[220 Pages Report] The global Mucopolysaccharidosis Treatment Market is estimated to be worth around US$ 2.38 Bn in 2022. With increasing prevalence of mucopolysaccharidosis (MSP) worldwide, the overall market is projected to grow at a robust CAGR of 5.9% between 2022 and 2029, reaching a valuation of US$ 3.37 Bn by 2029.
| Attribute | Details |
|---|---|
| Mucopolysaccharidosis treatment Market Estimated Size in 2022 | US$ 2.38 billion |
| Mucopolysaccharidosis treatment Market Projected Size in 2029 | US$ 3.37 billion |
| Mucopolysaccharidosis treatment Market Historical CAGR (2014 to 2021) | 5.2 % |
| Mucopolysaccharidosis treatment Market Value-Based CAGR (2022 to 2029) | 5.9% |
MSP II segment will continue to dominate the mucopolysaccharidosis market, accounting for a share of 33.2% in 2022. Rising prevalence of MSP disorders along with growing adoption of mucopolysaccharidosis treatment for MSP II is boosting the market.
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| Particulars | Details |
|---|---|
| H1, 2021 | 5.87% |
| H1, 2022 Projected | 5.94% |
| H1, 2022 Outlook | 5.44% |
| BPS Change to H1, 2022 (O) to H1, 2022 (P) | (-) 50 ↓ |
| BPS Change to H1, 2022 (O) to H1, 2021 | (-) 43 ↓ |
The comparative analysis and market growth rate of global mucopolysaccharides (MPS) treatment market has been studied by Future Market Insights. The global mucopolysaccharides (MPS) treatment market is predicted to drop by 43 Basis Share Point (BPS) in H1-2022 when compared to H1-2021, according to the FMI study.
Furthermore, the H1-2022 outlook and H1- 2022 projected growth rate showed declining variation in the BPS value by nearly 50 BPS. The fall in the BPS value for the mucopolysaccharides (MPS) treatment market can be attributed to the high cost of ERT and stem cell therapy treatment along with poor diagnosis rate and treatment rate in various emerging countries.
Regulatory compliances and clinical outcomes of treatment are dynamics which impact the market for mucopolysaccharides (MPS) treatment. The market is subject to be influenced by macro, regulatory, and industrial factors.
Furthermore, there has been an increase in the number of clinical trials in the recent time and thus, a huge number of pipeline products of many market players is estimated to flourish the market growth in the second half of the forecast period.
The global mucopolysaccharidosis treatment market is set to register a healthy CAGR of 5.9% between 2022 and 2029 in comparison to 5.2% CAGR registered from 2014 to 2021.
Rising health awareness, increasing prevalence of various forms of mucopolysaccharidosis worldwide, continuous advances in medical research and new drug launches and approvals are some of the factors driving the mucopolysaccharidosis treatment market.
Mucopolysaccharidosis is a group of inherited disorders in which the body is unable to breakdown glycosaminoglycan (long chains of sugars) due to absence or malfunctioning of certain enzymes. Presence of these disorders badly affects the physical and mental development of a person as well as damages the organs.
Although, there is no permanent cure for these disorders, mucopolysaccharidosis treatments such as enzyme replacement therapies and stem cell therapies can help to ease the symptoms.
Increasing prevalence of various type of mucopolysaccharidosis is a major force driving the global mucopolysaccharidosis treatment market. According to the National Organization for Rare Disorders (NORD), the total estimated prevalence of all forms of mucopolysaccharidosis is 1 in 25000 births.
Proper diagnosis of mucopolysaccharidosis is playing a vital role in expanding the growth of mucopolysaccharides treatment market. People with early diagnosis often opt for immediate treatment to relieve the symptoms and lead a normal life.
Key players operating in the market are continuously investing in research and development activities for new developing new therapeutics. Advances in stem cell therapies has provided a strong thrust to the mucopolysaccharidosis treatment market and the trend is likely to continue in the future.
Penetration of novel treatment approaches along with growing health awareness is anticipated to support the growth of mucopolysaccharidosis treatment market during the forecast period.
Spurred by aforementioned factors, the mucopolysaccharidosis treatment market is set to expand 1.4X through 2029.
Principal Consultant
Clinical trials has remained the key focus of mucopolysaccharidosis treatment during the last few years. Amid rise in the cases of rare diseases, academic researchers, clinicians and leading market players are increasingly focusing on developing new treatment options that can significantly relieve the symptoms and help the patients to lead a normal life.
Increasing clinical research is in turn creating lucrative growth prospects within mucopolysaccharidosis treatment market and the trend is likely to continue in future.
