Duchenne Muscular Dystrophy (DMD) Therapeutics Market by Type, Distribution Channel, and Geography - Forecast and Analysis 2023-2027

Duchenne Muscular Dystrophy (DMD) Therapeutics Market Research Report 2023-2027

The global Duchenne muscular dystrophy (DMD) therapeutics market size is estimated to grow by USD 2,397.67 million at a CAGR of 21.2% between 2022 and 2027. 

The price of Duchenne muscular dystrophy drugs is significantly high. This is because of a complicated production process and high R&D costs. Various governments have come up with patient assistance programs to reduce the cost burden associated with these therapeutics. The Patient Protection and Affordable Care Act is an initiative by the US government to provide health coverage for people at low cost. Patient assistance programs have also been set up by healthcare companies to help patients cope with the high cost of treatment. The program not only focuses on providing financial assistance but also on educating patients about the benefits of insurance, logistics related to the treatment, and other important information. A large number of manufacturers are also offering patient assistance programs for their main drugs, as a result of expected market entry in the forecast period. These programs are expected to help patients in overcoming the high costs associated with the treatment, which will increase adherence to the treatment and drive the growth of the market during the forecast period.

Technavio has segmented the market into:   

• Type is classified into biologics and small molecules
• Distribution Channel is classified into offline and online
• The geography segment includes key regions such as North America, Europe, Asia, and the Rest of World (ROW)

What will be the size of the Duchenne Muscular Dystrophy (DMD) Therapeutics Market During the Forecast Period?

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Duchenne Muscular Dystrophy (DMD) Therapeutics Market Segmentation by Type, Distribution Channel, and Geography Analysis

Type Analysis 

The market share growth by the biologics segment will be significant during the forecast period.  Biologics are substances derived from living organisms and contain certain components of live organisms such as proteins, monoclonal antibodies, peptides, oligonucleotides, genes, or cells. The biologics segment of the global Duchenne muscular dystrophy (DMD) therapeutics market consists of only one approved antisense oligonucleotide, namely EXONDYS 51 by Sarepta, for the treatment of the indication.

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The biologics segment was valued at USD 401.03 million in 2017. The segment also witnesses the presence of multiple novel biologics, such as cell therapy, gene therapy, and monoclonal antibodies, being developed in the late stages of clinical trials for the indication. Special drug designations from regulatory authorities such as the European Medicines Agency and the US Food and Drug Administration have been obtained for some of the biologics under development. These designations are expected to lead to quicker approvals, thereby adding value to the growth of the Duchenne muscular dystrophy therapeutics market in focus during the forecast period.

Small molecules are synthetically produced organic compounds with the ability to regulate a certain biological process. Small molecules bind to a specific biological macromolecule and either increase its function or alter and inhibit the regulation of a specific target.  Due to the expected launch of a number of medicinal products that are still undergoing evaluation in their final stages of trials, the segment of molecule drugs for Duchenne Muscular Dystrophy is expected to experience strong growth during the forecast period. However, the segment faces a threat from the strong dominance of biologics as a treatment option. Also, treatment using small molecule-based drugs is often associated with adverse effects, which can lead to low patient adherence. The small molecules segment is expected to grow at a lower rate than the biologics segment. 

Distributional Channel Analysis 

Based on distributional channels, the market has been segmented into offline and online. Offline stores include pharmacies and retail stores, such as Walgreen Co. (Walgreen), Walmart Inc.( Walmart), and others from where individuals buy Duchenne muscular dystrophy therapeutics. As a result of their widespread availability, Duchenne muscular dystrophies medications such as prednisone, prednisolone, and deflazacort are available at pharmacies leading to an increase in the sale of Duchenne muscular dystrophies therapeutics which is expected to fuel Duchenne muscular dystrophy (DMD) therapeutics Market growth over the forecast period. The number of people visiting these pharmacies and other stores is increasing significantly as pharmacists provide accurate information to buyers of DMD therapeutics. In spite of increased sales over the last two years, most companies have encountered difficulties in marketing their products at pharmacies for advertising purposes. Moreover, the concept of open shelves in drugstores has enabled consumers to buy products over the counter without a prescription. Therefore, such factors will drive the growth of the offline segment of the market in focus during the forecast period.

