.4 Others
8.2.7 Global Pompe Disease Treatment Market by Region
8.2.7.1 Market Overview
8.2.7.1.1 North America
8.2.7.1.2 Europe
8.2.7.1.3 Asia Pacific
8.2.7.1.4 Middle East and Africa
8.2.7.1.5 Latin America
9 North America Pompe Disease Treatment Market
9.1 Market Share by Country
9.2 United States of America
9.2.1 Historical Trend (2016-2022)
9.2.2 Forecast Trend (2023-2031)
9.3 Canada
9.3.1 Historical Trend (2016-2022)
9.3.2 Forecast Trend (2023-2031)
10 Europe Pompe Disease Treatment Market
10.1 Market Share by Country
10.2 United Kingdom
10.2.1 Historical Trend (2016-2022)
10.2.2 Forecast Trend (2023-2031)
10.3 Germany
10.3.1 Historical Trend (2016-2022)
10.3.2 Forecast Trend (2023-2031)
10.4 France
10.4.1 Historical Trend (2016-2022)
10.4.2 Forecast Trend (2023-2031)
10.5 Italy
10.5.1 Historical Trend (2016-2022)
10.5.2 Forecast Trend (2023-2031)
10.6 Others
11 Asia Pacific Pompe Disease Treatment Market
11.1 Market Share by Country
11.2 China
11.2.1 Historical Trend (2016-2022)
11.2.2 Forecast Trend (2023-2031)
11.3 Japan
11.3.1 Historical Trend (2016-2022)
11.3.2 Forecast Trend (2023-2031)
11.4 India
11.4.1 Historical Trend (2016-2022)
11.4.2 Forecast Trend (2023-2031)
11.5 ASEAN
11.5.1 Historical Trend (2016-2022)
11.5.2 Forecast Trend (2023-2031)
11.6 Australia
11.6.1 Historical Trend (2016-2022)
11.6.2 Forecast Trend (2023-2031)
11.7 Others
12 Latin America Pompe Disease Treatment Market
12.1 Market Share by Country
12.2 Brazil
12.2.1 Historical Trend (2016-2022)
12.2.2 Forecast Trend (2023-2031)
12.3 Argentina
12.3.1 Historical Trend (2016-2022)
12.3.2 Forecast Trend (2023-2031)
12.4 Mexico
12.4.1 Historical Trend (2016-2022)
12.4.2 Forecast Trend (2023-2031)
12.5 Others
13 Middle East and Africa Pompe Disease Treatment Market
13.1 Market Share by Country
13.2 Saudi Arabia
13.2.1 Historical Trend (2016-2022)
13.2.2 Forecast Trend (2023-2031)
13.3 United Arab Emirates
13.3.1 Historical Trend (2016-2022)
13.3.2 Forecast Trend (2023-2031)
13.4 Nigeria
13.4.1 Historical Trend (2016-2022)
13.4.2 Forecast Trend (2023-2031)
13.5 South Africa
13.5.1 Historical Trend (2016-2022)
13.5.2 Forecast Trend (2023-2031)
13.6 Others
14 Global Pompe Disease Treatment Market Dynamics
14.1 Market Drivers and Constraints
14.2 SWOT Analysis
14.3 Porter’s Five Forces Model
14.4 Key Demand Indicators
14.5 Key Price Indicators
14.6 Industry Events, Initiatives, and Trends
14.7 Value Chain Analysis
15 Supplier Landscape
15.1 Amicus Therapeutics, Inc.
15.1.1 Company Overview
15.1.2 Product Portfolio
15.1.3 Demographic Reach and Achievements
15.1.4 Mergers and Acquisitions
15.1.5 Certifications
15.2 BioMarin
15.2.1 Company Overview
15.2.2 Product Portfolio
15.2.3 Demographic Reach and Achievements
15.2.4 Mergers and Acquisitions
15.2.5 Certifications
15.3 Genzyme (Sanofi)
15.3.1 Company Overview
15.3.2 Product Portfolio
15.3.3 Demographic Reach and Achievements
15.3.4 Mergers and Acquisitions
15.3.5 Certifications
15.4 Audentes Therapeutics (Astellas Pharma Inc.)
15.4.1 Company Overview
15.4.2 Product Portfolio
15.4.3 Demographic Reach and Achievements
15.4.4 Mergers and Acquisitions
15.4.5 Certifications
15.5 EpiVax, Inc
15.5.1 Company Overview
15.5.2 Product Portfolio
15.5.3 Demographic Reach and Achievements
15.5.4 Mergers and Acquisitions
15.5.5 Certifications
15.6 Oxyrane
15.6.1 Company Overview
15.6.2 Product Portfolio
15.6.3 Demographic Reach and Achievements
15.6.4 Mergers and Acquisitions
15.6.5 Certifications
15.7 Sangamo Therapeutics
15.7.1 Company Overview
15.7.2 Product Portfolio
15.7.3 Demographic Reach and Achievements
15.7.4 Mergers and Acquisitions
15.7.5 Certifications
15.8 Bayer AG
15.8.1 Company Overview
15.8.2 Product Portfolio
15.8.3 Demographic Reach and Achievements
15.8.4 Mergers and Acquisitions
15.8.5 Certifications
15.9 AVROBIO, Inc.
15.9.1 Company Overview
15.9.2 Product Portfolio
15.9.3 Demographic Reach and Achievements
15.9.4 Mergers and Acquisitions
15.9.5 Certifications
15.10 Nova Biomedical
15.10.1 Company Overview
15.10.2 Product Portfolio
15.10.3 Demographic Reach and Achievements
15.10.4 Mergers and Acquisitions
15.10.5 Certifications
15.11 Trinity Biotech
15.11.1 Company Overview
15.11.2 Product Portfolio
15.11.3 Demographic Reach and Achievements
15.11.4 Mergers and Acquisitions
15.11.5 Certifications
16 Global Pompe Disease Treatment Market Distribution Model (Additional Insight)
16.1 Overview
16.2 Potential Distributors
16.3 Key Parameters for Distribution Partner Assessment
17 Payment Methods (Additional Insight)
17.1 Government Funded
17.2 Private Insurance
17.3 Out-of-Pocket

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