UK X-Linked Myotubular Myopathy (XLMTM) Market Analysis

$3,500.00$7,500.00

  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Europe
  • Country: UK
  • Report ID : 2592

  • Format: PPT, PDF

UK X-linked Myotubular Myopathy (XLMTM) Therapeutics Market: Segmented by Diagnosis, Therapeutics, and Distribution Channels – Growth, Trends, and Forecasts (2020 – 2028)

Published Date: June 2021

Clear
SKU: 2592 Categories: ,

Report Overview

The UK X-Linked Myotubular Myopathy (XLMTM) 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.

The X-linked myotubular myopathy (XLMTM) is the most common form of centronuclear myopathy (CNM). The XLMTM is a rare genetic neuromuscular disorder that is characterized by muscle weakness that is most typically severe but can range from mild to profound. Symptoms are often present at birth, though may develop later in infancy or early childhood. Rarely, symptoms may not present until adolescence or adulthood. Common symptoms include mild to profound muscle weakness, diminished muscle tone (hypotonia or “floppiness”), feeding difficulties, and potentially severe breathing complications (respiratory distress). Feeding difficulties and respiratory distress develop because of the weakness of the muscles that are involved in swallowing and breathing.

The XLMTM is caused by mutations to the myotubularin (MTM1) gene. The disorder is inherited as an X-linked condition. The disorder predominantly affects males, but female carriers, while typically asymptomatic, can develop a range of symptoms. According to a study conducted in the UK, it is estimated that the incidence of 17 per million births for severe X-linked myotubular myopathy (XLMTM) has been published in a report.

Market Growth Drivers

The rise in incidence and prevalence of X-Linked Myotubular Myopathy (XLMTM) is likely to propel market growth. Moreover, huge investment in research & development by key players is anticipated to drive the market growth over the forecast period. Several players are investing in the development of products/therapies like Gene therapy that is being studied as an approach to therapy for XLMTM. In gene therapy, the defective gene present in a patient is replaced with a normal gene to enable the production of the active enzyme and prevent the development and progression of the disease.

Furthermore, the introduction of new products by key players is anticipated to drive the X-Linked Myotubular Myopathy (XLMTM) market. The emergence of new therapies for acute phase or prevention of X-Linked Myotubular Myopathy (XLMTM) creates lucrative opportunities for the market over the forecast period.

Market Restraints

However, rising treatment costs and lack of awareness among patients and physicians about X-Linked Myotubular Myopathy are among the major factors acting as restraints and will continue to pose a challenge to the X-Linked Myotubular Myopathy treatment market over the forecast period.

TABLE OF CONTENTS

1. UK X-Linked Myotubular Myopathy Therapeutics Market Overview………..
A. Market Size
2. Market Growth Drivers & Restraints…………………………………………………….
Market Growth Drivers
A. Increasing Incidence and Prevalence of X-Linked Myotubular Myopathy
B. Huge Investments in Research & Development Activities
C. Emergence of Novel Therapies for X-Linked Myotubular Myopathy Treatment
Market Restraints
A. Rising Treatment Costs
B. Lack of Awareness
3. Major Types of X-Linked Myotubular Myopathy…………………………………………
A. Nemaline Myopathy
B. Central Core Myopathy
C. Congenital Myopathy
D. Minimulticore Myopathy
E. Centronuclear Myopathy
4. UK X-Linked Myotubular Myopathy Therapeutics Market Segmentation…………………………………
A. By Diagnosis
I. Molecular Genetic Testing
II. Surgical Removal
III. Muscle Biopsy
IV. Specialised Tests
V. Others
B. By Therapeutics
I. Physical Therapy
II. Occupational Therapy
III. Non-steroidal Anti-inflammatory Drugs (NSAIDs)
IV. Corticosteroids
V. Gene Therapy
VI. Protein Replacement Therapy
VII. Genetic Counselling
VIII. Others
C. By Distribution Channel
I. Hospital Pharmacies
II. Retail Pharmacies
III. Online Pharmacies
5. UK X-Linked Myotubular Myopathy Major Products Market Share……….…
A. Market Analysis, Insights and Forecast – By Revenue
6. Competitive Landscape…………………………………………………………………
A. Major Players
B. Products in Pipeline
7. Key Company Profiles…………………………………………………………………
A. Audentes 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 XLMTM therapeutics market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Diagnosis:

Based on the diagnosis, the XLMTM treatment market can be bifurcated into

  • Molecular genetic testing
  • Surgical removal
  • Muscle biopsy
  • Specialized tests
  • Others

The diagnosis is based upon the identification of additional characteristic symptoms (e.g., cryptorchidism, long fingers and toes, macrocephaly), a detailed family history, a thorough clinical evaluation, and a variety of specialized tests. The diagnosis of XLMTM is confirmed through molecular genetic testing, which can detect mutations in the MTM1 gene causative of the disorder. Molecular genetic testing can detect a mutation in approximately 60%-98% of affected individuals and is available on a clinical basis.

