The pompe disease market size has grown steadily in recent years. It will grow from $1.74 billion in 2024 to $1.8 billion in 2025 at a compound annual growth rate (CAGR) of 3.6%. The growth in the historic period can be attributed to increased awareness and diagnosis, orphan drug designation and incentives, global collaborations and research initiatives, 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 $2.32 billion in 2029 at a compound annual growth rate (CAGR) of 6.5%. The growth in the forecast period can be attributed to continued research and development, expansion of newborn screening programs, advancements in gene therapy, increasing global investment in rare diseases, patient-centric drug development, expansion of clinical trials and research initiatives, market emphasis on personalized medicine approaches. Major trends in the forecast period include product innovation, technological advancements, implementation of patient support programs, growing adoption of telemedicine for patient management, and collaborations 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 increasing healthcare expenditure is playing a significant role in driving the growth of the Pompe disease market in the future. Governments and insurance providers are progressively acknowledging the importance of ensuring access to innovative therapies. For example, in May 2023, a report from the Office for National Statistics, a UK government department, revealed that healthcare spending in the UK rose by 5.6% from 2022 to 2023, compared to a growth of just 0.9% in 2022. In 2023, the UK's healthcare expenditure was approximately $317.63 billion (£292 billion). Thus, the rising healthcare expenditure is expected to propel 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 prioritizing strategic partnerships to enhance product development, broaden market reach, and improve solutions for animal health. A strategic partnership generally refers to a collaborative relationship between two or more organizations that combine their resources, expertise, and efforts to achieve shared goals or objectives. For example, in June 2024, Shionogi & Company, Limited, a Japanese pharmaceutical firm, entered into a global licensing agreement with Maze Therapeutics, a US-based biotechnology company, for MZE001, an investigational oral medication aimed at treating Pompe disease. Under this agreement, Shionogi secures exclusive rights to MZE001 and its associated intellectual property, which includes an initial payment of $150 million, along with potential milestone and royalty payments. This agreement was finalized following the necessary 30-day review period mandated by U.S. antitrust law (HSR Act).
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 include F. Hoffmann-La Roche AG, Sanofi S.A., Amicus Therapeutics Inc., Sangamo BioSciences Inc., Asklepios BioPharmaceutical Inc., Audentes Therapeutics Inc., Genethon, Maze Therapeutics Inc., Avidity Biosciences Inc., EpiVax Inc., M6P Therapeutics, Oxyrane UK Limited, Valerion Therapeutics LLC, Abeona Therapeutics Inc., BioMarin Pharmaceutical, Genzyme Corporation, Novartis AG, Ultragenyx Pharmaceutical, Aro Biotherapeutics Company, Akashi Therapeutics, Protalix BioTherapeutics, AVROBIO Inc., Catabasis Pharmaceuticals.
North America was the largest region in the pompe disease market in 2024. 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.
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 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 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.
This product will be delivered within 3-5 business days.
The pompe disease market size is expected to see strong growth in the next few years. It will grow to $2.32 billion in 2029 at a compound annual growth rate (CAGR) of 6.5%. The growth in the forecast period can be attributed to continued research and development, expansion of newborn screening programs, advancements in gene therapy, increasing global investment in rare diseases, patient-centric drug development, expansion of clinical trials and research initiatives, market emphasis on personalized medicine approaches. Major trends in the forecast period include product innovation, technological advancements, implementation of patient support programs, growing adoption of telemedicine for patient management, and collaborations 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 increasing healthcare expenditure is playing a significant role in driving the growth of the Pompe disease market in the future. Governments and insurance providers are progressively acknowledging the importance of ensuring access to innovative therapies. For example, in May 2023, a report from the Office for National Statistics, a UK government department, revealed that healthcare spending in the UK rose by 5.6% from 2022 to 2023, compared to a growth of just 0.9% in 2022. In 2023, the UK's healthcare expenditure was approximately $317.63 billion (£292 billion). Thus, the rising healthcare expenditure is expected to propel 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 prioritizing strategic partnerships to enhance product development, broaden market reach, and improve solutions for animal health. A strategic partnership generally refers to a collaborative relationship between two or more organizations that combine their resources, expertise, and efforts to achieve shared goals or objectives. For example, in June 2024, Shionogi & Company, Limited, a Japanese pharmaceutical firm, entered into a global licensing agreement with Maze Therapeutics, a US-based biotechnology company, for MZE001, an investigational oral medication aimed at treating Pompe disease. Under this agreement, Shionogi secures exclusive rights to MZE001 and its associated intellectual property, which includes an initial payment of $150 million, along with potential milestone and royalty payments. This agreement was finalized following the necessary 30-day review period mandated by U.S. antitrust law (HSR Act).
