The mucopolysaccharidosis treatment market size has grown strongly in recent years. It will grow from $2.4 billion in 2024 to $2.59 billion in 2025 at a compound annual growth rate (CAGR) of 8%. The growth in the historic period can be attributed to enzyme replacement therapy (ERT), improved diagnostic tools, advocacy and awareness, gene therapy development, and healthcare infrastructure development.
The mucopolysaccharidosis treatment market size is expected to see strong growth in the next few years. It will grow to $3.51 billion in 2029 at a compound annual growth rate (CAGR) of 7.9%. The growth in the forecast period can be attributed to advancements in gene editing, regulatory changes and support, patient access initiatives, emerging therapeutic modalities, and precision medicine approaches. Major trends in the forecast period include caregiver support programs, focus on symptom management, education and training initiatives, expanded global access, and advances in biomarker development.
The rising prevalence of various forms of mucopolysaccharidosis is anticipated to boost the growth of the mucopolysaccharidosis treatment market in the future. Mucopolysaccharide is a type of complex carbohydrate that accumulates in body tissues due to the absence of the specific enzyme required for its breakdown. Mucopolysaccharidosis (MPS) therapy helps slow disease progression by reducing glycosaminoglycan (GAG) accumulation, managing symptoms, and preventing complications through various treatment approaches. For example, in July 2024, the Orphanet Journal of Rare Diseases, a peer-reviewed open-access medical journal based in France, reported that the incidence of mucopolysaccharidosis IVa (MPS IVa) is estimated at approximately 1 in 201,000 live births, although this number is believed to vary significantly across populations. Similarly, the birth prevalence of mucopolysaccharidosis VI (MPS VI) is estimated to range from 1 in 43,261 to 1 in 1,505,160, depending on the population. As a result, the increasing prevalence of different forms of mucopolysaccharidosis is expected to drive the mucopolysaccharidosis treatment market.
The rise in healthcare spending is contributing to the expansion of the mucopolysaccharidosis treatment market. Healthcare spending refers to the total financial resources dedicated to the healthcare sector within a particular region, country, or organization. The increase in healthcare spending is being directed toward mucopolysaccharidosis (MPS) treatment, aiming to improve access to specialized therapies, research, and medical infrastructure, thus enhancing the management and outcomes for individuals affected by this rare genetic disorder. For example, in July 2024, the American Medical Association, a U.S.-based professional organization of physicians and medical students, reported that health spending in the United States grew by 4.1% in 2022, reaching $4.5 trillion, or $13,493 per capita. Therefore, the rise in healthcare spending is driving the growth of the mucopolysaccharidosis treatment market moving forward.
Product innovation is an emerging trend gaining traction in the mucopolysaccharidosis treatment market. Leading companies in this market are concentrating on developing new products to maintain their competitive edge. For example, in September 2022, Ultragenyx Pharmaceutical Inc., a U.S.-based biopharmaceutical company, launched Vestronidase alfa (Mepsevii) in Japan for the treatment of Mucopolysaccharidosis Type VII (MPS VII or Sly Syndrome). This enzyme replacement therapy is designed to address the enzyme deficiency in MPS VII, helping break down glycosaminoglycans (GAGs) and reducing their buildup in tissues and organs. Mepsevii improves patients' mobility and respiratory function, offering a transformative treatment for individuals with this rare and progressive disorder. Its approval marks a significant advancement in providing essential care for rare disease patients in Japan.
Major players in the mucopolysaccharidosis treatment market are directing their focus towards cell therapies as a promising avenue for addressing rare genetic disorders. A case in point is ISP-001, developed by Immusoft, a US-based biotechnology company. ISP-001 represents a pioneering approach utilizing cell therapy to administer gene-encoded medicine, targeting the root cause of Mucopolysaccharidosis I (MPS I). In September 2022, Immusoft achieved a significant milestone with the FDA clearance of its Investigational New Drug (IND) application for ISP-001, marking a groundbreaking advancement in engineered B cell therapy for MPS I treatment.
In January 2024, Kyowa Kirin Co., Ltd., a Japan-based pharmaceutical company, acquired Orchard Therapeutics for an undisclosed sum. This acquisition is notable as it merges Kyowa Kirin’s extensive experience in biopharmaceuticals with Orchard’s innovative gene therapy platform, aimed at providing transformative treatments for patients with severe genetic disorders. Orchard Therapeutics, a UK-based biotech company, specializes in treatments for mucopolysaccharidosis, further enhancing the capabilities of both companies in addressing rare genetic diseases.