By Therapeutics:

On the basis of therapeutics, the XLMTM therapeutics market can be categorized into

  • Physical therapy
  • Occupational therapy
  • Non-steroidal anti-inflammatory drugs (NSAIDs)
  • Corticosteroids
  • Gene therapy
  • Protein replacement therapy
  • Genetic counseling
  • Others

The treatment of affected individuals usually requires intensive medical intervention. Some affected individuals will require prolonged, constant ventilation support. In gene therapy, the defective gene present in a patient is replaced with a normal gene to enable the production of the active enzyme and prevent the development and progression of the disease. Better market growth can be projected over the forecast period.

By Distribution Channel:

In terms of distribution channels, the XLMTM therapeutics market can be classified into

  • Hospital pharmacies
  • Retail pharmacies
  • Online pharmacies

An increasing number of people are being hospitalized for X-Linked Myotubular Myopathy. Therefore, hospital pharmacies are likely to be the largest distribution channel during the forecast period, as the drugs for X-Linked Myotubular Myopathy are being sold on a large scale through the hospital pharmacies.

Top Major Players

The X-Linked Myotubular Myopathy (XLMTM) therapeutics market is an emerging market, and the prominent players in the market are likely to adopt organic as well as inorganic strategies to garner maximum market share. These include collaboration, product launch, partnership, and acquisition. Major Key player operating in the UK market includes Audentes Therapeutics Inc.

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

Primary Interviews: We have developed a network of experts, freelancers and researchers across countries through which we engage with local experts to gather key data points and assumptions around each market. We also engage regularly with some of the best market research agencies such as Atheneum, GuidePoint, GLG, etc. to conduct surveys and interviews, and build intelligence. We have language translators as a part of our team, who between them can cover 30+ languages allowing us to extract better local insights.

Secondary Data Collection: We have developed strong expertise and experience in secondary data collection methods for developing unique data sets and research material. We gather data from multiple reliable sources to maintain the high level of accuracy and consistency. The market data is analysed and forecasted using appropriate statistical and coherent models. The report offers an overall analysis of the market size, growth, market share as well as segment-level analysis of the specific market. Our report includes precise, to the point information related to the overall market, competition, growth drivers, challenges, regulatory updates, and competition.

Data Sources: We have access to multiple highly reliable free and subscription data sources. We have many years of experience to understand which sources are more dependable for what and which to prefer for the reliable and latest information. The key sources of information include the following, but are not limited to:

  • Statistical Databases
  • Company Websites/Annual Reports
  • Trade Publications
  • Online Databases
  • Published Research Reports
  • Whitepapers
  • Press Releases of Key Market Players

Stage II: Market Data Analysis and Statistical Model

Market Trends: We generally look at macro parameter and micro indicators. The macro parameters includes changes in government policies, demand and supply of the market, government intervention programs and major market share. The micro indicators GDP growth, market size, market volume etc. We also understand nuances specific to each country like US, Canada, India, Germany, etc. and have worked across 60+ countries and hence no only understand global trends, but how these differ by country, how payment models, market structure, cultural parameters, etc. differ in each country.

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.

Data Triangulation & Validation:

Data triangulation of various sources and results of the research are carried out by benchmarking with reliable sources such as industry statistics, statistical databases and the company level averages etc.

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
  • Analyzing regulatory landscape to understand future growth
  • Market Segment based analysis to obtain revenue / volume
  • Analyzing current needs and determining penetration to estimate market

Insights: Our reports deliver actionable insights backed with supporting facts and figures to assist you in achieving exemplary growth. Our in-depth analyses are interspersed with relevant insights and statistics to offer an executive-level view of a given market. The description helps in correlating many minor factors affecting the market and its impact to the different segments within the market.
Data curated from the analysis and interpretation are drawn to portray all in one consolidated report.

Presentation & Reporting: The market research report is presented in different forms such as charts by using a scientific approach for easy understanding. Historic, current and future analysis is provided for each market in terms of both value and volume. The size of the market is interpreted in the US Dollar value and the respective unit, based on the product, for volume consumptions.
The foreign exchange rates are calculated on the respective dates and for the respective regions covered in the study.

To request a free sample copy this report, please complete the form below.

    We value your inquiry and offer free customization with every report to fulfil your exact research needs.

    This website is secure and your personal details are safe.



    This report addresses

    • Market intelligence to enable effective decision making
    • Growth opportunities and trend analysis
    • Competitive benchmarking
    • Historical data & forecasts
    • Regional opportunities

    Need more?

    • Speak to our analyst to know how this research was put together
    • Add more countries or regions as part of customisation
    • Understand how this report can have direct impact on your revenue

    Get a custom report based on your requirements