Australia Morquio Syndrome (MPS-IV) Market Analysis

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  • Type: Rare Disease Area
  • Published : April 2021
  • Region: APAC
  • Country: Australia
  • Report ID : 4087

  • Format: PPT, PDF

Australian Morquio Syndrome (MPS IV) Therapeutics Market: Segmented by Therapeutics, Route of Administration, Distribution Channel, and End Users – Size, Share, Impact, Growth, Trends, and Forecasts (2020 – 2028F)

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Report Overview

The Australian Morquio Syndrome (MPS IV) 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 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 Australian. 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.

Market Drivers and Restraints

Market Drivers:

The growth in the number of products in the preliminary and advanced stages of R&D along with the focus on treatment of Morquio Syndrome is anticipated to drive the growth in the MPS IV Therapeutics 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 favourable clinical trials results.

Increasing incidence of Morquio 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 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:

Huge price tag associated with Morquio Syndrome treatment therapeutics such as Vimizim (elosulfase alfa) has an exceptionally high price, 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 Therapeutics market. Other factors which may restrict the growth of MPS IV Therapeutics market is the lack of awareness and the regulatory and clinical hurdles.

 

1. Australian Morquio Syndrome Therapeutics Market Overview………….……..
A. Market Size
2. Market Growth Drivers and Restraints……………………………………….……..
A. Market Drivers
I. Robust Products Pipeline & Introduction of Novel Therapies
II. Adoption of Inorganic Growth Strategies by Prominent Players
III. Rise in Prevalence and Incidence of Australian Syndrome
B. Market Restraints
I. High cost of treatment and Regulatory & Clinical Hurdles
3. Major Types of Morquio Syndrome………………………………………………….
A. Morquio A Syndrome (MPS IVA)
B. Morquio B Syndrome (MPS IVB)
4. Morquio Syndrome Therapeutics Market Segmentation……………………………..
A. By Therapeutics
I. Enzyme Replacement Therapy (ERP)
II. Haematopoietic Stem Cell Transplantation (HSCT)
III. Substrate Reduction Enzyme Therapy (SERT)
IV. Gene Therapy
V. Other Medications
B. By Route of Administration
I. Oral
II. Intravenous
III. Intracerebroventicular (ICV)
IV. Intrathecal
V. Others
C. By Distribution Channel
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. Morquio Syndrome Major Products 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. Alexion 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 Morquio Syndrome Therapeutics Market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Therapeutics:

On the basis of therapeutics, the Morquio Syndrome Therapeutics market is segmented into enzyme replacement therapies (ERT), haematopoietic stem cell transplantation (HSCT), substrate reduction enzyme therapy (SERT), gene therapy and others medications. ERT consists of replacing the deficient or absent enzyme with a functional recombinant version through intravenous administration. Among all therapies, 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 Australian.

By Route of Administration:

Based on route of administration, the Morquio Syndrome Therapeutics 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 therapy, 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 Morquio Syndrome Therapeutics market has been segmented into hospital pharmacies, retail pharmacies, and 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 Morquio Syndrome Therapeutics market is segmented into hospital, specialty clinics, medical research centres, home-infusion and others. The key factor aiding in the dominance of 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, potential growth of hospital segment can be anticipated during the forecast period.

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”.

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Market Sizing and Analysis: Our expert data analytics team has created various market forecast models by employing the top-down approach i.e. start with large overall market and segment different areas and bottom-up approach i.e. start with population and epidemiology and roll up based on spend, etc., estimating the size of the market, and distributing among the geographic and/or product segments.

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)

  • Establishing market drivers and trends
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  • Market Segment based analysis to obtain revenue / volume
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Data curated from the analysis and interpretation are drawn to portray all in one consolidated report.

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The foreign exchange rates are calculated on the respective dates and for the respective regions covered in the study.

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