듀셴형 근이영양증 치료제 시장 : 약물 종류별, 유통 경로별, 최종사용자별, 제형별, 투여 경로별 - 세계 예측(2025-2032년)

The Duchenne Muscular Dystrophy Drugs Market is projected to grow by USD 3.36 billion at a CAGR of 8.87% by 2032.

Setting the stage for Duchenne muscular dystrophy therapeutic strategies by summarizing scientific progress, clinical priorities, and stakeholder imperatives across the ecosystem

Duchenne muscular dystrophy remains one of the most clinically and commercially complex rare disease areas, characterized by progressive muscle degeneration, substantial caregiver burden, and a persistent need for disease-modifying therapies. Scientific advances over the past decade have shifted the field beyond symptomatic care toward targeted molecular interventions, creating a dense ecosystem of therapeutic modalities, regulatory interactions, and payer engagement strategies. Clinicians, families, and policymakers increasingly demand evidence not only of biological activity, but of meaningful functional benefit, long-term safety, and sustainable access models.

Against this backdrop, industry participants face a landscape that requires integrated thinking across discovery, clinical development, manufacturing, and commercialization. Emerging modalities such as exon skipping, gene replacement strategies using AAV vectors, and refined corticosteroid regimens each carry distinct scientific rationales, regulatory considerations, and operational footprints. Moreover, as precision medicine techniques refine patient selection and outcome measurement, stakeholders must balance innovation with scalable manufacturing and distribution approaches. This executive summary synthesizes those dimensions, providing context for strategic decisions and highlighting levers that can accelerate translation from pipeline to patient impact.

Unprecedented scientific, manufacturing, and value-based care shifts are reshaping clinical development, distribution, and reimbursement strategies for Duchenne therapeutics

The past several years have produced transformative shifts that are redefining how Duchenne therapies are developed and delivered, with consequences for trial design, manufacturing, and long-term care models. Scientific innovations have enabled more precise targeting of the genetic defects underpinning the disease, prompting a move away from one-size-fits-all approaches toward modality-specific development paths that require bespoke regulatory and manufacturing strategies. Concurrently, measurement science has matured: novel functional endpoints, digital biomarkers, and patient-reported outcomes are being integrated into pivotal development programs to capture clinically meaningful benefit in smaller, more heterogeneous populations.

Operationally, the industry is experiencing a parallel transition. Gene-based therapeutics reliant on AAV vectors are driving urgent investments in vector manufacturing capacity, quality control, and long-term safety monitoring systems. The choice of serotype, such as AAV6 or AAV9, now influences distribution kinetics, tissue tropism, and manufacturing complexity, which in turn affects clinical strategy and commercial viability. At the same time, exon skipping agents and optimized corticosteroid approaches continue to be refined, offering differentiated profiles in terms of administration, monitoring, and patient populations. These technological and procedural shifts are fostering new partnerships between biotech innovators, contract manufacturers, and specialty pharmacies, while also prompting payers and health systems to explore outcomes-based reimbursement and risk-sharing models to manage high upfront costs with uncertain long-term benefit trajectories.

Taken together, these shifts demand that industry leaders integrate scientific, manufacturing, and commercial planning earlier in development to de-risk late-stage programs and align stakeholder expectations around real-world evidence collection and patient access.

Assessing how anticipated United States tariff adjustments in 2025 may influence supply chains, manufacturing localization choices, and commercial access strategies for Duchenne therapies

Trade policy changes and tariff considerations have become material factors in strategic planning for biopharma supply chains, and proposed tariff adjustments in the United States during 2025 represent an additional variable for companies operating in the Duchenne therapeutic space. Raw materials for biologics production, including plasmid DNA, specialized reagents, single-use systems, and AAV manufacturing inputs, often cross borders multiple times during the manufacturing lifecycle. Consequently, tariffs or customs reclassifications can increase landed costs, extend lead times, and create incentives to re-evaluate supplier footprints.

In response, organizations are reassessing near-term sourcing strategies and exploring diversification to mitigate exposure. Some are accelerating investments in localized production capacity or entering long-term supply agreements to lock predictable pricing and prioritize capacity allocation. These strategic choices have knock-on effects: onshoring or regionalizing production can reduce exposure to import duties but may require significant capital expenditure and time to qualify new manufacturing sites under stringent regulatory frameworks. Moreover, increased import costs can influence pricing discussions with payers and government purchasers, intensifying pressure to demonstrate durable clinical benefit and cost-effectiveness.