Various regulatory bodies are approving the clinical trials of innovative treatments. For instance, in February 2020 – FDA granted fast track designation to Lysogene's LYS-SAF302 program for the treatment of MPS IIIA. Currently the new second generation gene therapy (LYS-SAF302) is being investigated in the international phase 2/3 clinical trial.
Similarly in Feb 2021, JCR Pharmaceuticals Co., Ltd. received clearance of its Investigational New Drug (IND) application from the US Food and Drug Administration (FDA) to initiate a phase 3 clinical trial of JR-141 for the treatment of MPS II (Hunter syndrome) in the US.
Consistent clinical trials conducted to encourage the discovery of novel therapeutics is anticipated to expand the growth of mucopolysaccharidosis treatment market during the forecast period.
One of the major factors restraining the growth of mucopolysaccharidosis treatment market is the high cost associated with mucopolysaccharidosis treatment. Both ERT and stem cell therapies treatment are expensive. As a result, a large section population are not able to afford advanced treatments.
Moreover, lack of favorable reimbursement policies across and poor health awareness are limiting the growth of the market.
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“Drug Development and Approvals to Spur the U.S. Market Growth”
As per FMI, the U.S. will continue to dominate the North America mucopolysaccharidosis treatment market during the forecast period. The U.S. mucopolysaccharides treatment market is driven by rising awareness, new drug approvals, presence of favorable reimbursement policies, well established healthcare infrastructure and presence of leading market players.
The U.S. is one of the most advanced nations in the world. It is home to some of the leading pharmaceutical companies. Advancements in medical research has enabled the market players to develop innovative products with effective features. New treatments are being continuously introduced to counter the burden of rare diseases. This is, in turn triggering the growth of mucopolysaccharides treatment market.
Presence of regulatory bodies such as the U.S Food and Drug Administration (FDA) is playing a key role in expanding the U.S. mucopolysaccharidosis treatment market. Various steps are being taken to foster the drug development for rare diseases.
For instance, in February 2020, FDA issued a draft guidance regarding the rare disease, Sanofilippo syndrome. The draft is aimed to support the development of therapies for MPS III (Sanfilippo syndrome)
Moreover, the rise in the cases of mucopolysaccharidosis coupled with increasing diagnostic screening is positively impacting the growth of the U.S. mucopolysaccharidosis treatment market. According to an article published in the Orphanet Journal of Rare Diseases, prevalence of MPS in the U.S. is around 2.67 per 1 million.
Amid intensifying clinical trials, a healthy share has been projected for the U.S. mucopolysaccharides treatment market by 2029.
“Focus on Clinical Trials Presenting Attractive Growth Prospects in Japan”
Japan boasts the presence of advanced healthcare system. The government in Japan spends increasingly on developing healthcare infrastructure to accommodate the nation’s healthcare needs in the most efficient manner possible.
Over the last few decades, there has been an increase in the cases of mucopolysaccharidosis disorders. This has triggered the growth of mucopolysaccharidosis treatment market in the country.
Various novel treatments such as hunterase are being introduced in Japan to help the patients suffering from genetic MPS disorders. Moreover, various clinical trials are being conducted to find new and effective treatments for various type of mucopolysaccharidosis disorders.
For instance, in 2021, JCR Pharmaceuticals initiated a phase 3 clinical trial of JR-141 for the treatment of hunter syndrome.
“Rising Investments in Medical Research to Present Attractive Prospects for Growth in the U.K.”
As per FMI, the U.K. mucopolysaccharidosis treatment market is poised to exhibit strong growth during the forecast period. Increasing government support, rising prevalence of rare disorders and increasing investments in medical research are some of the factors driving the U.K. mucopolysaccharidosis treatment market.
The U.K. government spends millions of dollars on medical research annually. According to the National Institute for Health Research (NIHR), around € 4.5 billion was invested in the U.K. health research projects in 2018. This is positively impacting the market growth across the country.
Moreover, growing popularity of stem cell therapies for the effective treatment of various type of mucopolysaccharidosis will continue to accelerate the growth of the market during the forecast period from 2022 and 2029.
“Enzyme Replacement Therapy (ERT) Remains the Gold Standard Treatment for Mucopolysaccharidosis”
Based on treatment, mucopolysaccharidosis treatment market is segmented into enzyme replacement therapies (ERT) and stem cell therapies. Among these two, enzyme replacement therapies segment dominates the mucopolysaccharidosis treatment market.
