The global Duchenne Muscular Dystrophy (DMD) Drugs market size was valued at US$ 465 million in 2020 and is anticipated to grow at a CAGR of 44.3% during forecast period 2021 to 2030.
The disease is the most common form of muscular dystrophy affecting 16 to 20 infants per 100,000 live births.
DMD is caused due to absence or deformity in the DMD gene that codes for dystrophin - a key protein for maintaining muscular integrity. The condition primarily affects males, as it is an X-linked genetic disorder. Females carrying the mutation in the dystrophin gene are usually asymptomatic or show mild symptoms.
The market is projected to exhibit strong growth through the forecast period. The growth can be majorly attributed to factors such as the emergence of mutation-specific therapies, rising incidence of the condition, and improved diagnostics. Government initiatives supporting target-specific treatments and favorable reimbursement policies are expected to stoke the growth of the market. Furthermore, growing acceptance of targeted therapies, such as Exondys51, Translarna, and Emflaza, is likely to boost the therapeutic space.
The high cost of therapeutics remains an impediment to the growth of the DMD drugs landscape. However, affordable healthcare measures are being adopted by governments across all major regions, impacting the pricing strategies of companies as well as reimbursement scenario. Rising cost consciousness is poised to hinder premium pricing opportunities for upcoming DMD drugs.
On the basis of the therapeutic approach, the market is divided into mutation suppression, exon skipping, and steroid therapy. An estimated 15.0% of all DMD cases result from nonsense mutations, leading to a truncated protein. Drugs such as ataluren (Translarna) bypass nonsense mutations. Mutation-suppressive drugs tend to prolong ambulation and have fewer adverse effects than steroids.
The exon-skipping approach is anticipated to register the steepest growth among all therapeutic methods. Nearly 50.0% and 90.0% of patients suffering from DMD mutations can benefit from single-exon skipping and multi-exon skipping, respectively. Sarepta’s Exondys51 is currently the only approved exon-skipping drug for DMD. Increasing adoption of this drug class and the launch of pipeline drugs are expected to fuel growth prospects.
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 impedes their adoption.
Among the seven major markets, the U.S. dominated the DMD drugs market in 2017. The U.S. is expected to continue leading the market until 2023. High incidence of DMD in the U.S., booming treatment rate, and anticipated launch of pipeline products are some of the factors propelling the market in the country.
The EU5 has limited options for DMD as Exondys51 and Emflaza have not been approved in the region. Translarna remains the only treatment for nonsense mutation DMD patients who are in ambulatory state and are 5 years or older. The drug received conditional approval in Europe in 2014 and was labeled as an orphan medicinal product by the European Medicines Agency.
Japan is likely to witness the fastest growth among all the seven major markets during the forecast period. Currently, Prednisolone (corticosteroid) is the only approved drug for DMD. Japan’s 2014 guidelines for DMD recognized steroid therapy as the only treatment for muscle degeneration in DMD. However, the launch of pipeline drugs is poised to boost the development of other therapeutic approaches.
Sarepta, PTC, Santhera, Italfarmaco, and Catabasis. PTC and Sarepta
Therapeutic Approach Outlook
The Duchenne Muscular Dystrophy (DMD) Drugs market research report covers definition, classification, product classification, product application, development trend, product technology, competitive landscape, industrial chain structure, industry overview, national policy and planning analysis of the industry, the latest dynamic analysis, etc., and also includes major. The study includes drivers and restraints of the global market. It covers the impact of these drivers and restraints on the demand during the forecast period. The report also highlights opportunities in the market at the global level.
The report provides size (in terms of volume and value) of Duchenne Muscular Dystrophy (DMD) Drugs market for the base year 2020 and the forecast between 2021 and 2030. Market numbers have been estimated based on form and application. Market size and forecast for each application segment have been provided for the global and regional market.
This report focuses on the global Duchenne Muscular Dystrophy (DMD) Drugs market status, future forecast, growth opportunity, key market and key players. The study objectives are to present the Duchenne Muscular Dystrophy (DMD) Drugs market development in United States, Europe and China.
It is pertinent to consider that in a volatile global economy, we haven’t just conducted Duchenne Muscular Dystrophy (DMD) Drugs market forecasts in terms of CAGR, but also studied the market based on key parameters, including Year-on-Year (Y-o-Y) growth, to comprehend the certainty of the market and to find and present the lucrative opportunities in market.
In terms of production side, this report researches the Duchenne Muscular Dystrophy (DMD) Drugs capacity, production, value, ex-factory price, growth rate, market share for major manufacturers, regions (or countries) and type.
In terms of consumption side, this report focuses on the consumption of Duchenne Muscular Dystrophy (DMD) Drugs by regions (countries) and application.
Buyers of the report will have access to verified market figures, including global market size in terms of revenue and volume. As part of production analysis, the authors of the report have provided reliable estimations and calculations for global revenue and volume by Type segment of the global Duchenne Muscular Dystrophy (DMD) Drugs market. These figures have been provided in terms of both revenue and volume for the period 2017 to 2030. Additionally, the report provides accurate figures for production by region in terms of revenue as well as volume for the same period. The report also includes production capacity statistics for the same period.
With regard to production bases and technologies, the research in this report covers the production time, base distribution, technical parameters, research and development trends, technology sources, and sources of raw materials of major Duchenne Muscular Dystrophy (DMD) Drugs market companies.