Importantly, higher cross-border costs also elevate the value of supply chain transparency and inventory optimization, prompting firms to adopt more sophisticated forecasting, demand-sensing, and buffer stock policies. Regulatory and customs compliance teams will need to engage earlier in project planning to avoid unexpected classification changes that trigger tariffs. Overall, while tariffs are only one of many commercial variables, their cumulative impact can shape manufacturing strategies, partner selection, and the economics of bringing advanced Duchenne therapies to diverse markets.

Deriving practical commercial and clinical implications by integrating drug class, distribution channel, end-user context, form factors, and administration routes into segmentation-driven strategy

Segmentation analysis reveals distinct strategic implications when therapies are examined through multiple clinical and commercial lenses. When viewed by drug class, exon skipping agents, gene therapy approaches, and steroid regimens each demand unique clinical development paradigms and evidence packages; within gene therapy, the division between AAV-based and nonviral modalities affects vector selection, immunogenicity management, and manufacturing pathway decisions, while further consideration of AAV serotype choices such as serotype 6 and serotype 9 drives tissue targeting, dosing strategy, and production scale complexity. Examining distribution channels highlights the varying logistical and compliance requirements across hospital pharmacies, online pharmacies, and retail pharmacies, with each channel posing different cold-chain, reimbursement capture, and patient support needs. From an end-user perspective, the demands of home healthcare settings contrast sharply with hospital and specialty clinic environments, affecting training, monitoring cadence, and emergency preparedness for infusion-related events. Form and route of administration are central to adoption and patient adherence; capsules and oral tablets enable different patient journeys than injectables or intravenous administrations, and intramuscular, intravenous, and oral routes each carry specific clinical monitoring and administration infrastructure requirements.

Synthesizing these segmentation axes underscores that portfolio design cannot be siloed. For example, a gene replacement candidate using AAV9 intended for hospital-based intravenous administration will require different commercialization investments than an orally administered steroid therapy distributed through retail pharmacies for home use. Therefore, go-to-market planning must align clinical designs with downstream distribution, payer engagement, and patient-support services early in development. This integrated perspective enables tailored value propositions that reflect real-world use cases, reduce adoption friction, and support compelling evidence generation strategies for regulators and payers alike.

Evaluating regional regulatory, reimbursement, and operational nuances across the Americas, Europe Middle East & Africa, and Asia-Pacific to inform phased market entry and access strategies

Regional dynamics will shape the deployment and access pathways for Duchenne therapies, and each geography presents distinct regulatory, reimbursement, and operational considerations. In the Americas, there is a mature network of specialized centers, established rare disease clinical trial infrastructure, and payers that emphasize health technology assessment and value-based arrangements, which collectively influence the sequencing of approvals and commercial launches. In Europe, Middle East & Africa, regulatory pathways are diverse and reimbursement decision-making frequently involves national authorities with differing expectations for comparative effectiveness evidence, necessitating early alignment on heterogenous evidence strategies and localized health economic modeling. The Asia-Pacific region presents rapidly evolving clinical research capacity, a growing appetite for industry partnerships, and varied manufacturing ecosystems; however, market access strategies must account for divergent regulatory requirements, pricing controls, and patient access programs.

Consequently, regional strategies should prioritize flexible regulatory pathways, phased market entry plans, and partnerships that leverage local clinical expertise and distribution networks. Cross-border logistical planning is particularly important for advanced therapies with cold-chain or clinic-based administration needs, and regional manufacturing hubs can mitigate tariff or import vulnerabilities while supporting faster patient access. Ultimately, understanding these regional nuances enables more precise stakeholder engagement and increases the likelihood of early adoption by aligning product delivery models with local healthcare delivery architectures.

Analyzing how company strategies around vector engineering, manufacturing scale-up, and stakeholder engagement determine clinical progression and commercial readiness in Duchenne therapeutics

Company-level dynamics are pivotal in shaping the pace and direction of innovation in Duchenne therapeutics. Biotech firms focusing on exon skipping and small-molecule approaches continue to refine payload chemistry, delivery optimization, and patient-selection criteria to enhance clinical differentiation. Organizations pursuing gene replacement therapies are concentrating on vector engineering, serotype selection, and scalable GMP manufacturing to reduce batch-to-batch variability and support consistent clinical supply. Across the industry, strategic alliances-whether through research collaborations, licensing deals, or manufacturing partnerships-are frequently used to bridge capability gaps and accelerate time to clinic.

