Muscular Dystrophy – Landscape & Forecast – Disease Landscape & Forecast
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. Treatments are needed that can meaningfully delay or halt the progressive muscle degeneration associated with Duchenne muscular dystrophy (DMD), the most common childhood-onset form, as well as other forms of MD. The standard treatment for MD is glucocorticoids, which have proven effective in delaying the loss of ambulation. Recent conditional approvals of genotype-specific, disease-modifying therapies such as Sarepta’s Exondys 51, Vyondys 53, and Amondys 45; Nippon Shinyaku’s Viltolarsen; and PTC Therapeutics’ Translarna have expanded treatment options for DMD patients, but there are lingering concerns about these drugs’ clinical efficacy. 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 more-effective treatments 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 2031?
- What is the current treatment landscape 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 2031, and what will be their commercial impact on the MD market through 2031?
GEOGRAPHIES
United States, EU5
PRIMARY RESEARCH
Six country-specific interviews with thought-leading neurologists
Supported by survey data collected for this study
EPIDEMIOLOGY
Diagnosed prevalent and drug-treated cases of DMD, diagnosed prevalent cases of DMD by exon-skipping pattern and ambulatory status, diagnosed prevalent cases of Becker MD, limb-girdle MD, facioscapulohumeral MD, and myotonic dystrophy.
FORECAST
Drug-level sales and patient shares of key muscular dystrophy therapies through 2031
EMERGING THERAPIES
Phase III/PR: 6 drugs; Phase I/II: > 10 drugs; coverage of select early-phase and preclinical products.
Product Description
Niche & Rare Disease Landscape & Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.
Table of contents
- Muscular Dystrophy - Landscape & Forecast - Disease Landscape & Forecast
- Key findings
- Muscular dystrophy key findings
- Commercial outlook
- Key findings
- Regional sales of key therapies to treat muscular dystrophy: 2021 and 2031
- Muscular dystrophy SWOT analysis
- 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
- Exon-skipping therapy-specific trends
- Gene therapy-specific trends
- Nonsense read-through therapy-specific trends
- Deflazacort-specific trends
- Vamorolone-specific trends
- Givinostat-specific trends
- AMO-02-specific trends
- Losmapimod-specific trends
- Alternative market scenarios
- Alternative scenarios for the muscular dystrophy market through 2031
- Key findings
- Forecast
- Sales of key therapies in muscular dystrophy: 2021-2031
- 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
- Disease overview
- 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: 2021-2031
- Disease definition
- Methods
- Sources used for diagnosed prevalence of myotonic dystrophy
- Diagnosed prevalent cases of myotonic dystrophy: 2021-2031
- Disease definition
- Methods
- Sources used for diagnosed prevalent cases of type 1 myotonic dystrophy by subtype
- Diagnosed prevalent cases of myotonic dystrophy type 1 by subtype: 2021-2031
- 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: 2021-2031
- 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: 2021-2031
- Disease definition
- Methods
- Sources used for diagnosed prevalent cases of Becker muscular dystrophy
- Diagnosed prevalent cases of Becker muscular dystrophy: 2021-2031
- Disease definition
- Methods
- Sources used for diagnosed prevalent cases of limb-girdle muscular dystrophy
- Diagnosed prevalent cases of limb-girdle muscular dystrophy: 2021-2031
- Drug-treatable diagnosed prevalent cases of Duchenne muscular dystrophy: 2021-2031
- Key findings
- 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
- North Star Ambulatory Assessment (NSAA)
- 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
- Expert insight: Exon-skipping therapies
- 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 (eteplirsen) in the treatment of Duchenne muscular dystrophy
- Expert insight: Exondys 51
- 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 (golodirsen) in the treatment of Duchenne muscular dystrophy
- Expert insight: Vyondys 53
- Clinical trial outcomes for Amondys 45
- Advantages and disadvantages of Amondys 45
- Key results from select clinical trials investigating Amondys 45 for the treatment of Duchenne muscular dystrophy
- Ongoing clinical development of Amondys 45
- Key ongoing clinical trials of Amondys 45 (casimersen) in the treatment of Duchenne muscular dystrophy
- Expert insight: Amondys 45
- Clinical trial outcomes for Viltolarsen
- Advantages and disadvantages of Viltolarsen
