United Kingdom Morquio Syndrome (MPS IV) Therapeutics Market 2023 Is Ready to Set Outstanding Growth in Upcoming Years

Report Overview

The UK Morquio Syndrome 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.

Morquio Syndrome (MPS IV) is an ultra-rare autosomal recessive inherited disorder and a member of a group of hereditary metabolic diseases known as the mucopolysaccharidoses (MPS) which, in turn, are part of a larger group of diseases known as lysosomal storage disorders (LSDs). MPS IV is caused by the deficiency of the lysosomal hydrolase, N-acetylglucosamine-6-sulfate sulfatase (GALNS) enzyme. The deficiency of this enzyme accumulates glycosaminoglycans (GAG) such as keratan sulfate (KS) and chondroitin-6-sulfate (C6S) in multiple tissues, mainly bone, cartilage, heart valves, and cornea, leading to devastating skeletal dysplasia with incomplete ossification and successive imbalance of growth.

Patients with MPS IVA typically appear healthy at birth, but gradually develop multi-systemic manifestations that progress over time. MPS IV is an ultra-rare autosomal recessive inherited disorder with a point prevalence ranging between 1/599,000 live births in the UK. The frequency of imaging depends on both the patient's age and clinical condition. Physical growth slows and often stops around age 8. Children with the more severe form of MPS IV may not live beyond their twenties or thirties. On average, female patients lived 4 years longer than male patients.

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 Morquio syndrome is anticipated to drive the growth in the MPS IV treatment market during the forecast period. With the recent approval of the Vimizim (elosulfase alpha), 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.

Increasing incidence of MPS IV syndrome, rising health care industries, and increase in government investment to develop health care infrastructure also drive the market growth. Market players are more focused on the adoption of inorganic growth strategies such as collaborations and partnerships to strengthen their position and expected by key players to drive the market growth over the forecast period.

Market Restraints

The huge price tag associated with Morquio syndrome therapeutics such as Vimizim (elosulfase alfa) approved by NICE, has an exceptionally high price around 395,000 per patient per year making it one of the most expensive drugs in the world, is one of the major factors expected to limit the growth of MPS IV treatment market. Other factors which may restrict the growth of the MPS IV treatment market are the lack of awareness and the regulatory and clinical hurdles.

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Top Major Players

The UK Morquio syndrome therapeutics market provides a great opportunity for market players to enter and now more focused on the adoption of inorganic growth strategies to garner maximum market share and limit the high price tag of the therapeutics. The prominent players in the MPS IV treatment market include BioMarin Pharmaceuticals Inc, Alexion Pharmaceuticals Inc, Sangamo Therapeutics Inc, and Genzyme Corporation among others.

The report delivers the challenges in front of the United Kingdom Morquio Syndrome (MPS IV) Therapeutics Market 2023 that allows a better understanding of the market providing a helpful comprehensive evaluation and clear-cut dynamics associated with this market. The analyst has studied the current market scenario while concentrating on the new business objectives with the lucrative opportunities that are available. This report includes changes in customer requirements, customer preferences, and the vendor landscape in the industry with key factors that are expected to influence the overall dynamics of the United Kingdom Morquio Syndrome (MPS IV) Therapeuticss Market including the current trends, growth opportunities, restraints, and market drivers.

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Market Segmentation

By Therapeutics:

On the basis of therapeutics, the MPS IV therapeutics market is segmented into

Enzyme Replacement Therapies (ERT)
Hematopoietic Stem Cell Transplantation (HSCT)
Substrate Reduction Enzyme Therapy (SERT)
Gene Therapy
Other medications

ERT consists of replacing the deficient or absent enzyme with a functional recombinant version through intravenous administration. Among all therapeutics, ERT accounts for the largest proportion of the market, and the trend is expected to same during the forecast period due to the Vimizim (elosulfase alfa) which is an enzyme replacement therapy, approved by the regulatory authorities in the UK.