The online store segment is the fastest-growing segment in the global Duchenne muscular dystrophy (DMD) therapeutics market. Online platforms such as CVS Pharmacy, Inc. (CVS Pharmacy) provide customers with an option to discuss product reviews and recommendations through online chat rooms and discussion forums. In the last ten years, there has been a rise in sales of products by internet and mobile shopping stores due to an increased Internet penetration rate. As a result of the advantages afforded by privacy and convenience, Duchenne Muscular Dystrophy drug sales via online channels will continue to rise over the forecast period. Several vendors are promoting their products online through social media. They are also focusing on simplifying the online purchase of Duchenne muscular dystrophy (DMD) therapeutics by providing free return policies that allow consumers to return products due to discrepancies or damage. The online retailers focus on delivering Duchenne muscular dystrophy therapeutics at a low price through specific deals.

Regional Analysis

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North America is estimated to contribute 40% to the growth of the global market during the forecast period. Technavio’s analysts have elaborately explained the regional trends and drivers that will shape the market during the forecast period.

The Duchenne muscular dystrophy (DMD) therapeutics market in North America is expected to witness significant growth during the forecast period, primarily owing to the dominance of approved therapeutics in the region. The region is experiencing an increased use of highly effective drugs such as EXONDYS 51 from Sarepta, EMFLAZA, and TRANSLNAR by PTC Therapeutics. Due to the rapidly progressive and fatal nature of Duchenne muscular dystrophy, governments in countries such as the US and Canada have added the indication to the list of diseases that are required to be screened in newborns. This results in an increased incidence of the indication.

COVID Recovery Analysis

The COVID-19 outbreak led to a slight decline in the growth of the DMD therapeutics market in North America in 2020, owing to the temporary pause in the research and development of new drugs for Duchenne muscular dystrophy treatment and the decline in clinic visits by DMD patients because of the stay-at-home policies. But the region has seen market activities and revenue increase, thanks to the adoption of a telehealth policy in 2021 as well as its withdrawal from Stay at Home policies. In addition, the return to normal business and commercial activities and the speeding up of research programs for Duchenne muscular dystrophy (DMD) therapeutics are expected to drive the growth of the regional market during the forecast period.

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Key Duchenne Muscular Dystrophy (DMD) Therapeutics Market Players 

Vendors are implementing various strategies, such as strategic alliances, partnerships, mergers and acquisitions, geographical expansion, and product/service launches, to enhance their presence in the market. The report also includes detailed analyses of the competitive landscape of the market and information about 20 market vendors, including:

BioMarin Pharmaceutical Inc. - The company offers copper products under the brand Hindalco.

• Capricor Therapeutics Inc.
• CRISPR Therapeutics AG
• CVS Health Corp.
• Daiichi Sankyo Co. Ltd.
• Editas Medicine Inc.
• Eli Lilly and Co.
• F. Hoffmann La Roche Ltd.
• FibroGen Inc.
• Italfarmaco Holding SPA
• Nippon Shinyaku Co. Ltd.
• Otsuka Holdings Co. Ltd.
• Pfizer Inc.
• PTC Therapeutics Inc.
• Santhera Pharmaceuticals Holding AG

Qualitative and quantitative analysis of vendors has been conducted to help clients understand the wider business environment as well as the strengths and weaknesses of key market players. Data is qualitatively analyzed to categorize vendors as pure play, category-focused, industry-focused, and diversified; it is quantitatively analyzed to categorize vendors as dominant, leading, strong, tentative, and weak.

