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Morquio Syndrome (MPS-IV) Drug Market Size, Share, Growth & Forecast (2024-2032)
Morquio Syndrome (MPS-IV) Drug Market Size Was Valued at USD 1.69 Million in 2023, and is Projected to Reach USD 4.8 Million by 2032, Growing at a CAGR of 12.4% From 2024-2032.
IMR
Description
Morquio Syndrome (MPS-IV) Drug Market Synopsis:
Morquio Syndrome (MPS-IV) Drug Market Size Was Valued at USD 1.69 Million in 2023, and is Projected to Reach USD 4.8 Million by 2032, Growing at a CAGR of 12.4% From 2024-2032.
Morquio Syndrome or Mucopolysaccharidosis IV is a genetic disorder in which the deficiency of L-iduronate sulfatase results in the inability of the body to metabolize glycosaminoglycans which consequently leads to skeletal abnormalities, joint issues, and other complications. Management of MPS IV was centered on ERT, supportive and compassionate care, and gene therapy in the pipeline. This market is focused on drugs and treatment for MPS IV patients as well as trying to slow down the progression of the symptoms with an attempt to increase the quality of life of patients with these diseases.
Morquio Syndrome (MPS-IV) is a rare drug market, but it has been experiencing a gradual expansion in recent years owing to developing awareness, growth of genetic science and new ways of treatment. Currently it remains relatively a small but quite comprehensive with the main focus made on enzyme replacement therapeutic approach. The syndrome is very rare and therefore the market for the treatment remains small; nonetheless, the increased attention paid to the development of orphan drugs thanks to favorable governmental policies and initiatives makes this market attractive to pharmaceutical and biotech companies. The growth of the market is also fueled by the growing research funds as this has led to more players and new breakthrough solutions.
Additionally, increasing healthcare costs and advocacy group support are factors which drive this market growth. MPS IV thus results in a large health care concern which has a caregiver component due to the many health problems identified in the patients. This need has not gone unnoticed by major stakeholders in this field, which consists of healthcare practitioners, researchers, and biopharmaceutical companies, who have already established a continuous improvement ecosystem of MPS IV therapies over the years. Furthermore, increasing partnerships between research organizations and drug companies to develop such new and efficient therapeutic products further supports this argument.
Morquio Syndrome (MPS-IV) Drug Market Trend Analysis:
Advancements in Gene Therapy
Gene therapy is, in turn, seen to be assuming the role of a revolutionary trend in the Morquio Syndrome drug market. With recent scientific progress, Gene therapy is being studied as a potential long-term treatment which targets the source of the disease. Traditional treatment strategies using enzymes are different from gene therapy because the latter attacks the actual source of the problem, the gene mutation responsible for MPS IV, and may presumably arrest or reverse the disease. There are presently several ongoing clinical trials that are looking at the safety and effectiveness of these therapies: the initial findings which are encouraging suggest that there are indeed directed approaches to the disease.
This trend is beginning to take hold in researchers and investors because previous triumphs in other genetic disorders make apparent the opportunity for gene therapy in rare diseases. This gradual shift towards gene-based solutions also fits with a general trend in the biopharma space where targeted and precision medicines are favored. Delivery mechanisms and costs still remain to be an issue for the MPS IV market however the fight against MPS IV have prompted strong focus on gene therapies as the key to a monumental shift within the realm of medical treatment.
Growth in Orphan Drug Development
One of the highly important trends mentioned is the trend of focusing more on the orphan drugs which is a clear opportunity for the MPS-IV drug market.. The treatments developed for Morquio Syndrome can be considered as orphan drugs due to the lack of commercial interest in this illness and the above-mentioned incentives which include tax credits for finding orphan drugs and new tax incentives for marketing orphan drugs, extended patent exclusivity periods and reduced patent fees to facilitate the sale of orphan drugs. These incentives have motivated pharmaceutical companies to work on and ensure investment in MPS-IV treatment hence; enhanced research, development and technology advancement in the limited market.
Morquio Syndrome (MPS-IV) Drug Market Segment Analysis:
Morquio Syndrome (MPS-IV) Drug Market is Segmented on the basis of Type, Treatment, End User, and Region.
By Type, Solid Dosage Form segment is expected to dominate the market during the forecast period
The pharmaceutical industry research suggests that during the forecasting period, the administration, compliance and stability features of the segment called Solid Dosage Form will claim the leading position in the Morquio Syndrome (MPS-IV) drugs market.. Liquids and specifically injectable are difficult when it comes to packaging, storage, and transportation as compared to solid dosage forms for instance tablets and capsules. Further on, solid dosage forms and their results normally have higher durability on the average, the overall wastage level is minimized and the availability of the forms is more or less guaranteed. Thus, driving the projected market domination of the solid dosage forms, the diffuse application of special treatments in rare diseases such as Morquio Syndrome, where the use of solid dosage forms is far more effective and cost-saving for both manufacturers and obligatory purchasing heath care systems.