ERT remains the primary treatment option for mucopolysaccharidosis across the globe. Easy availability and continuous approvals remain the key factors fueling the adoption of enzymes replacement therapies for relieving the symptoms of mucopolysaccharidosis.
Enzyme replacement therapies replace or generate the deficient enzyme and thus effectively helps to treat the condition. Drugs such as aldurazyme, naglazyme, vimizim, elaprase, mepsevii and hunterase are being consumed on large scales for the treatment of mucopolysaccharides.
“MPS II Segment to Continue Accounting for Maximum Sales”
In terms of MPS type, the mucopolysaccharidosis treatment has been segmented into MPS I, MPS II, MPS IV A, MPS VI and MPS VII. Among these, MPS II (Hunter syndrome) segment dominates the global mucopolysaccharidosis treatment market, accounting for the largest share of 33.2% in 2022.
MPS II or hunter syndrome is a rare inherited genetic disorder caused by absence or malfunctioning of enzyme 2-sulfatas. Absence of this enzyme causes permanent progressive damage affecting mental development, appearance and organ functions. As a result, medical products such as hunterase and elaprase are being consumed to relieve the symptoms.
Increasing prevalence of MPS II coupled with presence of multiple treatment options is triggering the growth of MPS II segment and the trend is likely to continue in the future.
“Favorable Scenario Enables Dominance of Hospitals Segment”
In terms of end user, the global donor egg IVF market is segmented into hospital, specialty clinics, medical research centers and home-infusion.
Among these, the hospitals segment accounts for major revenue share in the global mucopolysaccharides treatment market in 2022. Presence of trained medical professionals and low-cost treatment are some of the factors prompting patients to opt for mucopolysaccharidosis treatment in hospitals.
Hospitals are expected to remain the only means of accessing the treatment for maximum number of people living in developing and underdeveloped regions. However, specialty clinics and home infusion segments are anticipated to grow at a robust CAGR over the forecast period.
Leading players operating in mucopolysaccharidosis treatment market are vigorously investing in research and development activities. They are adopted various organic and inorganic growth strategies such as mergers, new product launches and approvals, partnerships and acquisitions to gain a competitive edge in the market.
| Attribute | Details |
|---|---|
| Historical Data Available for | 2014-2021 |
| Forecast Period | 2022-2029 |
| Market Analysis | Units for Volume and US$ Mn for Value |
| Key Regions Covered | North America; Latin America; Europe; South Asia; East Asia; Oceania; and the Middle East & Africa |
| Key Countries Covered | USA, Canada, Mexico, Brazil, Germany, UK, France, Italy, Spain, Russia, Poland, China, Japan, South Korea, India, ASEAN, Turkey and South Africa |
| Key Segments Covered | Cycle Type, End Use and Region |
| Key Companies Profiled | BioMarin; Takeda Pharmaceutical Company Limited; Sanofi S.A.; Ultragenyx Pharmaceutical Inc.; Sangamo Therapeutics, Inc.; REGENXBIO Inc.; Sarepta Therapeutics; Abeona Therapeutics, Inc. |
| Report Coverage | Market Forecast, Company Share Analysis, Competition Intelligence, DROT Analysis, Market Dynamics and Challenges, and Strategic Growth Initiatives |
| Customization & Pricing | Available upon Request |
The mucopolysaccharidosis treatment market is estimated to be worth around US$ 2.34 Bn in 2022.
As per FMI, the mucopolysaccharidosis treatment market is forecast to register a steady CAGR of 5.9% between 2022 and 2029
The mucopolysaccharidosis treatment market grew at a CAGR of 5.2% between 2014 and 2021.
Continuous drug approvals, increasing government initiatives to tackle the burden of rare diseases and rising penetration of novel treatments across developing economies are some of the key trends shaping the growth of mucopolysaccharidosis treatment market.
BioMarin, Takeda Pharmaceutical Company Limited, Sanofi S.A., and Ultragenyx Pharmaceutical Inc. BioMarin, Sangamo Therapeutics, Inc., REGENXBIO Inc., Sarepta Therapeutics, Abeona Therapeutics, Inc. are some of the leading players operating in the mucopolysaccharidosis treatment market.