Duchenne Muscular Dystrophy (DMD) Therapeutics Market: Key Drivers, Trends, Challenges, and Customer Landscape

There are multiple factors influencing market growth. Our researchers analyzed the data with 2022 as the base year, along with the key drivers, trends, and challenges. 

Key Duchenne Muscular Dystrophy (DMD) Therapeutics Market Drivers

The high unmet need among patients with Duchenne muscular dystrophy (DMD) is a driving trend in Duchenne muscular dystrophy (DMD). Muscular dystrophies, which affect human skeletal muscles, are clinically and genetically heterogeneous disorders.  Duchenne muscular dystrophy is a hereditary childhood myopathy, which leads to progressive muscle weakening and atrophy, and premature death. At present, pharmacological therapy for Duchenne muscular dystrophy (DMD) is predominantly based on symptomatic treatment. The discovery of the dystrophin gene, which codes proteins that contribute to maintaining membrane stability in muscle cells, has been brought about by increased research efforts aimed at understanding the etiology of Duchenne Muscular Dystrophy.

Moreover, the knowledge about disease progress, etiology, and development of effective treatment for Duchenne Muscular Dystrophy was greatly improved through animal models with natural mutations or those that are caused by gene targeting. EXONDYS 51 and TRANSLARNA also provide only symptomatic relief to patients without curing the disease. As a result, despite recent approvals, the global market for Duchenne Muscular DystrophyTherapeutics represents a high unmet need for affordable treatments that can reverse the development of Duchenne Muscular Dystrophy. This creates a significant opportunity for vendors to enter the global Duchenne muscular dystrophy (DMD) therapeutics market to cater to the high unmet demand.

Significant Duchenne Muscular Dystrophy (DMD) Therapeutics Market Trends

The special drug designation for Duchenne muscular dystrophy (DMD) therapeutics is a primary trend in Duchenne muscular dystrophy (DMD).  One of the main benefits that pharmaceutical companies have been able to obtain with their therapeutic products for the treatment of Duchenne Muscular Dystrophy (DMD) is special drug designations granted by regulators. Many therapeutic candidates have received designations such as Orphan drug designation from regulatory bodies such as the US FDA and the EMA. There are regulatory and tax benefits available to vendors that have been granted orphan drug designation for their medicinal products. They also receive a waiver of marketing application user fees as well as market exclusivity for seven years.

Similarly, breakthrough therapy designation is granted by the US FDA to drugs that display superior efficacy and substantial treatment advantages over the drugs available in the market for the treatment of a certain indication. Such a designation makes it easier for suppliers to obtain priority review status in comparison with other drugs being evaluated. The faster approval of therapeutic use is a result of the specific designation of medicinal products on the market. This is expected to drive the growth of the global DMD therapeutics market in the coming years.

Major Duchenne Muscular Dystrophy (DMD) Therapeutics Market Challenges

The high cost of DMD therapeutics is a major challenge in Duchenne muscular dystrophy. Duchenne muscular dystrophy is a rare and fatal genetic disorder that primarily affects the male population and causes muscle weakness in early childhood.  It can also lead to premature death in adults due to respiratory and heart failure. It is a progressive muscle disorder caused by the deficiency of functional dystrophin protein. The essential component of proteins required to maintain the structural stability of skeletal muscle, including cardiac muscle, is dystrophin. Individuals with Duchenne muscular dystrophy lose their ability to walk by the age of ten years, followed by paralysis of the arms. This imposes a high disease burden on patients and their caregivers. The disease requires lifelong treatment with Duchenne muscular dystrophy therapeutics.

Despite the high efficacy of Duchenne muscular dystrophy therapeutics, the high cost of therapies imposes an economic burden on patients and their families. Due to the high cost of treatment, there will be a decrease in patients' adherence and an impact on therapeutic volumes and value for Duchenne Muscular Dystrophy. This is a major challenge for the global Duchenne muscular dystrophy (DMD) therapeutics market.

