Muscular dystrophy (MD) is a spectrum of genetic disorders characterized by muscle weakness that, in severe forms, can lead to loss of ambulation and early mortality. There is a high unmet need for effective treatments that can meaningfully delay or halt progressive muscle degeneration in Duchenne muscular dystrophy (DMD), the most common childhood-onset form, as well as in other forms of MD. The standard treatment option for MD patients is glucocorticoids, which have proven effective in delaying loss of ambulation. Recent conditional approvals of genotype-specific, disease-modifying therapies such as Sarepta’s Exondys 51 and Vyondys 53, and PTC Therapeutics’ Translarna have expanded treatment options for DMD patients; however, there are lingering concerns about the clinical efficacy of these drugs. Additionally, many therapies with diverse mechanisms of action are being developed to treat other forms of MD, including limb-girdle MD and Becker MD. Overall, there is still high unmet need for additional and, ideally, more-effective medications for DMD and other forms of MD.

QUESTIONS ANSWERED

  • What are the sizes of key MD patient populations in the United States and EU5 countries, and how will the population change through 2029?
  • What are the current treatment landscape and medical practice in MD, and where are the greatest unmet clinical needs according to experts? How well will pipeline therapies address current treatment gaps?
  • Which emerging therapies will launch before 2029, and what will be their commercial impact on the MD market through 2029?

