Summary

Pompe disease is a genetic disorder characterized by the accumulation of a complex sugar known as glycogen within the cells of the body. This condition arises due to a deficiency in the enzyme acid alfa glucosidase (GAA), responsible for breaking down complex carbohydrates in the body.

Pompe disease manifests in several forms, including the classic infantile form, non-classic infantile form, late-onset form, and others. The classic infantile form is the most widely recognized, presenting significant heart dysfunction and noticeable hypotonia within a few months of birth. Diagnosis involves various methods such as blood tests, genetic tests, prenatal tests, and others. Treatment options for Pompe disease encompass oral, parenteral, and alternative approaches. Therapies include enzyme replacement therapy (ERT), substrate reduction therapy (SRT), gene therapy, and other modalities utilized by hospitals, clinics, pharmaceuticals, researchers, and other stakeholders.

The pompe disease market research report is one of a series of new reports from The Business Research Company that provides pompe disease market statistics, including pompe disease industry global market size, regional shares, competitors with a pompe disease market share, detailed pompe disease market segments, market trends and opportunities, and any further data you may need to thrive in the pompe disease industry. This pompe disease market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.

The pompe disease market size has grown steadily in recent years. It will grow from $1.66 billion in 2023 to $1.73 billion in 2024 at a compound annual growth rate (CAGR) of 4.2%. The expansion observed in the historical period can be ascribed to heightened awareness and diagnosis, the designation of orphan drugs and associated incentives, worldwide collaborations and research initiatives, as well as the active involvement of patient advocacy and support groups.

The pompe disease market size is expected to see strong growth in the next few years. It will grow to $2.13 billion in 2028 at a compound annual growth rate (CAGR) of 5.3%. The anticipated growth in the forecast period can be credited to ongoing research and development, the extension of newborn screening programs, progress in gene therapy, a rise in global investment in rare diseases, a focus on patient-centric drug development, the enlargement of clinical trials and research initiatives, and an emphasis on personalized medicine approaches within the market. Key trends expected in the forecast period encompass product innovation, technological advancements, the execution of patient support programs, an increasing adoption of telemedicine for patient management, and collaborative efforts for expanded access programs.

The rising prevalence of Pompe disease is anticipated to drive the growth of the Pompe disease market. Also known as glycogen storage disease type II or acid maltase deficiency, this rare genetic disorder is characterized by the accumulation of glycogen, a complex sugar molecule, in various body tissues, particularly in muscles and organs. Treatments for Pompe disease include protein replacement therapy, alglucosidase alfa, and Nexviazyme for intravenous infusion. The increase in Pompe disease cases, such as those reported by the European Medicines Agency Highlights in June 2022, is expected to contribute to the expansion of the Pompe disease market. In Europe alone, there are approximately 224 infantile-onset, 471 juvenile-onset, and 20,490 adult-onset individuals with mutations potentially causing Pompe disease.

The Pompe disease market is also experiencing significant growth due to the escalating healthcare expenditure. Governments and insurance providers increasingly recognize the importance of ensuring access to innovative therapies. According to data from the Centers for Medicare & Medicaid Services (CMS) in June 2023, the national health expenditure (NHE) in the United States reached $4.3 trillion in 2021, growing by 2.7% and accounting for 18.3% of the Gross Domestic Product (GDP). This rise in healthcare expenditure is expected to drive the Pompe disease market forward.

Product innovations are a prominent trend in the Pompe disease market, with major companies adopting innovations to maintain their market position. For example, in September 2023, Amicus Therapeutics received approval from the U.S. Food and Drug Administration (FDA) for Pombiliti and Opfolda, a two-component therapy designed for adults with late-onset Pompe disease. The therapy combines a recombinant human GAA enzyme (rhGAA) for increased uptake into muscle cells with an enzyme stabilizer (Opfolda) to stabilize the enzyme in the blood. This FDA approval highlights the importance of advancements in science and medicine.

Major companies in the Pompe disease market are focused on developing therapies such as enzyme replacement therapy (ERT) for the treatment of late-onset Pompe disease, addressing severe symptoms such as respiratory disorders and muscle failure. For instance, in August 2021, Sanofi received approval from the European Commission for Nexviazyme, an enzyme replacement treatment (ERT) targeting the mannose-6-phosphate (M6P) receptor, a primary mechanism for cellular uptake in Pompe disease. Clinical trials have shown that Nexviazyme increases respiratory function and walking distance in patients.