Major companies operating in the mucopolysaccharidosis treatment market include Pfizer Inc., Johnson & Johnson, Roche Holding AG, Merck & Co. Inc., AbbVie Inc., Novartis AG, Sanofi S.A., Bristol-Myers Squibb Company, GlaxoSmithKline plc, Takeda Pharmaceutical Co. Ltd., Teva Pharmaceutical Industries Ltd., BioMarin Pharmaceuticals Inc., Mallinckrodt Pharmaceuticals, Green Cross Corporation, Sarepta Therapeutics Inc., Esteve SA, JCR Pharmaceuticals Co. Ltd., Ultragenyx Pharmaceutical Inc., RegenxBio Inc., Sangamo Therapeutics Inc., Audentes Therapeutics Inc., Eidos Therapeutics Inc., Neurogene Inc., Immusoft Corporation, Bioasis Technologies Inc., Paradigm Biopharmaceuticals Ltd., Rimedion Inc., Eloxx Pharmaceuticals Inc., iBio Inc., Abeona Therapeutics Inc.
North America was the largest region in the mucopolysaccharidosis treatment market in 2024. The regions covered in the mucopolysaccharidosis treatment market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa. The countries covered in the mucopolysaccharidosis treatment market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.
The treatment of Mucopolysaccharidosis (MPS) involves addressing hereditary illnesses characterized by the body's inability to break down glycosaminoglycan (long sugar chains) due to the deficiency or malfunction of specific enzymes, often lysosomal enzymes.
Two primary types of treatments for mucopolysaccharidosis are enzyme replacement therapy and stem cell therapy. Enzyme replacement therapy is a medical intervention designed for patients with chronic diseases resulting from enzyme deficiency or dysfunction. This approach involves providing replacement enzymes to individuals affected by various forms of mucopolysaccharidosis, including types I, II, IV, VI, and others. These replacement enzymes are administered through intravenous and intracerebroventricular routes of administration. The therapy is implemented in diverse healthcare settings, including hospitals, specialty clinics, and other medical facilities.
The mucopolysaccharidosis treatment market research report is one of a series of new reports that provides mucopolysaccharidosis treatment market statistics, including mucopolysaccharidosis treatment industry global market size, regional shares, competitors with a mucopolysaccharidosis treatment market share, detailed mucopolysaccharidosis treatment market segments, market trends and opportunities, and any further data you may need to thrive in the mucopolysaccharidosis treatment industry. This mucopolysaccharidosis treatment market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.
The mucopolysaccharides treatment market includes revenues earned by entities by providing medication services such as enzyme replacement therapy, stem cell transplantation, gene therapy, substrate reduction therapy, and palliative care. 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 mucopolysaccharidosis treatment market size is expected to see strong growth in the next few years. It will grow to $3.51 billion in 2029 at a compound annual growth rate (CAGR) of 7.9%. The growth in the forecast period can be attributed to advancements in gene editing, regulatory changes and support, patient access initiatives, emerging therapeutic modalities, and precision medicine approaches. Major trends in the forecast period include caregiver support programs, focus on symptom management, education and training initiatives, expanded global access, and advances in biomarker development.
The rising prevalence of various forms of mucopolysaccharidosis is anticipated to boost the growth of the mucopolysaccharidosis treatment market in the future. Mucopolysaccharide is a type of complex carbohydrate that accumulates in body tissues due to the absence of the specific enzyme required for its breakdown. Mucopolysaccharidosis (MPS) therapy helps slow disease progression by reducing glycosaminoglycan (GAG) accumulation, managing symptoms, and preventing complications through various treatment approaches. For example, in July 2024, the Orphanet Journal of Rare Diseases, a peer-reviewed open-access medical journal based in France, reported that the incidence of mucopolysaccharidosis IVa (MPS IVa) is estimated at approximately 1 in 201,000 live births, although this number is believed to vary significantly across populations. Similarly, the birth prevalence of mucopolysaccharidosis VI (MPS VI) is estimated to range from 1 in 43,261 to 1 in 1,505,160, depending on the population. As a result, the increasing prevalence of different forms of mucopolysaccharidosis is expected to drive the mucopolysaccharidosis treatment market.
The rise in healthcare spending is contributing to the expansion of the mucopolysaccharidosis treatment market. Healthcare spending refers to the total financial resources dedicated to the healthcare sector within a particular region, country, or organization. The increase in healthcare spending is being directed toward mucopolysaccharidosis (MPS) treatment, aiming to improve access to specialized therapies, research, and medical infrastructure, thus enhancing the management and outcomes for individuals affected by this rare genetic disorder. For example, in July 2024, the American Medical Association, a U.S.-based professional organization of physicians and medical students, reported that health spending in the United States grew by 4.1% in 2022, reaching $4.5 trillion, or $13,493 per capita. Therefore, the rise in healthcare spending is driving the growth of the mucopolysaccharidosis treatment market moving forward.