Table of contents

  • Muscular Dystrophy - Landscape & Forecast - Disease Landscape & Forecast
    • COVID-19
      • Key Findings
        • Muscular Dystrophy Key Findings - June 2020
      • Commercial Outlook
        • Key Findings
          • Regional Sales of Key Therapies to Treat Muscular Dystrophy: 2019 and 2029
          • Muscular Dystrophy SWOT Analysis
        • COVID-19: Areas of Potential Forecast Impact
          • Drivers and Constraints
            • What Factors Are Driving the Market for Muscular Dystrophy?
            • What Factors Are Constraining the Market for Muscular Dystrophy?
          • Drug-Class-Specific Trends
            • Gene Therapy-Specific Trends
            • Percent of Diagnosed Prevalent Duchenne Muscular Dystrophy Patients Treated with Gene Therapy
            • Exon Skipping Therapy-Specific Trends
            • Nonsense Read-Through Therapy
            • Immunomodulatory Therapy-Specific Trends
            • Antioxidants
            • Antifibrotics
          • Alternative Market Scenarios
            • Alternative Scenarios for the Muscular Dystrophy Market Through 2029
        • Forecast
          • Sales of Key Therapies in Muscular Dystrophy: 2019-2029
        • Etiology and Pathophysiology
          • Disease Overview
            • Etiology
              • Genetic Causes of Muscular Dystrophies
              • Genes Associated with Select Muscular Dystrophies
              • Dystrophin Mutations Underlying Duchenne and Becker Muscular Dystrophy
              • Limb-Girdle and Congenital Muscular Dystrophy
              • Myotonic Dystrophy
            • Pathophysiology
              • Duchenne and Becker Muscular Dystrophy
              • The Dystrophin-Associated Protein Complex and Other Proteins Involved in Muscular Dystrophies
              • Limb-Girdle and Congenital Muscular Dystrophy
              • Myotonic Dystrophy
            • Biomarkers for Duchenne Muscular Dystrophy
              • Disease Onset and Progression
                • Onset, Symptoms, and Progression of Duchenne and Becker Muscular Dystrophy
                • Early Signs of Duchenne Muscular Dystrophy
                • Staging of Duchenne Muscular Dystrophy
                • Onset and Progression of Disability in Duchenne Muscular Dystrophy
                • Onset, Symptoms, and Progression of Limb-Girdle Muscular Dystrophy
                • Onset, Symptoms, and Progression of Congenital Muscular Dystrophy
                • Onset, Symptoms, and Progression of Myotonic Dystrophy
                • Cardiac Complications in Myotonic Dystrophy
              • Key Pathways and Drug Targets
                • Antisense Oligonucleotide-Mediated Exon Skipping
            • Epidemiology
              • Key Findings
                • Epidemiology Populations
                  • Disease Definition
                  • Methods
                  • Sources Used for Diagnosed Prevalent Cases of Duchenne Muscular Dystrophy
                  • Diagnosed Prevalent Cases of Duchenne Muscular Dystrophy: 2019-2029
                  • Disease Definition
                  • Methods
                  • Sources Used for Diagnosed Prevalent Cases of Myotonic Dystrophy
                  • Diagnosed Prevalent Cases of Myotonic Dystrophy: 2019-2029
                  • Disease Definition
                  • Methods
                  • Sources Used for Diagnosed Prevalent Cases of Myotonic Dystrophy Type 1 by Subtype
                  • Diagnosed Prevalent Cases of Myotonic Dystrophy Type 1 by Subtype: 2019-2029
                  • Drug-Treatable Diagnosed Prevalent Cases of Duchenne Muscular Dystrophy: 2019-2029
                  • Disease Definition
                  • Methods
                  • Sources Used for Diagnosed Prevalent Cases of Duchenne Muscular Dystrophy by Exon Skipping Pattern
                  • Diagnosed Prevalent Cases of Duchenne Muscular Dystrophy by Exon Skipping Pattern: 2019-2029
                  • Disease Definition
                  • Methods
                  • Sources Used for Diagnosed Prevalent Cases of Duchenne Muscular Dystrophy by Ambulatory Status
                  • Diagnosed Prevalent Cases of Duchenne Muscular Dystrophy by Ambulatory Status: 2019-2029
                  • Disease Definition
                  • Methods
                  • Sources Used for Diagnosed Prevalent Cases of Becker Muscular Dystrophy
                  • Diagnosed Prevalent Cases of Becker Muscular Dystrophy: 2019-2029
                  • Disease Definition
                  • Methods
                  • Sources Used for Diagnosed Prevalent Cases of Limb-Girdle Muscular Dystrophy
                  • Diagnosed Prevalent Cases of Limb-Girdle Muscular Dystrophy: 2019-2029
              • Current Treatment
                • Key Findings
                  • Diagnosis
                    • Genetic Testing for Duchenne Muscular Dystrophy
                    • Newborn Screening for Duchenne Muscular Dystrophy
                    • Treatment Providers and Referral Patterns
                    • Expert Insight: Diagnosis of Muscular Dystrophy
                  • Treatment Goals
                    • Key Endpoints Used in Clinical Trials for Muscular Dystrophy
                    • The Six-Minute Walk Test
                    • Expert Insight: Treatment Goals