By treatment, Enzyme Replacement Therapy segment expected to held the largest share
Enzyme Replacement Therapy segment is expected to have the largest market share in Morquio Syndrome (MPS-IV) drug market due to the record proven efficacy in controlling the symptoms of this genetic disorder. ERT is effective in that it replaces the deficient enzyme – N-acetylgalactosamine-6-sulfatase that is necessary for the degradation of certain glycosaminoglycans in patients suffering from MPS-IV. There is strong evidence this therapy slows the progression of diseases, relieves symptoms of the condition and enhances the wellbeing of patients diagnosed with it. Considering the constant growing biotechnology field and evidence based proving advantages of ERT for MPS-IV, the treatment keeps on dominating the market and remaining the gold standard for MPS-IV therapy.
Morquio Syndrome (MPS-IV) Drug Market Regional Insights:
North America is Expected to Dominate the Market Over the Forecast period
North America occupied the largest share in the global Morquio Syndrome drug market in 2023 due to increased healthcare expenditure, availability of developed healthcare services, increasing research & development activities. Market particularly well developed in United States due to enzyme replacement therapies available here, high governmental support in research on ODD, and active organisations working in alleviation of conditions associated with ODD and better availability of treatments for these conditions. At the current rate, it is estimated that North America holds about 45-50% global control of the MPS-IV drugs market, showing concern in the provision of new therapies for such rare conditions as MPS IV. Europe comes next, with significant overall ratios from Germany, France and the United Kingdom where a favorable regulatory environment and emphasis for the therapy of rare diseases is observed.
Active Key Players in the Morquio Syndrome (MPS-IV) Drug Market:
Abeona Therapeutics (USA)
Amicus Therapeutics (USA)
BioMarin Pharmaceutical (USA)
Denali Therapeutics (USA)
Green Cross Corporation (South Korea)
JCR Pharmaceuticals (Japan)
Lysogene (France)
Orchard Therapeutics (UK)
Pfizer (USA)
RegenxBio (USA)
Sanofi (France)
Sarepta Therapeutics (USA)
Shire (Ireland)
Takeda Pharmaceutical (Japan)
Ultragenyx Pharmaceutical (USA)
Other Active Players
Chapter 1: Introduction
1.1 Scope and Coverage
Chapter 2:Executive Summary
Chapter 3: Market Landscape
3.1 Market Dynamics
3.1.1 Drivers
3.1.2 Restraints
3.1.3 Opportunities
3.1.4 Challenges
3.2 Market Trend Analysis
3.3 PESTLE Analysis
3.4 Porter’s Five Forces Analysis
3.5 Industry Value Chain Analysis
3.6 Ecosystem
3.7 Regulatory Landscape
3.8 Price Trend Analysis
3.9 Patent Analysis
3.10 Technology Evolution
3.11 Investment Pockets
3.12 Import-Export Analysis
Chapter 4: Morquio Syndrome (MPS-IV) Drug Market by Type
4.1 Morquio Syndrome (MPS-IV) Drug Market Snapshot and Growth Engine
4.2 Morquio Syndrome (MPS-IV) Drug Market Overview
4.3 Solid Dosage Form
4.3.1 Introduction and Market Overview
4.3.2 Historic and Forecasted Market Size in Value USD and Volume Units (2017-2032F)
4.3.3 Key Market Trends, Growth Factors and Opportunities
4.3.4 Solid Dosage Form: Geographic Segmentation Analysis
4.4 Liquid Dosage Form
4.4.1 Introduction and Market Overview
4.4.2 Historic and Forecasted Market Size in Value USD and Volume Units (2017-2032F)
4.4.3 Key Market Trends, Growth Factors and Opportunities
4.4.4 Liquid Dosage Form: Geographic Segmentation Analysis
Chapter 5: Morquio Syndrome (MPS-IV) Drug Market by Treatment
