Global Duchenne Muscular Dystrophy Drugs Market Analysis


  • Type: Rare Disease Area
  • Published : March 2021
  • Region: Worldwide
  • Country: Other
  • Report ID : 396

  • Format: PPT, PDF



Report Overview

The global Duchenne Muscular Dystrophy (DMD) drugs market size stood at USD 1,566 million in 2019 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

MPS-IV, refers to a rare inherited metabolic disorder or birth defect that mostly affects the skeleton. The FDA approval of VIMIZIM (elosulfase alpha), the first and the only specific treatment for the patients with this ultra-rare genetic condition, firmly established BioMarin’s position as a leading market player for the treatment of MPS diseases. Apart from the recent regulatory approvals, there has been increasing activity in the R&D for the Morquio syndrome treatment as evidenced by the greater inclusion of the disorder in the clinical pipelines of the major market players. For instance, Ultragenyx Pharmaceutical Inc. has a Phase 3 Study undergoing for the Morquio syndrome, Shire has an ongoing clinical trial for the pediatric patients suffering from the syndrome and ArmaGen, Inc. also has a drug (AGT-181) in the clinical trials. This combined with the greater awareness of the disease is also driving the growth in Morquio syndrome treatment market.

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 rare diseases is anticipated to drive the growth in the Morquio syndrome treatment market at a considerably fast rate. With the recent approval of the elosulfase alpha (VIMIZIM), 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. Market players are more focused on adoption of inorganic growth strategies such as collaborations and partnerships to strengthen their position in the global mucopolysaccharidosis (MPS) treatment market. Adoption of inorganic growth strategies by key players is expected to drive the market growth over the forecast period.

  1. Global Duchenne Muscular Dystrophy (DMD) Market Overview…………………
    1. Market Size
  2. Market Growth Drivers………………………………………………………………….
    1. Rise in Global Prevalence of Duchenne Muscular Dystrophy (DMD)
    2. Growing R&D Investments and Reimbursement Policies
  3. Major Duchenne Muscular Dystrophy (DMD)
    1. Congenital
    2. Genetic
  4. Duchenne Muscular Dystrophy (DMD) Market Segmentation……
    1. Drug Class
      1. Corticosteroids
      2. Others
    2. Distribution Channel
      1. Hospitals Pharmacies
      2. Online Pharmacies
      3. Others
    3. Geography
      1. North America Market Analysis
      2. Europe Market Analysis
      3. Asia-Pacific Market Analysis
      4. Middle-East and Africa Market Analysis
  5. Duchenne Muscular Dystrophy (DMD) Major Drugs Market Share…
    1. Key Findings
    2. Market Analysis, Insights and Forecast – By Drug Type
  6. Competitive Landscape…………………………………………………………………
    1. Major Players
    2. Products in Pipeline
  7. Key Company Profiles…………………………………………………………………..
    1. FibroGen, Inc., Overview, Product & Services, Strategies & Financials
    2. Summit Therapeutics plc, Overview, Product & Services, Strategies & Financials
    3. Santhera Pharmaceuticals Product & Services, Strategies & Financials
  8. Healthcare Policies and Regulatory Landscape……………………………………
    1. Policy changes and Reimbursement scenario
  9. Factors Driving Future Growth…………………………………………………………
    1. New Trends and Development of Duchenne Muscular Dystrophy (DMD) Market
    2. Future Opportunities
  10. Conclusion

Market Segmentation

By Drug Class:

Corticosteroids are the only class of drugs with proven efficacy in improving muscle strength in the case of DMD. Corticosteroids, such as deflazacort (Emflaza), have demonstrated delayed nonambulation; however, adverse effects of steroid therapy impede their adoption.

By Disease Indication:

By disease indication, DMD is inherited in an X-linked recessive pattern and may occur in people who do not have a family history of DMD. Duchenne muscular dystrophy (DMD) is also inherited in an X-linked recessive pattern. DMD is the most common childhood onset form of muscular dystrophy and affects males almost exclusively.

By Distribution Channel:

In terms of distribution channel, chronic inflammatory demyelinating polyneuropathy market have been segmented into specialty neurological clinics, hospitals, research & academic laboratories, and others. Hospitals have amassed most share of the global market due to their widespread presence and the preference for hospitals among patients. However, patients are increasingly seeing the value in specialty clinics where they may understand their illness better.

By Region:

The United States accounts for the largest market size of Duchenne Muscular Dystrophy, in comparison to EU5 (the United Kingdom, Germany, Italy, France, and Spain), and Japan. Among the EU5 countries, France had the highest market size, while Spain had the lowest market size of Duchenne Muscular Dystrophy (DMD). The increasing awareness of the disease assisted by organizational support along with the promising pipeline therapies is expected to fuel the market size during the forecasted period of 2019-2028.

Top 5 Major Players

The global Duchenne Muscular Dystrophy (DMD) Market is highly competitive, and the prominent players in the market have adopted various strategies to garner maximum market share. These include collaboration, product launch, partnership, and acquisition. Major players operating in the market include FibroGen, Inc., Summit Therapeutics plc, PTC Therapeutics, Santhera Pharmaceuticals, and Sarepta 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.

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