Investor and corporate priorities are also evolving: there is greater emphasis on operational execution, late-stage safety monitoring plans, and commercial readiness in parallel with clinical milestones. Companies with integrated strategies that align clinical development with manufacturing scale-up and payer engagement tend to navigate late-stage inflection points more effectively. In addition, patient advocacy groups and clinician networks are powerful conveners, influencing trial recruitment, endpoint selection, and post-approval evidence generation, so companies that invest in transparent stakeholder engagement and patient support infrastructure can strengthen both trial enrollment and long-term uptake. Finally, organizations that proactively address manufacturing capacity, supply chain resilience, and regulatory pathways are better positioned to translate R&D successes into accessible therapies for patients.

Adopt integrated actions linking early manufacturing planning, adaptive evidence generation, supply diversification, and patient-centered services to de-risk development and accelerate access

Industry leaders should adopt a coordinated, risk-balanced approach that links scientific strategy with manufacturing and market access planning to accelerate sustainable patient impact. Firstly, integrating manufacturing considerations into early clinical design decisions reduces the likelihood of late-stage supply constraints and enables clearer discussions with regulators about comparability and long-term follow-up. Secondly, firms should prioritize adaptive evidence generation that combines robust clinical endpoints with pragmatic real-world data collection to meet the needs of regulators and payers while supporting iterative product improvements. Thirdly, diversifying supplier networks and exploring regional manufacturing hubs can mitigate tariff and cross-border risks, while long-term capacity agreements with experienced biologics manufacturers can secure critical inputs.

Furthermore, investment in patient-centric services and digital monitoring platforms can improve adherence, capture meaningful outcomes, and strengthen value discussions with payers. Engaging patient advocacy groups early and transparently augments recruitment and aligns development programs with daily life improvements that matter to families. Lastly, leaders should pursue strategic partnerships-spanning research institutions, contract manufacturers, and specialty distribution channels-to fill capability gaps and accelerate time to broader access. Implementing these actions in concert will create a more resilient, evidence-driven approach to bringing transformative Duchenne therapies to patients.

Describe the mixed-methods analytical approach combining expert interviews, regulatory reviews, and supply chain mapping to validate strategic insights without relying on numerical market sizing

The research underpinning this executive synthesis utilized a mixed-methods approach to capture scientific, operational, and commercial dimensions relevant to Duchenne therapeutic development. Primary research included structured interviews with clinical experts, manufacturing specialists, payer advisors, and patient advocacy representatives to validate modality-specific challenges and evidence expectations. Secondary research synthesized peer-reviewed literature, regulatory guidance documents, clinical trial registries, and publicly disclosed corporate filings to map technology trajectories, safety considerations, and trial endpoint evolution. Cross-validation steps compared insights from interviews with documentary evidence to ensure consistency and reduce bias.

Analytical methods emphasized qualitative triangulation and scenario-based risk assessment rather than quantitative market sizing. Special attention was given to supply chain mapping for biologics manufacturing and to evaluating the interplay between serotype selection and clinical strategy for gene therapies. Wherever applicable, regulatory precedent and published long-term follow-up frameworks informed the assessment of post-market evidence needs. Limitations include the evolving nature of clinical data and policy environments; consequently, readers are advised to consider these findings alongside emerging trial results and regulatory decisions as they become available.

Synthesize strategic priorities and execution imperatives that will determine whether scientific advances in Duchenne therapies translate into durable clinical benefit and broad patient access

In conclusion, the Duchenne therapeutic landscape is at a pivotal juncture where scientific innovation, manufacturing realities, and payer expectations converge to shape which therapies reach patients and how they are adopted in practice. Progress in exon skipping, gene therapy vectors, and optimized steroid regimens offers real potential to alter disease trajectories, but realizing that potential depends on synchronized planning across R&D, manufacturing, and commercial teams. Strategic investments in scalable manufacturing, early engagement with regulators and payers, comprehensive real-world evidence plans, and robust patient support models will be decisive in translating clinical promise into sustainable patient access.

