UK MPS VI Market Analysis

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  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Europe
  • Country: UK
  • Report ID : 2588

  • Format: PPT, PDF

UK Mucopolysaccharidosis VI (MPS VI) Therapeutics Market: Segmented by Therapeutics, Route of Administration, Distribution Channels, and End Users – Size, Share, Impact, Growth, Trends, and Forecasts (2020 – 2028)

Published Date: June 2021

 

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SKU: 2588 Categories: ,

Report Overview

The UK Mucopolysaccharidosis VI Therapeutics market size stood at around USD xx billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

Mucopolysaccharidosis VI (MPS VI), also known as Maroteaux-Lamy syndrome is a member of a group of hereditary metabolic diseases known as the mucopolysaccharidoses which, in turn, are part of a larger group of diseases known as lysosomal storage disorders (LSDs). MPS VI is a rare genetic disorder characterized by a complete or partial lack of activity of the enzyme arylsulfatase B (also called N-acetylgalactosamine-4-sulfatase), encoded by the ARSB gene. MPS VI occurs due to mutations in the ARSB gene and is inherited as an autosomal recessive disorder. The deficiency or absence of this enzyme activity leads to the accumulation of complex carbohydrates called glycosaminoglycans (previously known as mucopolysaccharides) in the body. Abnormal accumulation of mucopolysaccharides leads to progressive involvement of multiple organ systems.

The symptoms and severity of Maroteaux-Lamy syndrome can vary dramatically from one person to another; some individuals only develop mild symptoms, while others develop severe, even life-threatening complications. Common symptoms can include coarse facial features, corneal clouding, joint abnormalities, various skeletal malformations, an abnormally enlarged liver and/or spleen (hepatosplenomegaly), and hearing loss. It is estimated that nearly 6% of the UK population (around 3.5 million people) will be affected by a rare disease at some point in their lives, and the MPS VI had the highest birth prevalence of 7.85 per 100,000 live births, comprising 46% of all MPS cases diagnosed.

Initial diagnosis is usually made between 6 and 24 months of age, and life expectancy depends on the severity of symptoms and is around 20 to 30 years. People with MPS VI often begin to show signs and symptoms of MPS VI during early childhood, these include changes to the physical appearance as well as developing other clinical features in the cardiovascular and respiratory systems. Importantly, MPS VI does not affect intelligence.

Market Growth Drivers

The growth in the number of products in the preliminary and advanced stages of R&D along with the focus on the treatment of Maroteaux-Lamy syndrome is anticipated to drive the growth in the MPS VI treatment market at a considerably fast rate. With the approval of BioMarin’s Naglazyme (galsulfase), the market is expected to be boosted several products currently in the pipeline of various companies can be anticipated to undergo regulatory approvals for the marketing, if they have favorable clinical trials results.

The increasing incidence and prevalence also boost the MPS VI treatment market growth during the forecast period. Market players are more focused on the adoption of inorganic growth strategies such as collaborations and partnerships to strengthen their position in the MPS VI treatment market. The adoption of inorganic growth strategies by key players is expected to drive the market growth over the forecast period.

Market Restraints

However, rising treatment costs and a lack of awareness among patients and physicians about MPS VI are among the major factors acting as restraints and will continue to pose a challenge to the MPS VI treatment market in the forecast period.

TABLE OF CONTENTS

1. UK Mucopolysaccharidosis VI Therapeutics Market Overview…………
A. Market Size
2. Market Growth Drivers and Restraints……………………………………….…
Market Growth Drivers
A. Robust Products Pipeline & Introduction of Novel Therapies
B. Rising Government Initiatives and Increased R&D Activities
C. Rise in Prevalence and Incidence of Mucopolysaccharidosis VI
Market Restraints
A. Rising Treatment Costs
B. Lack of Awareness
3. Major Types of Mucopolysaccharidosis VI………………………………………….
A. Macrocephaly
B. Hydrocephaly
C. Others
4. Mucopolysaccharidosis VI Therapeutics Market Segmentation………………..
A. By Therapeutics
I. Enzyme Replacement Therapy (ERP)
II. Haematopoietic Stem Cell Transplantation (HSCT)
III. Gene Therapy
IV. Others
B. By Route of Administration
I. Oral
II. Intravenous
III. Others
C. By Distribution Channels
I. Hospital Pharmacies
II. Retail Pharmacies
III. Online Pharmacies
D. By End Users
I. Hospitals
II. Speciality Clinics
III. Medical Research Centres
IV. Home Infusion
V. Others
5. Mucopolysaccharidosis VI Major Drugs Market Share………………………
A. Market Analysis, Insights and Forecast – By Revenue Type
6. Competitive Landscape…………………………………………………………………
A. Major Players
B. Products in Pipeline
7. Key Company Profiles………………………………………………………………….
A. BioMarin Company overview, Product & Services, Strategies & Financials
B. Inventiva Company overview, Product & Services, Strategies & Financials
C. Genzyme Company overview, Product & Services, Strategies & Financials
8. Healthcare Policies and Regulatory Landscape……………………………………
A. Policy changes and Reimbursement scenario
9. Factors Driving Future Growth…………………………………………………………
A. New Trends and Development of Mucopolysaccharidosis VI Therapeutics Market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Therapeutics:

