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The Duchenne muscular dystrophy therapeutics market size is estimated to grow by USD 4.37 billion at a CAGR of 28.21% between 2023 and 2028. The Duchenne muscular dystrophy (DMD) market is experiencing significant expansion due to several key drivers. Firstly, the substantial unmet medical need among DMD patients is fueling the demand for innovative and effective therapies like RNA-based therapeutics. Secondly, strategic alliances among industry players are essential for advancing research and development of healthcare services. These collaborations often result in groundbreaking treatments and enhanced patient outcomes. Moreover, patient assistance programs play a crucial role in increasing therapy accessibility by mitigating the high costs associated with DMD treatment. Furthermore, advancements in healthcare infrastructure and support systems facilitate the delivery of care, collectively addressing critical needs, fostering innovation, and improving accessibility. These factors collectively contribute to the growth of the DMD market.
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The Duchenne muscular dystrophy therapeutics market research report provides comprehensive data (region-wise segment analysis), with forecasts and estimates in "USD Billion" for the period 2024 to 2028, as well as historical data from 2018 to 2022 for the following segments.
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 market consists of only one approved antisense oligonucleotide for the treatment of the indication.
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The biologics segment was valued at USD 480.10 million in 2018. 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 market in focus during the forecast period.
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North America is estimated to contribute 39% to the growth of the global Duchenne muscular dystrophy therapeutics 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 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. 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. Hence, such factors are fuelling the growth of this segment during the forecast period.
The high unmet need among patients with Duchenne muscular dystrophy (DMD) is a key factor driving Duchenne muscular dystrophy therapeutics market growth. 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. As a result, despite recent approvals, the global market represents a high unmet need for affordable treatments that can reverse the development of Duchenne muscular dystrophy. This creates a significant opportunity for companies to enter the global market to cater to the high unmet demand. Hence, such factors are driving the market during the forecast period.
The special drug designation for Duchenne muscular dystrophy (DMD) therapeutics is a primary trend in the market. 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 companies 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 market in the coming years. Hence, such factors are driving the market during the forecast period.
The high cost of DMD therapeutics is a major challenge impacting Duchenne muscular dystrophy therapeutics market growth. 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 market during the forecast period.
The Duchenne muscular dystrophy therapeutics market forecasting 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 Duchenne muscular dystrophy therapeutics market growth analysis report also includes key purchase criteria and drivers of price sensitivity to help companies evaluate and develop their market growth and forecasting strategies.
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Customer Landscape
Companies 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 key companies, including:
BioMarin Pharmaceutical Inc. - The company offers DMD therapeutics such as BMN 351.
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., The Cleveland Clinic Foundation, Vertex Pharmaceuticals Inc., and Wave Life Sciences Ltd.
Qualitative and quantitative analysis of companies 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 companies as pure play, category-focused, industry-focused, and diversified; it is quantitatively analyzed to categorize companies as dominant, leading, strong, tentative, and weak.
The Duchenne muscular dystrophy (DMD) therapeutics market in the US is experiencing notable advancements and progress. Parent Project Muscular Dystrophy (PPMD), a leading advocacy and research organization, has been instrumental in driving innovation in this field. Pharmaceutical companies have introduced groundbreaking treatments, such as tabrecta and elamipretide, for managing DMD symptoms. Gene therapy, including fordadistrogene movaparvovec and micro-dystrophin gene therapy programs, are making strides in targeting specific mutations related to DMD. Additionally, translarna (ataluren) and exon skipping drugs represent mutation-targeted therapies aimed at addressing the underlying genetic causes of DMD. Steroid therapy remains a standard treatment for managing symptoms in ambulatory DMD patients.
Despite these advancements, challenges persist in the DMD therapeutics market. Regulatory hurdles and the absence of standardized protocols impede the approval and widespread adoption of novel treatments. The high cost of DMD treatment continues to be a significant concern for patients and healthcare providers. Factors influencing the market include nonsense mutations, the molecular-based therapies segment, small molecules, and the high cost of DMD treatment. Collaborations between pharmaceutical companies, research organizations, and academic institutions are vital for addressing these challenges and advancing progress in DMD therapeutics.
Moreover, the market is witnessing significant gene therapy advancements and mutation-specific therapies. The emergence of the exon-skipping platform and drugs like eteplirsen is expanding treatment possibilities for this progressive disorder. However, challenges such as the lack of standardized protocols and high costs remain. The Food and Drug Administration (FDA) plays a crucial role in approving these innovative therapies. The DMD treatment market is influenced by factors like hospital pharmacies and healthcare infrastructure. Investment pockets in regions like Portland, Oregon are essential for driving progress. Strategic business decisions are imperative to navigate the complexities of this genetic disease and expand therapeutic options for individuals with Duchenne muscular dystrophy.
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Market Scope |
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Report Coverage |
Details |
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Page number |
167 |
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Base year |
2023 |
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Historic period |
2018-2022 |
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Forecast period |
2024-2028 |
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Growth momentum & CAGR |
Accelerate at a CAGR of 28.21% |
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Market growth 2024-2028 |
USD 4.37 billion |
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Market structure |
Fragmented |
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YoY growth 2023-2024(%) |
21.6 |
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Regional analysis |
North America, Europe, Asia, and Rest of World (ROW) |
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Performing market contribution |
North America at 39% |
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Key countries |
US, Germany, UK, and Canada |
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Competitive landscape |
Leading Companies, Market Positioning of Companies, Competitive Strategies, and Industry Risks |
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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., The Cleveland Clinic Foundation, Vertex Pharmaceuticals Inc., and Wave Life Sciences Ltd. |
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Market dynamics |
Parent market growth analysis, Market forecasting, Market growth inducers and obstacles, Fast-growing and slow-growing segment analysis, COVID-19 impact and recovery analysis and future consumer dynamics, Market condition analysis for forecast period. |
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Customization purview |
If our Duchenne muscular dystrophy therapeutics market forecast 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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1 Executive Summary
2 Market Landscape
3 Market Sizing
4 Historic Market Size
5 Five Forces Analysis
6 Market Segmentation by Type
7 Market Segmentation by Distribution Channel
8 Customer Landscape
9 Geographic Landscape
10 Drivers, Challenges, and Opportunity/Restraints
11 Competitive Landscape
12 Competitive Analysis
13 Appendix
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