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Key Duchenne Muscular Dystrophy (DMD) Therapeutics Market Customer Landscape

The market report includes the adoption lifecycle of the market, covering from the innovator’s stage to the laggard’s stage. It focuses on adoption rates in different regions based on penetration. Furthermore, the report also includes key purchase criteria and drivers of price sensitivity to help companies evaluate and develop their growth strategies.

Global Duchenne Muscular Dystrophy (DMD) Therapeutics Market Customer Landscape

Segment Overview

The Duchenne muscular dystrophy (DMD) therapeutics market report forecasts market growth by revenue at global, regional & country levels and provides an analysis of the latest trends and growth opportunities from 2017 to 2027. 

• Type Outlook (USD Million, 2017 - 2027)
  • Biologics
  • Small molecules
  • Private

• Distribution Channel Outlook (USD Million, 2017 - 2027)
  • Offline
  • Online

• Region Outlook (USD Million, 2017 - 2027)
  • North America
    • The U.S.
    • Canada
  • Europe
    • The U.K.
    • Germany
    • France
    • Rest of Europe
  • Asia
    • China
    • India
  • ROW
    • Australia
    • Saudi Arabia
    • Brazil

Duchenne Muscular Dystrophy (DMD) Therapeutics Market Scope
Report Coverage	Details
Page number	154
Base year	2022
Historic period	2017-2021
Forecast period	2023-2027
Growth momentum & CAGR	Accelerate at a CAGR of 21.2%
Market growth 2023-2027	USD 2397.67 million
Market structure	Fragmented
YoY growth 2022-2023(%)	19.51
Regional analysis	North America, Europe, Asia, and the Rest of the World (ROW)
Performing market contribution	North America at 40%
Key countries	US, Canada, Germany, UK, and China
Competitive landscape	Leading Vendors, Market Positioning of Vendors, Competitive Strategies, and Industry Risks
Key companies profiled	BioMarin Pharmaceutical Inc., Capricor Therapeutics Inc., CRISPR Therapeutics AG, CVS Health Corp., Daiichi Sankyo Co. Ltd., Editas Medicine Inc., Eli Lilly and Co., F. Hoffmann La Roche Ltd., FibroGen Inc., Italfarmaco Holding SPA, Nippon Shinyaku Co. Ltd., Otsuka Holdings Co. Ltd., Pfizer Inc., PTC Therapeutics Inc., Santhera Pharmaceuticals Holding AG, Sarepta Therapeutics Inc, Solid Biosciences Inc., The Cleveland Clinic Foundation, Vertex Pharmaceuticals Inc., and Wave Life Sciences Ltd.
Market dynamics	Parent market analysis, Market growth inducers and obstacles, Fast-growing and slow-growing segment analysis, COVID-19 impact and recovery analysis and future consumer dynamics, and Market condition analysis for the forecast period.
Customization purview	If our report has not included the data that you are looking for, you can reach out to our analysts and get segments customized.

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What are the Key Data Covered in this Duchenne Muscular Dystrophy (DMD) Therapeutics Market Research Report?

• CAGR of the market during the forecast period
• Detailed information on factors that will drive the growth of the Duchenne muscular dystrophy (DMD) market between 2023 and 2027
• Precise estimation of the size of the market in focus on the parent market
• Detailed analysis of customer landscape covering Diver of price sensitivity, Adoption of the lifecycle, and key purchase criteria 
• Vendor landscape comparing criticality of inputs and factors of differentiation
• Exclusive Matrix on vendor position and Classification
• Accurate predictions about upcoming trends and changes in consumer behavior
• Growth of the market industry across North America, Europe, Asia, and the Rest of the World (ROW)
• A thorough analysis of the market’s competitive landscape and detailed information about vendors
• Comprehensive analysis of factors that will challenge the growth of Duchenne muscular dystrophy (DMD) therapeutics market vendors

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