On the basis of therapeutics, the MPS VI therapeutics market can be segmented into

  • Enzyme Replacement Therapies (ERT)
  • Hematopoietic Stem Cell Transplantation (HSCT)
  • Gene Therapy
  • Others

ERT consists of replacing the deficient or absent enzyme with a functional recombinant version through intravenous administration. At present, enzyme replacement therapy accounts for the largest proportion of the market, and the trend is expected to remain the same during the forecast period.

By Route of Administration:

Based on the route of administration, the MPS VI therapeutics market can be segmented into

  • Oral
  • Intravenous (IV)
  • Others

The intravenous type is anticipated to account for the majority of the revenue share of the route of administration segment. Because Naglazyme (galsulfase) is the most preferred therapeutic which is given as an intravenous infusion. Therefore, further projected to control its market share during the forecast period.

By Distribution Channels:

In terms of distribution channels, the MPS VI therapeutics market can be bifurcated into

  • Hospital pharmacies
  • Retail pharmacies
  • Online pharmacies

The hospitals have amassed the most share of the market due to their widespread presence and the preference for hospitals among patients. Thus, the hospital segment is anticipated for robust growth during the forecast period.

By End Users:

Based on end-user, the MPS VI therapeutics market can be segmented into

  • Hospitals
  • Specialty Clinics
  • Medical Research Centres
  • Home-infusion
  • Others

Naglazyme (galsulfase) is the best solution for Maroteaux-Lamy syndrome which is given as a weekly infusion lasting about 4 hours that can be administered at home. Thus, the potential growth of home infusion can be anticipated during the forecast period.

Top Major Players

The UK MPS VI therapeutics market is highly competitive, as many of the players are well engaged to deliver the best therapeutic solution for the treatment of Maroteaux-Lamy syndrome. The prominent players in the market have adopted various strategies to garner maximum market share. BioMarin’s Naglazyme (galsulfase) has been approved as the enzyme replacement therapy for the treatment of Maroteaux-Lamy syndrome. Thus, the key players are anticipated to have lucrative growth opportunities in the future with the rising demand for MPS VI treatment. Major players operating in the MPS VI treatment market include BioMarin Pharmaceuticals Inc, Inventiva Pharma, Fondazione Telethon, and Genzyme Corporation among others.

Research Methodology

Insights10 will provide you reports within 10 key parameters which are:

  1. Market overview
  2. Market growth drivers & restraints
  3. Epidemiology or disease type
  4. Market segmentation
  5. Market share
  6. Competitive landscape
  7. Key company profiles
  8. Healthcare policies & regulatory framework
  9. Reimbursement scenario
  10. Factors driving future growth

Based on our many years of experience, we believe that these are the parameters that are critical to decision making for business stakeholders. Our focused approach to develop reports focused on 10 key parameters, enabled us to arrive at the name “Insights10”.

Our analysis methodology involves three critical stages:

Stage I: Market Data Collection

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The top-down approach mainly used for new product forecasting and bottom-up approach is used for demand estimation of any product for different countries summed up to form total market. We are able to round off insights and build stronger forecasts because we always do both these methods and triangulate the final numbers.

The study on the market covers the analysis of the leading geographies such as North America, Europe, Asia-Pacific, and Africa for the period of 2020 to 2028. The qualitative analysis covers the industry landscape and trends, market opportunities, competitive landscape and policy and regulatory scenario and the quantitative analysis covers different market estimates and forecasts.

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We make sure to finalize the numbers in alignment to the market research. Firstly, our internal experts ensure thorough validation and checking to ensure the accurate and precise analysis and then validation is also done using multiple data analysis model.  Two level validation is done and entails finalization of report scope and to way of representation pattern.

Stage III: Interpretation and Presentation

Analysis & Interpretation: The information gathered is then analysed and synthesized. A second series of interviews are done if necessary to check and validate. The future opportunities are analysed by understanding product commercialization and many other factors. It also comprises the analysis of data discrepancies observed across various data sources. Information procured from secondary and primary results are then, interpreted by considering following parameters: (a partial list)

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