5.1 Morquio Syndrome (MPS-IV) Drug Market Snapshot and Growth Engine
5.2 Morquio Syndrome (MPS-IV) Drug Market Overview
5.3 Enzyme Replacement Therapy
5.3.1 Introduction and Market Overview
5.3.2 Historic and Forecasted Market Size in Value USD and Volume Units (2017-2032F)
5.3.3 Key Market Trends, Growth Factors and Opportunities
5.3.4 Enzyme Replacement Therapy: Geographic Segmentation Analysis
5.4 Hematopoietic Stem Cell Therapy
5.4.1 Introduction and Market Overview
5.4.2 Historic and Forecasted Market Size in Value USD and Volume Units (2017-2032F)
5.4.3 Key Market Trends, Growth Factors and Opportunities
5.4.4 Hematopoietic Stem Cell Therapy: Geographic Segmentation Analysis
5.5 Gene Therapy
5.5.1 Introduction and Market Overview
5.5.2 Historic and Forecasted Market Size in Value USD and Volume Units (2017-2032F)
5.5.3 Key Market Trends, Growth Factors and Opportunities
5.5.4 Gene Therapy: Geographic Segmentation Analysis
5.6 Others
5.6.1 Introduction and Market Overview
5.6.2 Historic and Forecasted Market Size in Value USD and Volume Units (2017-2032F)
5.6.3 Key Market Trends, Growth Factors and Opportunities
5.6.4 Others: Geographic Segmentation Analysis
Chapter 6: Morquio Syndrome (MPS-IV) Drug Market by End User
6.1 Morquio Syndrome (MPS-IV) Drug Market Snapshot and Growth Engine
6.2 Morquio Syndrome (MPS-IV) Drug Market Overview
6.3 Hospital Pharmacies
6.3.1 Introduction and Market Overview
6.3.2 Historic and Forecasted Market Size in Value USD and Volume Units (2017-2032F)
6.3.3 Key Market Trends, Growth Factors and Opportunities
6.3.4 Hospital Pharmacies: Geographic Segmentation Analysis
6.4 Online Pharmacies
6.4.1 Introduction and Market Overview
6.4.2 Historic and Forecasted Market Size in Value USD and Volume Units (2017-2032F)
6.4.3 Key Market Trends, Growth Factors and Opportunities
6.4.4 Online Pharmacies: Geographic Segmentation Analysis
6.5 Retail Pharmacies
6.5.1 Introduction and Market Overview
6.5.2 Historic and Forecasted Market Size in Value USD and Volume Units (2017-2032F)
6.5.3 Key Market Trends, Growth Factors and Opportunities
6.5.4 Retail Pharmacies: Geographic Segmentation Analysis
Chapter 7: Company Profiles and Competitive Analysis
7.1 Competitive Landscape
7.1.1 Competitive Benchmarking
7.1.2 Morquio Syndrome (MPS-IV) Drug Market Share by Manufacturer (2023)
7.1.3 Industry BCG Matrix
7.1.4 Heat Map Analysis
7.1.5 Mergers and Acquisitions
7.2 ABEONA THERAPEUTICS (USA)
7.2.1 Company Overview
7.2.2 Key Executives
7.2.3 Company Snapshot
7.2.4 Role of the Company in the Market
7.2.5 Sustainability and Social Responsibility
7.2.6 Operating Business Segments
7.2.7 Product Portfolio
7.2.8 Business Performance
7.2.9 Key Strategic Moves and Recent Developments
7.2.10 SWOT Analysis
7.3 AMICUS THERAPEUTICS (USA)
7.4 BIOMARIN PHARMACEUTICAL (USA)
7.5 DENALI THERAPEUTICS (USA)
7.6 GREEN CROSS CORPORATION (SOUTH KOREA)
7.7 JCR PHARMACEUTICALS (JAPAN)
7.8 LYSOGENE (FRANCE)
7.9 ORCHARD THERAPEUTICS (UK)
7.10 PFIZER (USA)
7.11 REGENXBIO (USA)
7.12 SANOFI (FRANCE)
7.13 SAREPTA THERAPEUTICS (USA)
7.14 SHIRE (IRELAND)
7.15 TAKEDA PHARMACEUTICAL (JAPAN)
7.16 ULTRAGENYX PHARMACEUTICAL (USA)
7.17
7.18 OTHER ACTIVE PLAYERS
Chapter 8: Global Morquio Syndrome (MPS-IV) Drug Market By Region
8.1 Overview
8.2. North America Morquio Syndrome (MPS-IV) Drug Market
8.2.1 Key Market Trends, Growth Factors and Opportunities
8.2.2 Top Key Companies
8.2.3 Historic and Forecasted Market Size by Segments
8.2.4 Historic and Forecasted Market Size By Type
8.2.4.1 Solid Dosage Form
8.2.4.2 Liquid Dosage Form