As stakeholders navigate tariff policies, regional regulatory heterogeneity, and evolving evidence standards, flexibility and proactive stakeholder alignment will differentiate organizations that can rapidly convert scientific breakthroughs into durable clinical and commercial outcomes. The path forward requires integrated execution, transparent engagement with patient communities, and adaptive evidence strategies that prioritize measured benefit for patients while addressing the long-term sustainability of therapeutic access.

Table of Contents

1. Preface

• 1.1. Objectives of the Study
• 1.2. Market Segmentation & Coverage
• 1.3. Years Considered for the Study
• 1.4. Currency & Pricing
• 1.5. Language
• 1.6. Stakeholders

2. Research Methodology

3. Executive Summary

4. Market Overview

5. Market Insights

• 5.1. Expansion of AAV-based micro-dystrophin gene therapy trials in pediatric patients
• 5.2. Acceleration of CRISPR-Cas9 genome editing programs targeting exon 51 mutations
• 5.3. Increasing regulatory acceptance of surrogate endpoints in Duchenne drug approvals
• 5.4. Emerging collaboration between biotech innovators and patient advocacy groups for trial design
• 5.5. Development of multiplex exon-skipping therapies customized to individual dystrophin gene mutations
• 5.6. Growing focus on combination regimens integrating anti-inflammatory and muscle regeneration agents
• 5.7. Adoption of digital health platforms for remote monitoring of functional outcomes in DMD trials
• 5.8. Advancements in next-generation AAV vectors for enhanced muscle tissue tropism and safety
• 5.9. Rising investments in stem cell therapy approaches to restore dystrophin-producing myocytes
• 5.10. Emergence of multi-country registries capturing real-world evidence on DMD treatment outcomes

6. Cumulative Impact of United States Tariffs 2025

7. Cumulative Impact of Artificial Intelligence 2025

8. Duchenne Muscular Dystrophy Drugs Market, by Drug Class

• 8.1. Exon Skipping
• 8.2. Gene Therapy
  • 8.2.1. Aav Based
    • 8.2.1.1. Serotype 6
    • 8.2.1.2. Serotype 9
  • 8.2.2. Nonviral
• 8.3. Steroid Therapy

9. Duchenne Muscular Dystrophy Drugs Market, by Distribution Channel

• 9.1. Hospital Pharmacies
• 9.2. Online Pharmacies
• 9.3. Retail Pharmacies

10. Duchenne Muscular Dystrophy Drugs Market, by End User

• 10.1. Home Healthcare Settings
• 10.2. Hospitals
• 10.3. Specialty Clinics

11. Duchenne Muscular Dystrophy Drugs Market, by Form

• 11.1. Capsule
• 11.2. Injectable
• 11.3. Oral Tablet

12. Duchenne Muscular Dystrophy Drugs Market, by Route Of Administration

• 12.1. Intramuscular
• 12.2. Intravenous
• 12.3. Oral

13. Duchenne Muscular Dystrophy Drugs Market, by Region

• 13.1. Americas
  • 13.1.1. North America
  • 13.1.2. Latin America
• 13.2. Europe, Middle East & Africa
  • 13.2.1. Europe
  • 13.2.2. Middle East
  • 13.2.3. Africa
• 13.3. Asia-Pacific

14. Duchenne Muscular Dystrophy Drugs Market, by Group

• 14.1. ASEAN
• 14.2. GCC
• 14.3. European Union
• 14.4. BRICS
• 14.5. G7
• 14.6. NATO

15. Duchenne Muscular Dystrophy Drugs Market, by Country

• 15.1. United States
• 15.2. Canada
• 15.3. Mexico
• 15.4. Brazil
• 15.5. United Kingdom
• 15.6. Germany
• 15.7. France
• 15.8. Russia
• 15.9. Italy
• 15.10. Spain
• 15.11. China
• 15.12. India
• 15.13. Japan
• 15.14. Australia
• 15.15. South Korea

16. Competitive Landscape

• 16.1. Market Share Analysis, 2024
• 16.2. FPNV Positioning Matrix, 2024
• 16.3. Competitive Analysis
  • 16.3.1. Sarepta Therapeutics, Inc.
  • 16.3.2. Pfizer Inc.
  • 16.3.3. BioMarin Pharmaceutical Inc.
  • 16.3.4. Roche Holding AG
  • 16.3.5. Bayer Aktiengesellschaft
  • 16.3.6. Solid Biosciences, Inc.
  • 16.3.7. Cytokinetics, Inc.
  • 16.3.8. NS Pharma, Inc.