Regarding the analysis of the industry chain, the research of this report covers the raw materials and equipment of Duchenne Muscular Dystrophy (DMD) Drugs market upstream, downstream customers, marketing channels, industry development trends and investment strategy recommendations. The more specific analysis also includes the main application areas of market and consumption, major regions and Consumption, major Chinese producers, distributors, raw material suppliers, equipment providers and their contact information, industry chain relationship analysis.
The research in this report also includes product parameters, production process, cost structure, and data information classified by region, technology and application. Finally, the paper model new project SWOT analysis and investment feasibility study of the case model.
Overall, this is an in-depth research report specifically for the Duchenne Muscular Dystrophy (DMD) Drugs industry. The research center uses an objective and fair way to conduct an in-depth analysis of the development trend of the industry, providing support and evidence for customer competition analysis, development planning, and investment decision-making. In the course of operation, the project has received support and assistance from technicians and marketing personnel in various links of the industry chain.
The Duchenne Muscular Dystrophy (DMD) Drugs market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, production capacities, company strengths and weaknesses, product launch, product width and breadth, application dominance. The above data points provided are only related to the companies’ focus related to Duchenne Muscular Dystrophy (DMD) Drugs market.
Prominent players in the market are predicted to face tough competition from the new entrants. However, some of the key players are targeting to acquire the startup companies in order to maintain their dominance in the global market. For a detailed analysis of key companies, their strengths, weaknesses, threats, and opportunities are measured in the report by using industry-standard tools such as the SWOT analysis. Regional coverage of key companies is covered in the report to measure their dominance. Key manufacturers of Duchenne Muscular Dystrophy (DMD) Drugs market are focusing on introducing new products to meet the needs of the patrons. The feasibility of new products is also measured by using industry-standard tools.
Key companies are increasing their investments in research and development activities for the discovery of new products. There has also been a rise in the government funding for the introduction of new Duchenne Muscular Dystrophy (DMD) Drugs market. These factors have benefited the growth of the global market for Duchenne Muscular Dystrophy (DMD) Drugs. Going forward, key companies are predicted to benefit from the new product launches and the adoption of technological advancements. Technical advancements have benefited many industries and the global industry is not an exception.
New product launches and the expansion of already existing business are predicted to benefit the key players in maintaining their dominance in the global market for Duchenne Muscular Dystrophy (DMD) Drugs. The global market is segmented on the basis of region, application, en-users and product type. Based on region, the market is divided into North America, Europe, Asia-Pacific, Latin America and Middle East and Africa (MEA).
In this study, the years considered to estimate the market size of Duchenne Muscular Dystrophy (DMD) Drugs are as follows:
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- Market segmentation analysis including qualitative and quantitative research incorporating the impact of economic and policy aspects
- Regional and country level analysis integrating the demand and supply forces that are influencing the growth of the market.
- Market value USD Million and volume Units Million data for each segment and sub-segment
- Competitive landscape involving the market share of major players, along with the new projects and strategies adopted by players in the past five years
- Comprehensive company profiles covering the product offerings, key financial information, recent developments, SWOT analysis, and strategies employed by the major market players
In-depth interviews and discussions were conducted with several key market participants and opinion leaders to compile the research report.
This research study involved the extensive usage of both primary and secondary data sources. The research process involved the study of various factors affecting the industry, including the government policy, market environment, competitive landscape, historical data, present trends in the market, technological innovation, upcoming technologies and the technical progress in related industry, and market risks, opportunities, market barriers and challenges. The following illustrative figure shows the market research methodology applied in this report.
Market Size Estimation
Top-down and bottom-up approaches are used to estimate and validate the global market size for company, regional division, product type and application (end users).
The market estimations in this report are based on the selling price (excluding any discounts provided by the manufacturer, distributor, wholesaler or traders). Market share analysis, assigned to each of the segments and regions are achieved through product utilization rate and average selling price.
Major manufacturers & their revenues, percentage splits, market shares, growth rates and breakdowns of the product markets are determined through secondary sources and verified through the primary sources.
All possible factors that influence the markets included in this research study have been accounted for, viewed in extensive detail, verified through primary research, and analyzed to get the final quantitative and qualitative data. The market size for top-level markets and sub-segments is normalized, and the effect of inflation, economic downturns, and regulatory & policy changes or others factors are accounted for in the market forecast. This data is combined and added with detailed inputs and analysis from Vision Research Reports and presented in this report.
Market Breakdown and Data Triangulation
After complete market engineering with calculations for market statistics; market size estimations; market forecasting; market breakdown; and data triangulation. Extensive primary research was conducted to gather information and verify and validate the critical numbers arrived at. In the complete market engineering process, both top-down and bottom-up approaches were extensively used, along with several data triangulation methods, to perform market estimation and market forecasting for the overall market segments and sub-segments listed in this report.
Secondary Sources occupies approximately 25% of data sources, such as press releases, annual reports, Non-Profit organizations, industry associations, governmental agencies and customs data, and so on. This research study includes secondary sources; directories; databases such as Bloomberg Business, Wind Info, Hoovers, Factiva (Dow Jones & Company), TRADING ECONOMICS, and avention; Investing News Network; statista; Federal Reserve Economic Data; annual reports; investor presentations; and SEC filings of companies.
In the primary research process, various sources from both the supply and demand sides were interviewed to obtain qualitative and quantitative information for this report. The primary sources from the supply side include product manufacturers (and their competitors), opinion leaders, industry experts, research institutions, distributors, dealer and traders, as well as the raw materials suppliers and producers, etc.
The primary sources from the demand side include industry experts such as business leaders, marketing and sales directors, technology and innovation directors, supply chain executive, end users (product buyers), and related key executives from various key companies and organizations operating in the global market.
The study objectives of this report are:
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