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 include F. Hoffmann-La Roche AG, Sanofi S.A., Amicus Therapeutics Inc., Sangamo BioSciences Inc., Asklepios BioPharmaceutical Inc., Audentes Therapeutics Inc., Genethon, Maze Therapeutics Inc., Avidity Biosciences Inc., EpiVax Inc., M6P Therapeutics, Oxyrane UK Limited, Valerion Therapeutics LLC, Abeona Therapeutics Inc., BioMarin Pharmaceutical, Genzyme Corporation, Novartis AG, Ultragenyx Pharmaceutical, Aro Biotherapeutics Company, Akashi Therapeutics, Protalix BioTherapeutics, AVROBIO Inc., Catabasis Pharmaceuticals.
North America was the largest region in the pompe disease market in 2024. 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.
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 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 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.
This product will be delivered within 3-5 business days.
Table of Contents
1. Executive Summary2. Pompe Disease Market Characteristics3. Pompe Disease Market Trends and Strategies4. Pompe Disease Market - Macro Economic Scenario including the impact of Interest Rates, Inflation, Geopolitics and Covid and Recovery on the Market32. Global Pompe Disease Market Competitive Benchmarking and Dashboard33. Key Mergers and Acquisitions in the Pompe Disease Market34. Recent Developments in the Pompe Disease Market
5. Global Pompe Disease Growth Analysis and Strategic Analysis Framework
6. Pompe Disease Market Segmentation
7. Pompe Disease Market Regional and Country Analysis
8. Asia-Pacific Pompe Disease Market
9. China Pompe Disease Market
10. India Pompe Disease Market
11. Japan Pompe Disease Market
12. Australia Pompe Disease Market
13. Indonesia Pompe Disease Market
14. South Korea Pompe Disease Market
15. Western Europe Pompe Disease Market
16. UK Pompe Disease Market
17. Germany Pompe Disease Market
18. France Pompe Disease Market
19. Italy Pompe Disease Market
20. Spain Pompe Disease Market
21. Eastern Europe Pompe Disease Market
22. Russia Pompe Disease Market
23. North America Pompe Disease Market
24. USA Pompe Disease Market
25. Canada Pompe Disease Market
26. South America Pompe Disease Market
27. Brazil Pompe Disease Market
28. Middle East Pompe Disease Market
29. Africa Pompe Disease Market
30. Pompe Disease Market Competitive Landscape and Company Profiles
31. Pompe Disease Market Other Major and Innovative Companies
35. Pompe Disease Market High Potential Countries, Segments and Strategies
36. Appendix
Executive Summary
Pompe Disease Global Market Report 2025 provides strategists, marketers and senior management with the critical information they need to assess the market.This report focuses on pompe disease market which is experiencing strong growth. The report gives a guide to the trends which will be shaping the market over the next ten years and beyond.
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Description
Where is the largest and fastest growing market for pompe disease? How does the market relate to the overall economy, demography and other similar markets? What forces will shape the market going forward? The pompe disease market global report answers all these questions and many more.The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market’s historic and forecast market growth by geography.
- The market characteristics section of the report defines and explains the market.