8.2.5 Historic and Forecasted Market Size By Treatment
8.2.5.1 Enzyme Replacement Therapy
8.2.5.2 Hematopoietic Stem Cell Therapy
8.2.5.3 Gene Therapy
8.2.5.4 Others
8.2.6 Historic and Forecasted Market Size By End User
8.2.6.1 Hospital Pharmacies
8.2.6.2 Online Pharmacies
8.2.6.3 Retail Pharmacies
8.2.7 Historic and Forecast Market Size by Country
8.2.7.1 US
8.2.7.2 Canada
8.2.7.3 Mexico
8.3. Eastern Europe Morquio Syndrome (MPS-IV) Drug Market
8.3.1 Key Market Trends, Growth Factors and Opportunities
8.3.2 Top Key Companies
8.3.3 Historic and Forecasted Market Size by Segments
8.3.4 Historic and Forecasted Market Size By Type
8.3.4.1 Solid Dosage Form
8.3.4.2 Liquid Dosage Form
8.3.5 Historic and Forecasted Market Size By Treatment
8.3.5.1 Enzyme Replacement Therapy
8.3.5.2 Hematopoietic Stem Cell Therapy
8.3.5.3 Gene Therapy
8.3.5.4 Others
8.3.6 Historic and Forecasted Market Size By End User
8.3.6.1 Hospital Pharmacies
8.3.6.2 Online Pharmacies
8.3.6.3 Retail Pharmacies
8.3.7 Historic and Forecast Market Size by Country
8.3.7.1 Russia
8.3.7.2 Bulgaria
8.3.7.3 The Czech Republic
8.3.7.4 Hungary
8.3.7.5 Poland
8.3.7.6 Romania
8.3.7.7 Rest of Eastern Europe
8.4. Western Europe Morquio Syndrome (MPS-IV) Drug Market
8.4.1 Key Market Trends, Growth Factors and Opportunities
8.4.2 Top Key Companies
8.4.3 Historic and Forecasted Market Size by Segments
8.4.4 Historic and Forecasted Market Size By Type
8.4.4.1 Solid Dosage Form
8.4.4.2 Liquid Dosage Form
8.4.5 Historic and Forecasted Market Size By Treatment
8.4.5.1 Enzyme Replacement Therapy
8.4.5.2 Hematopoietic Stem Cell Therapy
8.4.5.3 Gene Therapy
8.4.5.4 Others
8.4.6 Historic and Forecasted Market Size By End User
8.4.6.1 Hospital Pharmacies
8.4.6.2 Online Pharmacies
8.4.6.3 Retail Pharmacies
8.4.7 Historic and Forecast Market Size by Country
8.4.7.1 Germany
8.4.7.2 UK
8.4.7.3 France
8.4.7.4 The Netherlands
8.4.7.5 Italy
8.4.7.6 Spain
8.4.7.7 Rest of Western Europe
8.5. Asia Pacific Morquio Syndrome (MPS-IV) Drug Market
8.5.1 Key Market Trends, Growth Factors and Opportunities
8.5.2 Top Key Companies
8.5.3 Historic and Forecasted Market Size by Segments
8.5.4 Historic and Forecasted Market Size By Type
8.5.4.1 Solid Dosage Form
8.5.4.2 Liquid Dosage Form
8.5.5 Historic and Forecasted Market Size By Treatment
8.5.5.1 Enzyme Replacement Therapy
8.5.5.2 Hematopoietic Stem Cell Therapy
8.5.5.3 Gene Therapy
8.5.5.4 Others
8.5.6 Historic and Forecasted Market Size By End User
8.5.6.1 Hospital Pharmacies
8.5.6.2 Online Pharmacies
8.5.6.3 Retail Pharmacies
8.5.7 Historic and Forecast Market Size by Country
8.5.7.1 China
8.5.7.2 India
8.5.7.3 Japan
8.5.7.4 South Korea
8.5.7.5 Malaysia
8.5.7.6 Thailand
8.5.7.7 Vietnam
8.5.7.8 The Philippines
8.5.7.9 Australia
8.5.7.10 New Zealand
8.5.7.11 Rest of APAC
8.6. Middle East & Africa Morquio Syndrome (MPS-IV) Drug Market
8.6.1 Key Market Trends, Growth Factors and Opportunities
8.6.2 Top Key Companies
8.6.3 Historic and Forecasted Market Size by Segments
8.6.4 Historic and Forecasted Market Size By Type
8.6.4.1 Solid Dosage Form
8.6.4.2 Liquid Dosage Form
8.6.5 Historic and Forecasted Market Size By Treatment
8.6.5.1 Enzyme Replacement Therapy
8.6.5.2 Hematopoietic Stem Cell Therapy
8.6.5.3 Gene Therapy
8.6.5.4 Others
8.6.6 Historic and Forecasted Market Size By End User
8.6.6.1 Hospital Pharmacies
8.6.6.2 Online Pharmacies
8.6.6.3 Retail Pharmacies
8.6.7 Historic and Forecast Market Size by Country
8.6.7.1 Turkiye
8.6.7.2 Bahrain
8.6.7.3 Kuwait
8.6.7.4 Saudi Arabia
8.6.7.5 Qatar
8.6.7.6 UAE
8.6.7.7 Israel
8.6.7.8 South Africa