                  • Key Current Therapies
                    • Mechanism of Action of Key Current Drugs or Drug Classes Used for Muscular Dystrophy
                    • Current Treatments Used for Muscular Dystrophy
                    • Clinical Trial Outcomes for Glucocorticoids
                    • Advantages and Disadvantages of Glucocorticoids
                    • Management of Glucocorticoid Side Effects
                    • Expert Insight: Glucocorticoids
                    • Clinical Trial Outcomes for Exondys 51
                    • Advantages and Disadvantages of Exondys 51
                    • Key Results from Select Clinical Trials Investigating Exondys 51 for the Treatment of Duchenne Muscular Dystrophy
                    • Ongoing Clinical Development of Exondys 51
                    • Key Ongoing Clinical Trials of Exondys 51 in the Treatment of Duchenne Muscular Dystrophy
                    • Expert Insight: Exondys 51
                    • Vyondys 53
                    • Clinical Trial Outcomes for Vyondys 53
                    • Advantages and Disadvantages of Vyondys 53
                    • Key Results from Select Clinical Trials Investigating Vyondys 53 for the Treatment of Duchenne Muscular Dystrophy
                    • Ongoing Clinical Development of Vyondys 53
                    • Key Ongoing Clinical Trials of Vyondys 53 in the Treatment of Duchenne Muscular Dystrophy
                    • Expert Insight: Vyondys 53
                    • Clinical Trial Outcomes for Translarna
                    • Advantages and Disadvantages of Translarna
                    • Key Results from Select Clinical Trials Investigating Translarna for the Treatment of Duchenne Muscular Dystrophy
                    • Ongoing Clinical Development of Translarna
                    • Key Ongoing Clinical Trials of Translarna in the Treatment of Duchenne Muscular Dystrophy
                    • Expert Insight: Translarna
                    • Key Therapies for Symptomatic Management of Cardiac Complications in Duchenne Muscular Dystrophy
                  • Medical Practice
                    • Regional Muscular Dystrophy Treatment Guidelines
                    • Factors Influencing Drug Selection in Duchenne Muscular Dystrophy
                    • Multidisciplinary Management Team for Muscular Dystrophy
                    • Expert Insight: Multidisciplinary Care
                    • Region-Specific Treatment Practices
                    • Generalized Treatment Decision Tree for Duchenne Muscular Dystrophy
                • Unmet Need Overview
                  • Current and Future Attainment of Unmet Needs in Muscular Dystrophy
                  • Top Unmet Needs in Muscular Dystrophy: Current and Future Attainment
                • Emerging Therapies
                  • Key Findings
                    • Pipeline Trends in Muscular Dystrophy
                  • Key Emerging Therapies
                    • Key Therapies in Development for Muscular Dystrophy
                    • Estimated Launch Dates of Key Emerging Therapies for the Treatment of Muscular Dystrophy
                    • Casimersen Profile
                    • Key Ongoing Clinical Trials Investigating Casimersen for the Treatment of Duchenne Muscular Dystrophy
                    • Analysis of the Clinical Development for Casimersen
                    • Expert Insight: Casimersen
                    • Expectations for Launch and Sales Opportunity for Casimersen in Duchenne Muscular Dystrophy
                    • Viltolarsen Profile
                    • Key Ongoing Clinical Trials Investigating Viltolarsen for the Treatment of Duchenne Muscular Dystrophy
                    • Analysis of the Clinical Development for Viltolarsen
                    • Expert Insight: Viltolarsen
                    • Expectations for Launch and Sales Opportunity for Viltolarsen in Duchenne Muscular Dystrophy
                    • Idebenone Profile
                    • Key Ongoing Clinical Trials of Idebenone in the Treatment of Duchenne Muscular Dystrophy
                    • Analysis of the Clinical Development Program for Idebenone
                    • Expert Insight: Idebenone
                    • Expectations for Launch and Sales Opportunity of Idebenone in Duchenne Muscular Dystrophy
                    • Vamorolone
                    • Key Ongoing Clinical Trials Investigating Vamorolone for the Treatment of Duchenne Muscular Dystrophy
                    • Analysis of the Clinical Development Program for Vamorolone
                    • Expert Insight: Vamorolone
                    • Expectations for Launch and Sales Opportunity of Vamorolone in Duchenne Muscular Dystrophy
                    • Key Ongoing Clinical Trials Investigating Edasalonexent for the Treatment of Duchenne Muscular Dystrophy
                    • Analysis of the Clinical Development for Edasalonexent
                    • Expectations for Launch and Sales Opportunity of Edasalonexent in Duchenne Muscular Dystrophy
                    • Key Ongoing Clinical Trials Investigating AMO-02 for the Treatment of Myotonic Dystrophy
                    • Analysis of the Clinical Development Program for AMO-02
                    • Expectations for Launch and Sales Opportunity of AMO-02 in Myotonic Dystrophy