- The market size section gives the market size ($b) covering both the historic growth of the market, and forecasting its development.
- The forecasts are made after considering the major factors currently impacting the market. These include:
- The forecasts are made after considering the major factors currently impacting the market. These include the Russia-Ukraine war, rising inflation, higher interest rates, and the legacy of the COVID-19 pandemic.
- Market segmentations break down the market into sub markets.
- The regional and country breakdowns section gives an analysis of the market in each geography and the size of the market by geography and compares their historic and forecast growth. It covers the growth trajectory of COVID-19 for all regions, key developed countries and major emerging markets.
- The competitive landscape chapter gives a description of the competitive nature of the market, market shares, and a description of the leading companies. Key financial deals which have shaped the market in recent years are identified.
- The trends and strategies section analyses the shape of the market as it emerges from the crisis and suggests how companies can grow as the market recovers.
Scope
Markets Covered:
1) By Type: Classic Infantile Form, Non-Classic Infantile Form, Late-Onset Form, Other Types2) By Diagnosis: Blood Test, Genetic Test, Prenatal Test, Other Diagnosis
3) By Route of Administration: Oral, Parenteral, Other routes of administrations
4) By Therapy Type: Enzyme Replacement Therapy (ERT), Substrate Reduction Therapy (SRT), Gene Therapy, Other Therapies
5) By End Users: Hospitals or Clinics, Pharmaceuticals, Researchers, Other End-Users
Subsegments:
1) By Classic Infantile Form: Early-Onset Classic Pompe Disease; Severe Classic Pompe Disease2) By Non-Classic Infantile Form: Mild Non-Classic Infantile Pompe Disease; Variants of Non-Classic Infantile Form
3) By Late-Onset Form: Adult-Onset Pompe Disease; Juvenile-Onset Pompe Disease
4) By Other Types: Asymptomatic Carriers; Other Rare Variants of Pompe Disease
Key Companies Mentioned: F. Hoffmann-La Roche AG; Sanofi S.A.; Amicus Therapeutics Inc.; Sangamo BioSciences Inc.; Asklepios BioPharmaceutical Inc.
Countries: Australia; Brazil; China; France; Germany; India; Indonesia; Japan; Russia; South Korea; UK; USA; Canada; Italy; Spain
Regions: Asia-Pacific; Western Europe; Eastern Europe; North America; South America; Middle East; Africa
Time Series: Five years historic and ten years forecast.
Data: Ratios of market size and growth to related markets, GDP proportions, expenditure per capita.
Data Segmentation: Country and regional historic and forecast data, market share of competitors, market segments.
Sourcing and Referencing: Data and analysis throughout the report is sourced using end notes.
Delivery Format: PDF, Word and Excel Data Dashboard.
Companies Mentioned
- F. Hoffmann-La Roche AG
- Sanofi S.A.
- Amicus Therapeutics Inc.
- Sangamo BioSciences Inc.
- Asklepios BioPharmaceutical Inc.
- Audentes Therapeutics Inc.
- Genethon
- Maze Therapeutics Inc.
- Avidity Biosciences Inc.
- EpiVax Inc.
- M6P Therapeutics
- Oxyrane UK Limited
- Valerion Therapeutics LLC
- Abeona Therapeutics Inc.
- BioMarin Pharmaceutical
- Genzyme Corporation
- Novartis AG
- Ultragenyx Pharmaceutical
- Aro Biotherapeutics Company
- Akashi Therapeutics
- Protalix BioTherapeutics
- AVROBIO Inc.
- Catabasis Pharmaceuticals
Table Information
| Report Attribute | Details |
|---|---|
| No. of Pages | 200 |
| Published | February 2025 |
| Forecast Period | 2025 - 2029 |
| Estimated Market Value ( USD | $ 1.8 Billion |
| Forecasted Market Value ( USD | $ 2.32 Billion |
| Compound Annual Growth Rate | 6.5% |
| Regions Covered | Global |
| No. of Companies Mentioned | 23 |