8.7. South America Morquio Syndrome (MPS-IV) Drug Market
8.7.1 Key Market Trends, Growth Factors and Opportunities
8.7.2 Top Key Companies
8.7.3 Historic and Forecasted Market Size by Segments
8.7.4 Historic and Forecasted Market Size By Type
8.7.4.1 Solid Dosage Form
8.7.4.2 Liquid Dosage Form
8.7.5 Historic and Forecasted Market Size By Treatment
8.7.5.1 Enzyme Replacement Therapy
8.7.5.2 Hematopoietic Stem Cell Therapy
8.7.5.3 Gene Therapy
8.7.5.4 Others
8.7.6 Historic and Forecasted Market Size By End User
8.7.6.1 Hospital Pharmacies
8.7.6.2 Online Pharmacies
8.7.6.3 Retail Pharmacies
8.7.7 Historic and Forecast Market Size by Country
8.7.7.1 Brazil
8.7.7.2 Argentina
8.7.7.3 Rest of SA
Chapter 9 Analyst Viewpoint and Conclusion
9.1 Recommendations and Concluding Analysis
9.2 Potential Market Strategies
Chapter 10 Research Methodology
10.1 Research Process
10.2 Primary Research
10.3 Secondary Research
Q1: What would be the forecast period in the Morquio Syndrome (MPS-IV) Drug Market research report?
A1: The forecast period in the Morquio Syndrome (MPS-IV) Drug Market research report is 2024-2032.
Q2: Who are the key players in the Morquio Syndrome (MPS-IV) Drug Market?
A2: Abeona Therapeutics (USA), Amicus Therapeutics (USA), BioMarin Pharmaceutical (USA), Denali Therapeutics (USA), Green Cross Corporation (South Korea), JCR Pharmaceuticals (Japan), Lysogene (France), Orchard Therapeutics (UK), Pfizer (USA), RegenxBio (USA), Sanofi (France), Sarepta Therapeutics (USA), Shire (Ireland), Takeda Pharmaceutical (Japan), Ultragenyx Pharmaceutical (USA), and Other Active Players.
Q3: What are the segments of the Morquio Syndrome (MPS-IV) Drug Market?
A3: The Morquio Syndrome (MPS-IV) Drug Market is segmented into Type, Treatment, End User and region. By Type, the market is categorized into Solid Dosage Form, Liquid Dosage Form. By Treatment, the market is categorized into Enzyme Replacement Therapy, Hematopoietic Stem Cell Therapy, Gene Therapy, Others. By End User, the market is categorized into Hospital Pharmacies, Online Pharmacies, Retail Pharmacies. By region, it is analyzed across North America (U.S., Canada, Mexico), Eastern Europe (Russia, Bulgaria, The Czech Republic, Hungary, Poland, Romania, Rest of Eastern Europe), Western Europe (Germany, UK, France, The Netherlands, Italy, Russia, Spain, Rest of Western Europe), Asia Pacific (China, India, Japan, South Korea, Malaysia, Thailand, Vietnam, The Philippines, Australia, New-Zealand, Rest of APAC), Middle East & Africa (Turkiye, Bahrain, Kuwait, Saudi Arabia, Qatar, UAE, Israel, South Africa), South America (Brazil, Argentina, Rest of SA).
Q4: What is the Morquio Syndrome (MPS-IV) Drug Market?
A4: Morquio Syndrome or Mucopolysaccharidosis IV is a genetic disorder in which the deficiency of L-iduronate sulfatase results in the inability of the body to metabolize glycosaminoglycans which consequently leads to skeletal abnormalities, joint issues, and other complications. Management of MPS IV was centered on ERT, supportive and compassionate care, and gene therapy in the pipeline. This market is focused on drugs and treatment for MPS IV patients as well as trying to slow down the progression of the symptoms with an attempt to increase the quality of life of patients with these diseases.
Q5: How big is the Morquio Syndrome (MPS-IV) Drug Market?
A5: Morquio Syndrome (MPS-IV) Drug Market Size Was Valued at USD 1.69 Million in 2023, and is Projected to Reach USD 4.8 Million by 2032, Growing at a CAGR of 12.4% From 2024-2032.
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