- Key results from select clinical trials investigating Viltolarsen for the treatment of Duchenne muscular dystrophy
- Ongoing clinical development of Viltolarsen
- Key ongoing clinical trials of Viltolarsen in the treatment of Duchenne muscular dystrophy
- Expert insight: Viltolarsen
- 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 the symptomatic management of cardiac complications in Duchenne muscular dystrophy
- Medical practice
- 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
- Key findings
- Unmet need overview
- Current and future attainment of unmet needs in muscular dystrophy
- Top unmet needs in muscular dystrophy: current and future attainment
- Expert insight: unmet needs
- Emerging therapies
- Key findings
- Key emerging therapies
- Key therapies in development for muscular dystrophy
- Estimated launch dates of key emerging therapies for the treatment of muscular dystrophy
- Vamorolone
- Vamorolone profile
- 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 the launch and sales opportunity of vamorolone in Duchenne muscular dystrophy
- AMO-02 profile
- Key ongoing clinical trials investigating AMO-02 for the treatment of myotonic dystrophy
- Analysis of the clinical development program for AMO-02
- Expert insight: AMO-02
- Expectations for the launch and sales opportunity of AMO-02 in myotonic dystrophy
- Givinostat profile
- Key ongoing clinical trials of givinostat for the treatment of Duchenne muscular dystrophy
- Analysis of the clinical development program for givinostat
- Expectations for the launch and sales opportunity of givinostat in Duchenne muscular dystrophy
- Pamrevlumab profile
- Key ongoing clinical trials investigating pamrevlumab for the treatment of Duchenne muscular dystrophy
- Analysis of the clinical development program for pamrevlumab
- Expectations for the 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
- Expert insight: SRP-9001
- Expectations for the 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
- Expert insight: PF-06939926
- Expectations for the launch and sales opportunity of PF-06939926 in Duchenne muscular dystrophy
- A comparative account of the two most advanced dystrophin gene therapies in development for Duchenne muscular dystrophy
- Key ongoing clinical trials of SGT-001 for the treatment of Duchenne muscular dystrophy
- Analysis of the clinical development program for SGT-001
- Expectations for the launch and sales opportunity of SGT-001 in Duchenne muscular dystrophy
- CAP-1002 profile
- Key ongoing clinical trials of CAP-1002 for the treatment of Duchenne muscular dystrophy
- Analysis of the clinical development program for CAP-1002
- Expectations for the launch and sales opportunity of CAP-1002 in Duchenne muscular dystrophy
- SRP-9003 profile
- Key ongoing clinical trials investigating SRP-9003 for the treatment of Limb-girdle muscular dystrophy
- Analysis of the clinical development program for SRP-9003
- Expert insight: SRP-9003
- Expectations for the launch and sales opportunity of SRP-9003 in limb-girdle muscular dystrophy
- Losmapimod profile
- Key ongoing clinical trials investigating losmapimod for the treatment of FSHD
- Analysis of the clinical development program for losmapimod
- Expectations for the launch and sales opportunity of losmapimod in FSHD
- Early-phase pipeline analysis
- Early-phase emerging therapies
- 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
- Key findings
- Access & reimbursement overview
- Region-specific reimbursement practices
- General reimbursement environment: United States
- General reimbursement environment: EU5
- Region-specific reimbursement practices
- Appendix
- Abbreviation table
- Muscular dystrophy bibliography
- Key findings
Raina Priyadarshini, Ph.D., is a senior analyst on the Infectious, Niche, and Rare Diseases team at Clarivate. Previously, she was an analyst in the biopharmaceutical industry, where she developed novel disease target-drug combinations for rare CNS and hematological disorders. Dr. Priyadarshini trained as a molecular biologist; her research focused on the interplay between tumor suppressor proteins and oncoproteins. She received her doctorate from the National Institute of Immunology.
Dr. Harpreet Kaur, B.D.S., M.P.H., is an associate epidemiologist at Clarivate. Prior to joining the company, she was awarded a National Clinical Research Fellowship by the Indian Council of Medical Research (ICMR) and worked as a clinical scientist in the broader area of adolescent reproductive health. She obtained her master’s degree in social epidemiology from the Tata Institute of Social Sciences in Mumbai and graduated with a degree in dentistry from Punjab University in Chandigarh.