In January 2022, Hansa Biopharma AB and Asklepios BioPharmaceutical Inc. collaborated to assess the potential use of imlifidase as a pre-treatment before administering AskBio’s gene therapy for Pompe disease. This partnership involves a pre-clinical and clinical feasibility program for patients with pre-existing neutralizing antibodies to adeno-associated virus (AAV). Hansa will receive a $5 million payment, and AskBio holds an exclusive option for a full development and commercialization agreement based on the results of an initial Phase I/II study. AskBio will lead the pre-clinical and clinical studies, while Hansa will provide materials and additional support outlined in the feasibility program.

Major companies operating in the pompe disease market report are F. Hoffmann-La Roche AG, Bayer AG, Novartis AG, Sanofi S.A., Thermo Fisher Scientific Inc., Fresenius Kabi AG, Danaher Corporation, Becton, Dickinson and Company, Astellas Pharma Inc., BioMérieux SA, BioMarin Pharmaceutical Inc., Nova Biomedical Corporation, Instrumentation Laboratory Company, Amicus Therapeutics Inc., Sekisui Diagnostics LLC, Spark Therapeutics Inc., Sangamo BioSciences Inc., Asklepios BioPharmaceutical Inc., Avrobio Inc., EKF Diagnostics Holdings PLC, Trinity Biotech PLC, Chembio Diagnostics Inc., Audentes Therapeutics Inc., Rougine Darou, Genethon, Maze Therapeutics Inc., Avidity Biosciences Inc., EpiVax Inc., M6P Therapeutics, Oxyrane UK Limited, Valerion Therapeutics LLC

North America was the largest region in the pompe disease market in 2023. The regions covered in the pompe disease market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa.

The countries covered in the pompe disease market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

The pompe disease market includes revenues earned by entities by lumizyme, alglucosidase alfa, nexviazyme, and aval glucosidase alfa. The market value includes the value of related goods sold by the service provider or included within the service offering. Only goods and services traded between entities or sold to end consumers are included.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