                    • Key Ongoing Clinical Trials of Givinostat for the Treatment of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
                    • Analysis of the Clinical Development Program for Givinostat
                    • Expectations for Launch and Sales Opportunity of Givinostat in Duchenne Muscular Dystrophy
                    • Key Ongoing Clinical Trials Investigating Pamrevlumab for the Treatment of Duchenne Muscular Dystrophy
                    • Analysis of the Clinical Development Program for Pamrevlumab
                    • Expectations for Launch and Sales Opportunity of Pamrevlumab in Duchenne Muscular Dystrophy
                    • Expert Insight: Dystrophin-Based Gene Therapies
                    • SRP-9001 Profile
                    • Key Ongoing Clinical Trials Investigating SRP-9001 for the Treatment of Duchenne Muscular Dystrophy
                    • Analysis of the Clinical Development Program for SRP-9001
                    • Expectations for Launch and Sales Opportunity of SRP-9001 in Duchenne Muscular Dystrophy
                    • PF-06939926 Profile
                    • Key Ongoing Clinical Trials Investigating PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
                    • Analysis of the Clinical Development Program for PF-06939926
                    • Expectations for Launch and Sales Opportunity of PF-06939926 in Duchenne Muscular Dystrophy
                    • SGT-001 Profile
                    • Analysis of the Clinical Development Program for SGT-001
                    • Expectations for Launch and Sales Opportunity of SGT-001 in Duchenne Muscular Dystrophy
                    • Key Ongoing Clinical Trials Investigating SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy
                    • Analysis of the Clinical Development Program for SRP-9003
                    • Expectations for Launch and Sales Opportunity of SRP-9003 in Limb-Girdle Muscular Dystrophy
                  • Early-Phase Pipeline Analysis
                    • Select Compounds in Early-Phase Development for Muscular Dystrophy
                  • Key Discontinuations and Failures in Muscular Dystrophy
                    • Patient Registries
                      • Patient Registries for Muscular Dystrophy
                      • Prominent Muscular Dystrophy Patient Organizations
                    • Orphan-Drug Designation
                    • Access and Reimbursement Overview
                      • Region-Specific Reimbursement Practices
                        • General Reimbursement Environment: United States
                        • General Reimbursement Environment: EU5
                    • Methodology
                      • Bottom-Up Market Forecasting Overview
                        • Patient Populations
                        • Drug-Specific Assumptions
                      • Bottom-Up Forecast Assumptions
                        • Drug-Treatment Assumptions in Muscular Dystrophy
                        • Patient-Share Assumptions in Muscular Dystrophy
                        • General Statements About Pricing
                        • Sources for Drug Prices
                        • 2019 Exchange Rates
                        • Price per Treated Day: 2019
                        • Price per Treated Day: 2029
                        • Dosing, Days of Therapy, and Compliance
                        • Generic Erosion
                        • Generic Erosion Assumptions
                        • Out-Year Forecasting
                        • Emerging Therapy Prices
                      • Primary Market Research
                        • Experts Interviewed
                    • Appendix
                      • Key Abbreviations Related to Muscular Dystrophy
                      • Brands, Marketers, and Generic Availability of Key Therapies for Muscular Dystrophy by Market
                      • Muscular Dystrophy Bibliography

                  Author(s): Nuno Tiago Giao Antunes, PhD; Shilpa Thakur

                  Nuno T. Antunes, Ph.D., is senior business insights analyst on the Infectious, Niche, and Rare Diseases team at Decision Resources Group, specializing in antibacterial agents. Previously, he was a Latin America Market Access senior analyst in the Global Market Access Insights Team, where he developed expertise in market access, pricing and reimbursement, health technology assessment, and health policy.

                  Nuno holds a Ph.D. in animal health from the Universidad de las Palmas de Gran Canaria, Spain, and a D.V.M. degree from the Universidade de Trás-os-Montes e Alto Douro, Portugal. Prior to joining DRG, he conducted research in antimicrobial resistance and antimicrobial development, and worked in the medical devices industry as a scientist.

                  Shilpa Thakur is a medical graduate with a M.P.H from the Postgraduate Institute of Medical Education and Research with a specialization in epidemiology and biostatistics. She specializes in developing epidemiological forecasts for the multiple indications within the DRG syndicated portfolio.

                  Prior to joining Decision Resources, she monitored HIV sentinel surveillance 2016-2017 in Himachal Pradesh. She also has worked on to see the patterns of Antimicrobial resistance in India.