By Route of Administration:

Based on the route of administration, the MPS IV therapeutic market can be segmented into

Oral
Intravenous (IV)
Intracerebroventricular (ICV)
Intrathecal
Others

Among others, the intravenous type is anticipated to account for the majority of the revenue share of the route of administration segment. Because the most commonly approved therapeutics, Vimizim (elosulfase alfa) for Morquio syndrome treatment is administered by intravenous injections only, thus anticipated to control over its market share during the forecast period.

By Distribution Channels:

In terms of distribution channels, the MPS IV therapeutic market has been segmented into

Hospital pharmacies
Retail pharmacies
Online pharmacies

The hospital pharmacies segment has accounted for the highest market share in 2020 as the major therapeutics are prescription products. Therefore, hospital pharmacies are further anticipated also to witness growth during the forecast period.

By End Users:

Based on end-user, the MPS IV therapeutic market is segmented into

Hospital
Specialty Clinics
Medical Research Centres
Home-infusion
Others

The key factor aiding in the dominance of the hospital segment is the appropriate and safe administration of critical therapeutics used in MPS IV treatment by trained medical professionals who can administer only in hospitals with care and proper guidelines. Thus, the potential growth of the hospital segment can be anticipated during the forecast period.

Top Major Players

The UK Morquio syndrome therapeutics market provides a great opportunity for market players to enter and now more focused on the adoption of inorganic growth strategies to garner maximum market share and limit the high price tag of the therapeutics. The prominent players in the MPS IV treatment market include BioMarin Pharmaceuticals Inc, Alexion Pharmaceuticals Inc, Sangamo Therapeutics Inc, and Genzyme Corporation among others.

The United Kingdom Morquio Syndrome (MPS IV) Therapeutics Market report is an easy-to-understand document giving section-wise details about the global market. Starting with the brief outline of the overall market, it will put forth the all-inclusive evaluated market statistics and different parameters for the forecast period. The report covers numerous aspects of the United Kingdom Morquio Syndrome (MPS IV) Therapeutics market divided into product kind, application, and end-use, offering the report to assist the customers in improving their ability to make precise decisions related to the business under the United Kingdom Morquio Syndrome (MPS IV) Therapeutics Market.

International Market Players will use the correct market facts and figures and applied math studies provided within the report back to perceive this and future growth of the worldwide Electrophoretic United Kingdom Morquio Syndrome (MPS IV) Therapeutics market. Each section of the research study is specially prepared to explore key aspects of the global United Kingdom Morquio Syndrome (MPS IV) Therapeutics Market. Buyers of the report will have access to accurate PESTLE, SWOT, and other types of analysis on the global United Kingdom Morquio Syndrome (MPS IV) Therapeutics market. Moreover, it offers highly accurate estimations on the CAGR, market share, and market size of key regions and countries.

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Strategic Points Covered in Table of Content of Global United Kingdom Morquio Syndrome (MPS IV) Therapeutics Market:

Chapter 1: Introduction, market driving force product Objective of Study and Research Scope the United Kingdom Morquio Syndrome (MPS IV) Therapeutics market
Chapter 2: Exclusive Summary - the basic information of the United Kingdom Morquio Syndrome (MPS IV) Therapeutics Market.
Chapter 3: Displaying the Market Dynamics- Drivers, Trends and Challenges & Opportunities of the United Kingdom Morquio Syndrome (MPS IV) Therapeutics
Chapter 4: Presenting the United Kingdom Morquio Syndrome (MPS IV) Therapeutics Market Factor Analysis, Porters Five Forces, Supply/Value Chain, PESTEL analysis, Market Entropy, Patent/Trademark Analysis.
Chapter 5: Displaying the by Type, End User and Region/Country
Chapter 6: Evaluating the leading manufacturers of the United Kingdom Morquio Syndrome (MPS IV) Therapeutics market which consists of its Competitive Landscape, Peer Group Analysis, BCG Matrix & Company Profile
Chapter 7: To evaluate the market by segments, by countries and by Manufacturers/Company with revenue share and sales by key countries in these various regions
Chapter 8 & 9: Displaying the Appendix, Methodology and Data Source

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