Product innovation is an emerging trend gaining traction in the mucopolysaccharidosis treatment market. Leading companies in this market are concentrating on developing new products to maintain their competitive edge. For example, in September 2022, Ultragenyx Pharmaceutical Inc., a U.S.-based biopharmaceutical company, launched Vestronidase alfa (Mepsevii) in Japan for the treatment of Mucopolysaccharidosis Type VII (MPS VII or Sly Syndrome). This enzyme replacement therapy is designed to address the enzyme deficiency in MPS VII, helping break down glycosaminoglycans (GAGs) and reducing their buildup in tissues and organs. Mepsevii improves patients' mobility and respiratory function, offering a transformative treatment for individuals with this rare and progressive disorder. Its approval marks a significant advancement in providing essential care for rare disease patients in Japan.
Major players in the mucopolysaccharidosis treatment market are directing their focus towards cell therapies as a promising avenue for addressing rare genetic disorders. A case in point is ISP-001, developed by Immusoft, a US-based biotechnology company. ISP-001 represents a pioneering approach utilizing cell therapy to administer gene-encoded medicine, targeting the root cause of Mucopolysaccharidosis I (MPS I). In September 2022, Immusoft achieved a significant milestone with the FDA clearance of its Investigational New Drug (IND) application for ISP-001, marking a groundbreaking advancement in engineered B cell therapy for MPS I treatment.
In January 2024, Kyowa Kirin Co., Ltd., a Japan-based pharmaceutical company, acquired Orchard Therapeutics for an undisclosed sum. This acquisition is notable as it merges Kyowa Kirin’s extensive experience in biopharmaceuticals with Orchard’s innovative gene therapy platform, aimed at providing transformative treatments for patients with severe genetic disorders. Orchard Therapeutics, a UK-based biotech company, specializes in treatments for mucopolysaccharidosis, further enhancing the capabilities of both companies in addressing rare genetic diseases.
Major companies operating in the mucopolysaccharidosis treatment market include Pfizer Inc., Johnson & Johnson, Roche Holding AG, Merck & Co. Inc., AbbVie Inc., Novartis AG, Sanofi S.A., Bristol-Myers Squibb Company, GlaxoSmithKline plc, Takeda Pharmaceutical Co. Ltd., Teva Pharmaceutical Industries Ltd., BioMarin Pharmaceuticals Inc., Mallinckrodt Pharmaceuticals, Green Cross Corporation, Sarepta Therapeutics Inc., Esteve SA, JCR Pharmaceuticals Co. Ltd., Ultragenyx Pharmaceutical Inc., RegenxBio Inc., Sangamo Therapeutics Inc., Audentes Therapeutics Inc., Eidos Therapeutics Inc., Neurogene Inc., Immusoft Corporation, Bioasis Technologies Inc., Paradigm Biopharmaceuticals Ltd., Rimedion Inc., Eloxx Pharmaceuticals Inc., iBio Inc., Abeona Therapeutics Inc.
North America was the largest region in the mucopolysaccharidosis treatment market in 2024. The regions covered in the mucopolysaccharidosis treatment market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa. The countries covered in the mucopolysaccharidosis treatment market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.
The treatment of Mucopolysaccharidosis (MPS) involves addressing hereditary illnesses characterized by the body's inability to break down glycosaminoglycan (long sugar chains) due to the deficiency or malfunction of specific enzymes, often lysosomal enzymes.
Two primary types of treatments for mucopolysaccharidosis are enzyme replacement therapy and stem cell therapy. Enzyme replacement therapy is a medical intervention designed for patients with chronic diseases resulting from enzyme deficiency or dysfunction. This approach involves providing replacement enzymes to individuals affected by various forms of mucopolysaccharidosis, including types I, II, IV, VI, and others. These replacement enzymes are administered through intravenous and intracerebroventricular routes of administration. The therapy is implemented in diverse healthcare settings, including hospitals, specialty clinics, and other medical facilities.
The mucopolysaccharidosis treatment market research report is one of a series of new reports that provides mucopolysaccharidosis treatment market statistics, including mucopolysaccharidosis treatment industry global market size, regional shares, competitors with a mucopolysaccharidosis treatment market share, detailed mucopolysaccharidosis treatment market segments, market trends and opportunities, and any further data you may need to thrive in the mucopolysaccharidosis treatment industry. This mucopolysaccharidosis treatment market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.