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Table of Contents

1. Executive Summary
2. Pompe Disease Market Characteristics
3. Pompe Disease Market Trends And Strategies
4. Pompe Disease Market - Macro Economic Scenario
4.1. Impact Of High Inflation On The Market
4.2. Ukraine-Russia War Impact On The Market
4.3. COVID-19 Impact On The Market
5. Global Pompe Disease Market Size and Growth
5.1. Global Pompe Disease Market Drivers and Restraints
5.1.1. Drivers Of The Market
5.1.2. Restraints Of The Market
5.2. Global Pompe Disease Historic Market Size and Growth, 2018 - 2023, Value ($ Billion)
5.3. Global Pompe Disease Forecast Market Size and Growth, 2023 - 2028, 2033F, Value ($ Billion)
6. Pompe Disease Market Segmentation
6.1. Global Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
Classic Infantile Form
Non-Classic Infantile Form
Late-Onset Form
Other Types
6.2. Global Pompe Disease Market, Segmentation By Diagnosis, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
Blood Test
Genetic Test
Prenatal Test
Other Diagnoses
6.3. Global Pompe Disease Market, Segmentation By Routes of Administration, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
Oral
Parenteral
Other routes of administration
6.4. Global Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
Enzyme Replacement Therapy (ERT)
Substrate Reduction Therapy (SRT)
Gene Therapy
Other Therapies
6.5. Global Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
Hospitals or Clinics
Pharmaceuticals
Researchers
Other End-Users
7. Pompe Disease Market Regional And Country Analysis
7.1. Global Pompe Disease Market, Split By Region, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
7.2. Global Pompe Disease Market, Split By Country, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
8. Asia-Pacific Pompe Disease Market
8.1. Asia-Pacific Pompe Disease Market Overview
Region Information, Impact Of COVID-19, Market Information, Background Information, Government Initiatives, Regulations, Regulatory Bodies, Major Associations, Taxes Levied, Corporate Tax Structure, Investments, Major Companies
8.2. Asia-Pacific Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
8.3. Asia-Pacific Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
8.4. Asia-Pacific Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
9. China Pompe Disease Market
9.1. China Pompe Disease Market Overview
9.2. China Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F,$ Billion
9.3. China Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F,$ Billion
9.4. China Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F,$ Billion
10. India Pompe Disease Market
10.1. India Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
10.2. India Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
10.3. India Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
11. Japan Pompe Disease Market
11.1. Japan Pompe Disease Market Overview
11.2. Japan Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
11.3. Japan Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
11.4. Japan Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
12. Australia Pompe Disease Market
12.1. Australia Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
12.2. Australia Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
12.3. Australia Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
13. Indonesia Pompe Disease Market
13.1. Indonesia Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
13.2. Indonesia Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
13.3. Indonesia Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
14. South Korea Pompe Disease Market
14.1. South Korea Pompe Disease Market Overview
14.2. South Korea Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
14.3. South Korea Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
14.4. South Korea Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
15. Western Europe Pompe Disease Market
15.1. Western Europe Pompe Disease Market Overview
15.2. Western Europe Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
15.3. Western Europe Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
15.4. Western Europe Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
16. UK Pompe Disease Market
16.1. UK Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
16.2. UK Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
16.3. UK Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
17. Germany Pompe Disease Market
17.1. Germany Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
17.2. Germany Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
17.3. Germany Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
18. France Pompe Disease Market
18.1. France Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
18.2. France Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
18.3. France Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
19. Italy Pompe Disease Market
19.1. Italy Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
19.2. Italy Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
19.3. Italy Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
20. Spain Pompe Disease Market
20.1. Spain Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
20.2. Spain Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
20.3. Spain Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
21. Eastern Europe Pompe Disease Market
21.1. Eastern Europe Pompe Disease Market Overview
21.2. Eastern Europe Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
21.3. Eastern Europe Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
21.4. Eastern Europe Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
22. Russia Pompe Disease Market
22.1. Russia Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
22.2. Russia Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
22.3. Russia Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
23. North America Pompe Disease Market
23.1. North America Pompe Disease Market Overview
23.2. North America Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
23.3. North America Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
23.4. North America Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
24. USA Pompe Disease Market
24.1. USA Pompe Disease Market Overview
24.2. USA Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
24.3. USA Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
24.4. USA Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
25. Canada Pompe Disease Market
25.1. Canada Pompe Disease Market Overview
25.2. Canada Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
25.3. Canada Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
25.4. Canada Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
26. South America Pompe Disease Market
26.1. South America Pompe Disease Market Overview
26.2. South America Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
26.3. South America Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
26.4. South America Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
27. Brazil Pompe Disease Market
27.1. Brazil Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
27.2. Brazil Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
27.3. Brazil Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
28. Middle East Pompe Disease Market
28.1. Middle East Pompe Disease Market Overview
28.2. Middle East Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
28.3. Middle East Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
28.4. Middle East Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
29. Africa Pompe Disease Market
29.1. Africa Pompe Disease Market Overview
29.2. Africa Pompe Disease Market, Segmentation By Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
29.3. Africa Pompe Disease Market, Segmentation By Therapy Type, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
29.4. Africa Pompe Disease Market, Segmentation By End Users, Historic and Forecast, 2018-2023, 2023-2028F, 2033F, $ Billion
30. Pompe Disease Market Competitive Landscape And Company Profiles
30.1. Pompe Disease Market Competitive Landscape
30.2. Pompe Disease Market Company Profiles
30.2.1. F. Hoffmann-La Roche AG
30.2.1.1. Overview
30.2.1.2. Products and Services
30.2.1.3. Strategy
30.2.1.4. Financial Performance
30.2.2. Bayer AG
30.2.2.1. Overview
30.2.2.2. Products and Services
30.2.2.3. Strategy
30.2.2.4. Financial Performance
30.2.3. Novartis AG
30.2.3.1. Overview
30.2.3.2. Products and Services
30.2.3.3. Strategy
30.2.3.4. Financial Performance
30.2.4. Sanofi S.A.
30.2.4.1. Overview
30.2.4.2. Products and Services
30.2.4.3. Strategy
30.2.4.4. Financial Performance
30.2.5. Thermo Fisher Scientific Inc.
30.2.5.1. Overview
30.2.5.2. Products and Services
30.2.5.3. Strategy
30.2.5.4. Financial Performance
31. Pompe Disease Market Other Major And Innovative Companies
31.1. Fresenius Kabi AG
31.2. Danaher Corporation
31.3. Becton, Dickinson and Company
31.4. Astellas Pharma Inc.
31.5. BioMérieux SA
31.6. BioMarin Pharmaceutical Inc.
31.7. Nova Biomedical Corporation
31.8. Instrumentation Laboratory Company
31.9. Amicus Therapeutics Inc.
31.10. Sekisui Diagnostics LLC
31.11. Spark Therapeutics Inc.
31.12. Sangamo BioSciences Inc.
31.13. Asklepios BioPharmaceutical Inc.
31.14. Avrobio Inc.
31.15. EKF Diagnostics Holdings plc
32. Global Pompe Disease Market Competitive Benchmarking
33. Global Pompe Disease Market Competitive Dashboard
34. Key Mergers And Acquisitions In The Pompe Disease Market
35. Pompe Disease Market Future Outlook and Potential Analysis
35.1 Pompe Disease Market In 2028 - Countries Offering Most New Opportunities
35.2 Pompe Disease Market In 2028 - Segments Offering Most New Opportunities
35.3 Pompe Disease Market In 2028 - Growth Strategies
35.3.1 Market Trend Based Strategies
35.3.2 Competitor Strategies
36. Appendix
36.1. Abbreviations
36.2. Currencies
36.3. Historic And Forecast Inflation Rates
36.4. Research Inquiries
36.5. The Business Research Company
36.6. Copyright And Disclaimer