PUBLISHER: DataM Intelligence | PRODUCT CODE: 1374882
PUBLISHER: DataM Intelligence | PRODUCT CODE: 1374882
Global Morquio Syndrome Therapy Market - 2023-2030
Overview
Morquio syndrome is a infrequent genetic disorder impacting a child's bones and spine, organs, and physical capabilities. Kids with this disorder are deficient or do not produce sufficient amounts of the enzymes that metabolizes sugar chains inherently produced in the body. These chains stockpile in cells, blood, tendons, and ligaments, inducing wear and trear over time. In 2014, the FDA authorized a recombinant human GALNS enzyme replacement therapy (elosulfase alfa, or Vimizim) for the treatment of MPS IVA while other therapy of MPS IV is symptomatic and supportive.
Market Dynamics: Drivers & Restraints
Authorizations From Regulatory Bodies
The new approvals for the existing Morquio Syndrome therapy and increasing support from distinct regulatory authorities are expected to boost the global market growth during the forecast period. For instance, in June 2019, BioMarin Pharmaceutical Inc. obtained the National Medical Products Administration (NMPA) approval for its Vimizim (elosulfase alfa) in the treatment of individuals with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. Vimizim is the first treatment in China approved for this condition.
Moreover, the NICE in March 2022, issued the conclusive draft guidance suggesting elosulfase alfa (also called Vimizin and made by BioMarin) for recurring usage in the NHS for the therapy of mucopolysaccharidosis type 4A (also known as MPS 4A and Morquio A syndrome). Furthermore, the increasing research activities, growing awareness and plrevalence are also expected to boost the global market growth during the forecast period.
High Therapy Cost
The high cost of the therapy is expected to hamper the global market growth during the forecast period. For instance, the cost of elosulfase alfa in few nations is in beyween $2,080,000 to $6,240,000 per year, making it problematic for some health plans to afford it. Furthermore, the low treatment choices, low prevalence and high drug development costs are also expected to hamper the global market growth during the forecast period.
Segment Analysis
The global morquio syndrome therapy market is segmented based on type, therapy, route of administration, distribution channel and region.
MPS IV Type A is Expected to Dominate Market
Owing to the increase in positive research investigation outcomes and high prevalence of MPS IV Type A than MPS IV Type B, it is estimated to dominate the global market during the forecast period. For instance, a team at the Universitat Autonoma de Barcelona (UAB), Spain, has developed the first rat model summarizing all disabling alterations encountered by individuals with Mucopolysaccharidosis type IVA, also termed Morquio A disease. The team has even created a gene therapy that fully restores the extreme whole-body alterations in the rat model after a single intravenous viral vector administration, opening the door to a prospective therapy that might be administered at young ages in patients detected with this rare disease, thereby averting bone malformations, osteoarthritic intricacies, and other life-threatening alterations.
Moreover, according to the National Organization for Rare Disorders, Inc. MPS IVA is detected in 95% of individuals impacted by MPS IV while MPS IVB is detected in around 5% of affected individuals.
Geographical Analysis
North America is Expected to Dominate the Global Market
Owing to the increase research initiatives and funding are expected to boost the North American market, dominating the global market during the forecast period. For instance, in May 2023, as a component of the Foundation for the National Institutes of Health (FNIH) Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC), Nemours Children's Health has been picked to execute a first-of-its-kind gene therapy clinical trial for Morquio A syndrome.
Again, in August 2023, a unique initiative strives to eliminate hindrance, delivering $97 million in funds and expertise to assist investigators in testing gene therapy in individuals with Morquio and seven other ultrarare diseases as soon as next year. The program, a coalition of nonprofits, government, and industry, is directed by the non-profit Foundation for the National Institutes of Health. Furthermore, the growing awareness and prevalence of the condition is also contributing to the regional market growth during the forecast period.
COVID-19 Impact Analysis
Enzyme replacement therapy (ERT) is a common treatment for certain types of lysosomal storage diseases (LSDs), which involves intravenously administering weekly or biweekly doses. ERT has been found to improve respiratory and cardiac function, alleviate organomegaly, improve range of motion, and enhance the overall health-related quality of life for some types of mucopolysaccharidoses (MPS).