The mucopolysaccharides treatment market includes revenues earned by entities by providing medication services such as enzyme replacement therapy, stem cell transplantation, gene therapy, substrate reduction therapy, and palliative care. 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. Mucopolysaccharidosis Treatment Market Characteristics3. Mucopolysaccharidosis Treatment Market Trends and Strategies4. Mucopolysaccharidosis Treatment Market - Macro Economic Scenario including the impact of Interest Rates, Inflation, Geopolitics and Covid and Recovery on the Market32. Global Mucopolysaccharidosis Treatment Market Competitive Benchmarking and Dashboard33. Key Mergers and Acquisitions in the Mucopolysaccharidosis Treatment Market34. Recent Developments in the Mucopolysaccharidosis Treatment Market
5. Global Mucopolysaccharidosis Treatment Growth Analysis and Strategic Analysis Framework
6. Mucopolysaccharidosis Treatment Market Segmentation
7. Mucopolysaccharidosis Treatment Market Regional and Country Analysis
8. Asia-Pacific Mucopolysaccharidosis Treatment Market
9. China Mucopolysaccharidosis Treatment Market
10. India Mucopolysaccharidosis Treatment Market
11. Japan Mucopolysaccharidosis Treatment Market
12. Australia Mucopolysaccharidosis Treatment Market
13. Indonesia Mucopolysaccharidosis Treatment Market
14. South Korea Mucopolysaccharidosis Treatment Market
15. Western Europe Mucopolysaccharidosis Treatment Market
16. UK Mucopolysaccharidosis Treatment Market
17. Germany Mucopolysaccharidosis Treatment Market
18. France Mucopolysaccharidosis Treatment Market
19. Italy Mucopolysaccharidosis Treatment Market
20. Spain Mucopolysaccharidosis Treatment Market
21. Eastern Europe Mucopolysaccharidosis Treatment Market
22. Russia Mucopolysaccharidosis Treatment Market
23. North America Mucopolysaccharidosis Treatment Market
24. USA Mucopolysaccharidosis Treatment Market
25. Canada Mucopolysaccharidosis Treatment Market
26. South America Mucopolysaccharidosis Treatment Market
27. Brazil Mucopolysaccharidosis Treatment Market
28. Middle East Mucopolysaccharidosis Treatment Market
29. Africa Mucopolysaccharidosis Treatment Market
30. Mucopolysaccharidosis Treatment Market Competitive Landscape and Company Profiles
31. Mucopolysaccharidosis Treatment Market Other Major and Innovative Companies
35. Mucopolysaccharidosis Treatment Market High Potential Countries, Segments and Strategies
36. Appendix
Executive Summary
Mucopolysaccharidosis Treatment Global Market Report 2025 provides strategists, marketers and senior management with the critical information they need to assess the market.This report focuses on mucopolysaccharidosis treatment 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 mucopolysaccharidosis treatment? How does the market relate to the overall economy, demography and other similar markets? What forces will shape the market going forward? The mucopolysaccharidosis treatment 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 Treatment: Enzyme Replacement Therapy; Stem Cell Therapy2) By Disease: Mucopolysaccharidosis- I; Mucopolysaccharidosis- II; Mucopolysaccharidosis-IV; Mucopolysaccharidosis-VI; Other Diseases
3) By Route of Administration: Intravenous; Intracerebroventricular
4) By End-User: Hospitals; Specialty Clinics; Other End-Users
Subsegments:
1) By Enzyme Replacement Therapy: Idursulfase (Elaprase); Laronidase (Aldurazyme); Velaglucerase Alfa (Vpriv); Galsulfase (Naglazyme)2) By Stem Cell Therapy: Hematopoietic Stem Cell Transplantation (HSCT); Umbilical Cord Blood Transplantation; Gene Therapy Approaches
Key Companies Mentioned: Pfizer Inc.; Johnson & Johnson; Roche Holding AG; Merck & Co. Inc.; AbbVie 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
- Pfizer Inc.
- Johnson & Johnson
- Roche Holding AG
- Merck & Co. Inc.
- AbbVie Inc.
- Novartis AG
- Sanofi S.A.
- Bristol-Myers Squibb Company
- GlaxoSmithKline plc
- Takeda Pharmaceutical Co. Ltd.
- Teva Pharmaceutical Industries Ltd.
- BioMarin Pharmaceuticals Inc.
- Mallinckrodt Pharmaceuticals
- Green Cross Corporation
- Sarepta Therapeutics Inc.
- Esteve SA
- JCR Pharmaceuticals Co. Ltd.
- Ultragenyx Pharmaceutical Inc.
- RegenxBio Inc.
- Sangamo Therapeutics Inc.
- Audentes Therapeutics Inc.
- Eidos Therapeutics Inc.
- Neurogene Inc.
- Immusoft Corporation
- Bioasis Technologies Inc.
- Paradigm Biopharmaceuticals Ltd.
- Rimedion Inc.
- Eloxx Pharmaceuticals Inc.
- iBio Inc.
- Abeona Therapeutics Inc.
Table Information
Report Attribute | Details |
---|---|
No. of Pages | 200 |
Published | February 2025 |
Forecast Period | 2025 - 2029 |
Estimated Market Value ( USD | $ 2.59 Billion |
Forecasted Market Value ( USD | $ 3.51 Billion |
Compound Annual Growth Rate | 7.9% |
Regions Covered | Global |
No. of Companies Mentioned | 30 |