1. Executive Summary
1.1. Global Market Outlook
1.2. Demand Side Trends
1.3. Supply Side Trends
1.4. Analysis and Recommendations
2. Market Overview
2.1. Market Coverage / Taxonomy
2.2. Market Definition / Scope / Limitations
3. Key Market Trends
3.1. Key Trends Impacting the Market
3.2. Technological Developments
4. Key Success Factors
4.1. Regulatory Scenario
4.2. Pipeline Analysis
4.3. Snapshot of Developments for Mucopolysaccharidosis Type 3
4.4. Designed Designation
4.5. Rare Disease Framework
5. Global Mucopolysaccharidosis Treatment Market Analysis 2014-2021 and Forecast, 2022-2029
5.1. Historical Market Value (US$ Mn) Analysis, 2014-2021
5.2. Current and Future Market Value (US$ Mn) Projections, 2022-2029
5.2.1. Y-o-Y Growth Trend Analysis
5.2.2. Absolute $ Opportunity Analysis
6. Market Background
6.1. Macro-Economic Factors
6.1.1. Global GDP Growth Outlook
6.1.2. Global Healthcare Expenditure Outlook
6.2. Forecast Factors - Relevance & Impact
6.2.1. Improved Services in Concerned Economies
6.2.2. Drug Candidates in Pipeline
6.2.3. Drug Approval Process in Different Economies
6.2.4. Growth in Production Capacity
6.2.5. Change in Cost of Treatment
6.3. Market Dynamics
6.3.1. Drivers
6.3.2. Restraints
6.3.3. Opportunity Analysis
7. Global Mucopolysaccharidosis Treatment Market Analysis 2014-2021 and Forecast 2022-2029, by Treatment Type
7.1. Introduction / Key Findings
7.2. Historical Market Size (US$ Mn) Analysis By Treatment Type, 2014 - 2021
7.3. Current and Future Market Size (US$ Mn) Analysis and Forecast By Treatment Type, 2022 - 2029
7.3.1. Enzyme Replacement Therapies
7.3.2. Stem Cell Therapies
7.3.2.1. Bone Marrow Transplantation (BMT)
7.3.2.2. Umbilical Cord Blood Transplantation (UCBT)
7.4. Market Attractiveness Analysis By Treatment Type
8. Global Mucopolysaccharidosis Treatment Market Analysis 2014-2021 and Forecast 2022-2029, by Type of MPS
8.1. Introduction / Key Findings
8.2. Historical Market Size (US$ Mn) Analysis By Type of MPS, 2014 - 2021
8.3. Current and Future Market Size (US$ Mn) Analysis and Forecast By Type of MPS, 2022 - 2029
8.3.1. MPS I
8.3.2. MPS II
8.3.3. MPS IV A
8.3.4. MPS VI
8.3.5. MPS VII
8.4. Market Attractiveness Analysis By Type of MPS
9. Global Mucopolysaccharidosis Treatment Market Analysis 2014-2021 and Forecast 2022-2029, by End User
9.1. Introduction / Key Findings
9.2. Historical Market Size (US$ Mn) Analysis By End User, 2014 - 2021
9.3. Current and Future Market Size (US$ Mn) Analysis and Forecast By End User, 2022 - 2029
9.3.1. Hospital
9.3.2. Specialty Clinics
9.3.3. Medical Research Centers
9.3.4. Home-infusion
9.4. Market Attractiveness Analysis By End User
10. Global Mucopolysaccharidosis Treatment Market Analysis 2014-2021 and Forecast 2022-2029, by Region
10.1. Introduction
10.2. Historical Market Size (US$ Mn) Analysis By Region, 2014 - 2021
10.3. Current Market Size (US$ Mn) Analysis and Forecast By Region, 2022 - 2029
10.3.1. North America
10.3.2. Latin America
10.3.3. Europe
10.3.4. East Asia
10.3.5. South Asia
10.3.6. MEA
10.3.7. Oceania
10.4. Market Attractiveness Analysis By Region
11. North America Mucopolysaccharidosis Treatment Market Analysis 2014-2021 and Forecast 2022-2029
11.1. Introduction
11.2. Historical Market Size (US$ Mn) Trend Analysis By Market Taxonomy, 2014 - 2021
11.3. Market Size (US$ Mn) Forecast By Market Taxonomy, 2022 - 2029
11.3.1. By Country
11.3.1.1. U.S.
11.3.1.2. Canada
11.3.2. By Treatment Type
11.3.3. By Type of MPS
11.3.4. By End User
11.4. Market Attractiveness Analysis