However, the global market growth for scheduled ERT was negatively affected due to COVID-19 disruptions. Lysosomal storage diseases are high-risk illnesses because of their involvement with respiratory, renal, and cardiac functions. The COVID-19 pandemic has also disrupted the availability of ERT, which has had a significant impact on the global market growth.
Market Segmentation
By Therapy
- Enzyme Replacement Therapy
- Vimizim (elosulfase alfa)
- Supportive/Symptomatic Therapy
- Painkillers
- Supplements
- Others
- Others
By Type
- MPS IV Type A
- MPS IV Type B
By Route of Administration
- Injectable
- Oral
- Other
By Distribution Channel
- Hospital Pharmacies
- Retail Pharmacies
- Online Pharmacies
- Others
By Region
- North America
- U.S.
- Canada
- Mexico
- Europe
- Germany
- UK
- France
- Italy
- Spain
- Rest of Europe
- South America
- Brazil
- Argentina
- Rest of South America
- Asia-Pacific
- China
- India
- Japan
- Australia
- Rest of Asia-Pacific
- Middle East and Africa
Key Developments
- In March 2022, the National Institute for Health and Care Excellence (NICE) issued conclusive draft guidance recommending the recurring use of elosulfase alfa, also known as Vimizin and produced by BioMarin, for the treatment of mucopolysaccharidosis type 4A (MPS 4A) or Morquio A syndrome in the NHS.
- In 2019, Vimizim (elosulfase alfa) by BioMarin Pharmaceutical Inc. has received approval from the National Medical Products Administration (NMPA) to treat patients with mucopolysaccharidosis type IVA (MPS IVA) or Morquio A syndrome. This marks the first ever approved treatment for this rare condition in China.
Competitive Landscape
The major global players in the market include: BioMarin, JCR Pharmaceuticals Co., Ltd., Novartis AG, Takeda Pharmaceutical Company Limited., Alexion Pharmaceuticals, Inc., Regenxbio Inc., Ultragenyx Pharmaceutical Inc., Abbvie Inc., and Abeona Therapeutics Inc. among others.
Why Purchase the Report?
- To visualize the global morquio syndrome therapy market segmentation based on type, therapy, route of administration, distribution channel and region as well as understand key commercial assets and players.
- Identify commercial opportunities by analyzing trends and co-development.
- Excel data sheet with numerous data points of morquio syndrome therapy market-level with all segments.
- PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
- Product mapping available as excel consisting of key products of all the major players.
The global morquio syndrome therapy market report would provide approximately 69 tables, 67 figures and 192 pages.
Target Audience 2023
- Manufacturers/ Buyers
- Industry Investors/Investment Bankers
- Research Professionals
- Emerging Companies
Table of Contents
1. Methodology and Scope
- 1.1. Research Methodology
- 1.2. Research Objective and Scope of the Report
2. Definition and Overview
3. Executive Summary
- 3.1. Snippet by Type
- 3.2. Snippet by Therapy
- 3.3. Snippet by Route of Administration
- 3.4. Snippet by Distribution Channel
- 3.5. Snippet by Region
4. Dynamics
- 4.1. Impacting Factors
- 4.1.1. Drivers
- 4.1.1.1. Authorizations From Regulatory Bodies
- 4.1.2. Restraints
- 4.1.2.1. High Therapy Cost
- 4.1.3. Opportunity
- 4.1.4. Impact Analysis
- 4.1.1. Drivers
5. Industry Analysis
- 5.1. Porter's Five Force Analysis
- 5.2. Supply Chain Analysis
- 5.3. Pricing Analysis
- 5.4. Regulatory Analysis
- 5.5. Reimbursement Analysis
- 5.6. Patent Analysis
- 5.7. Pipeline Analysis
- 5.8. Epidemiology
- 5.9. SWOT Analysis
- 5.10. DMI Opinion
6. COVID-19 Analysis
- 6.1. Analysis of COVID-19
- 6.1.1. Scenario Before COVID
- 6.1.2. Scenario During COVID
- 6.1.3. Scenario Post COVID
- 6.2. Pricing Dynamics Amid COVID-19
- 6.3. Demand-Supply Spectrum
- 6.4. Government Initiatives Related to the Market During Pandemic
- 6.5. Manufacturers Strategic Initiatives
- 6.6. Conclusion
7. By Type
- 7.1. Introduction
- 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
- 7.1.2. Market Attractiveness Index, By Type
- 7.2. Enzyme Replacement Therapy*
- 7.2.1. Introduction
- 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