12. Latin America Mucopolysaccharidosis Treatment Market Analysis 2014-2021 and Forecast 2022-2029
12.1. Introduction
12.2. Historical Market Size (US$ Mn) Trend Analysis By Market Taxonomy, 2014 - 2021
12.3. Market Size (US$ Mn) Forecast By Market Taxonomy, 2022 - 2029
12.3.1. By Country
12.3.1.1. Brazil
12.3.1.2. Mexico
12.3.1.3. Argentina
12.3.1.4. Rest of Latin America
12.3.2. By Treatment Type
12.3.3. By Type of MPS
12.3.4. By End User
12.4. Market Attractiveness Analysis
13. Europe Mucopolysaccharidosis Treatment Market Analysis 2014-2021 and Forecast 2022-2029
13.1. Introduction
13.2. Historical Market Size (US$ Mn) Trend Analysis By Market Taxonomy, 2014 - 2021
13.3. Market Size (US$ Mn) Forecast By Market Taxonomy, 2022 - 2029
13.3.1. By Country
13.3.1.1. Germany
13.3.1.2. Italy
13.3.1.3. France
13.3.1.4. U.K.
13.3.1.5. Spain
13.3.1.6. BENELUX
13.3.1.7. Russia
13.3.1.8. Rest of Europe
13.3.2. By Treatment Type
13.3.3. By Type of MPS
13.3.4. By End User
13.4. Market Attractiveness Analysis
14. South Asia Hysteroscopy Systems Market Analysis 2014-2021 and Forecast 2022-2029
14.1. Introduction
14.2. Historical Market Size (US$ Mn) Trend Analysis By Market Taxonomy, 2014 - 2021
14.3. Market Size (US$ Mn) Forecast By Market Taxonomy, 2022 - 2029
14.3.1. By Country
14.3.1.1. India
14.3.1.2. Thailand
14.3.1.3. Indonesia
14.3.1.4. Malaysia
14.3.1.5. Rest of South Asia
14.3.2. By Treatment Type
14.3.3. By Type of MPS
14.3.4. By End User
14.4. Market Attractiveness Analysis
15. East Asia Hysteroscopy Systems Market Analysis 2014-2021 and Forecast 2022-2029
15.1. Introduction
15.2. Historical Market Size (US$ Mn) Trend Analysis By Market Taxonomy, 2014 - 2021
15.3. Market Size (US$ Mn) Forecast By Market Taxonomy, 2022 - 2029
15.3.1. By Country
15.3.1.1. China
15.3.1.2. Japan
15.3.1.3. South Korea
15.3.2. By Treatment Type
15.3.3. By Type of MPS
15.3.4. By End User
15.4. Market Attractiveness Analysis
16. Oceania Mucopolysaccharidosis Treatment Market Analysis 2014-2021 and Forecast 2022-2029
16.1. Introduction
16.2. Historical Market Size (US$ Mn) Trend Analysis By Market Taxonomy, 2014 - 2021
16.3. Market Size (US$ Mn) Forecast By Market Taxonomy, 2022 - 2029
16.3.1. By Country
16.3.1.1. Australia
16.3.1.2. New Zealand
16.3.2. By Treatment Type
16.3.3. By Type of MPS
16.3.4. By End User
16.4. Market Attractiveness Analysis
17. Middle East and Africa Mucopolysaccharidosis Treatment Market Analysis 2014-2021 and Forecast 2022-2029
17.1. Introduction
17.2. Historical Market Size (US$ Mn) Trend Analysis By Market Taxonomy, 2014 - 2021
17.3. Market Size (US$ Mn) Forecast By Market Taxonomy, 2022 - 2029
17.3.1. By Country
17.3.1.1. GCC Countries
17.3.1.2. South Africa
17.3.1.3. Rest of Middle East and Africa
17.3.2. By Treatment Type
17.3.3. By Type of MPS
17.3.4. By End User
17.4. Market Attractiveness Analysis
18. Market Structure Analysis
18.1. Market Share Analysis
18.2. Competition Mapping
19. Competition Analysis
19.1. Company Dashboard
19.2. Company Profile
19.2.1. Sanofi S.A.
19.2.1.1. Overview
19.2.1.2. Service Portfolio
19.2.1.3. Analyst Commentary
19.2.2. Shire
19.2.3. BioMarin Pharmaceutical Inc.
19.2.4. Ultragenyx Pharmaceutical Inc.
19.2.5. Sarepta Therapeutics
19.2.6. Abeona Therapeutics, Inc.
19.2.7. ArmaGen
19.2.8. Eloxx Pharmaceuticals
19.2.9. Esteve
19.2.10. Immusoft Corporation
19.2.11. Inventiva
19.2.12. Orchard Therapeutics Limited
19.2.13. REGENXBIO Inc.
19.2.14. Sangamo Therapeutics, Inc.
20. Assumptions and Acronyms Used
21. Research Methodology
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Mucopolysaccharidosis (MPS) Treatment Market