- 7.2.3. Vimizim (elosulfase alfa)
- 7.3. Supportive/Symptomatic Therapy
- 7.3.1. Painkillers
- 7.3.2. Supplements
- 7.3.3. Others
- 7.4. Others
8. By Therapy
- 8.1. Introduction
- 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy
- 8.1.2. Market Attractiveness Index, By Therapy
- 8.2. MPS IV Type A*
- 8.2.1. Introduction
- 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
- 8.3. MPS IV Type B
9. By Route of Administration
- 9.1. Introduction
- 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
- 9.1.2. Market Attractiveness Index, By Route of Administration
- 9.2. Injectable*
- 9.2.1. Introduction
- 9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
- 9.3. Oral
- 9.4. Other
10. By Distribution Channel
- 10.1. Introduction
- 10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
- 10.1.2. Market Attractiveness Index, By Distribution Channel
- 10.2. Hospital Pharmacies*
- 10.2.1. Introduction
- 10.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
- 10.3. Retail Pharmacies
- 10.4. Online Pharmacies
- 10.5. Others
11. By Region
- 11.1. Introduction
- 11.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
- 11.1.2. Market Attractiveness Index, By Region
- 11.2. North America
- 11.2.1. Introduction
- 11.2.2. Key Region-Specific Dynamics
- 11.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
- 11.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy
- 11.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
- 11.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
- 11.2.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
- 11.2.7.1. U.S.
- 11.2.7.2. Canada
- 11.2.7.3. Mexico
- 11.3. Europe
- 11.3.1. Introduction
- 11.3.2. Key Region-Specific Dynamics
- 11.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
- 11.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy
- 11.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
- 11.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
- 11.3.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
- 11.3.7.1. Germany
- 11.3.7.2. UK
- 11.3.7.3. France
- 11.3.7.4. Italy
- 11.3.7.5. Spain
- 11.3.7.6. Rest of Europe
- 11.4. South America
- 11.4.1. Introduction
- 11.4.2. Key Region-Specific Dynamics
- 11.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
- 11.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy
- 11.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
- 11.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
- 11.4.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
- 11.4.7.1. Brazil
- 11.4.7.2. Argentina
- 11.4.7.3. Rest of South America
- 11.5. Asia-Pacific
- 11.5.1. Introduction
- 11.5.2. Key Region-Specific Dynamics
- 11.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
- 11.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy
- 11.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
- 11.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
- 11.5.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
- 11.5.7.1. China
- 11.5.7.2. India
- 11.5.7.3. Japan
- 11.5.7.4. Australia
- 11.5.7.5. Rest of Asia-Pacific
- 11.6. Middle East and Africa
- 11.6.1. Introduction
- 11.6.2. Key Region-Specific Dynamics
- 11.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
- 11.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy
- 11.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
- 11.6.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
12. Competitive Landscape
- 12.1. Competitive Scenario
- 12.2. Market Positioning/Share Analysis
- 12.3. Mergers and Acquisitions Analysis
13. Company Profiles
- 13.1. BioMarin*
- 13.1.1. Company Overview
- 13.1.2. Product Portfolio and Description
- 13.1.3. Financial Overview
- 13.1.4. Key Developments
- 13.2. JCR Pharmaceuticals Co., Ltd.
- 13.3. Novartis AG
- 13.4. Takeda Pharmaceutical Company Limited.
- 13.5. Alexion Pharmaceuticals, Inc., Inc.
- 13.6. REGENXBIO INC.
- 13.7. Abeona Therapeutics Inc.
- 13.8. AbbVie Inc.
- 13.9. Ultragenyx Pharmaceutical Inc.
- 13.10. Akero Therapeutics, Inc.
LIST NOT EXHAUSTIVE
14. Appendix
- 14.1. About Us and Services
- 14.2. Contact Us
