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OpportunityAnalyzer: Duchenne Muscular Dystrophy - Opportunity and Market Analysis to 2019

Summary

Duchenne muscular dystrophy (DMD) is a rare genetic disorder affecting approximately 1 in 3,500 newborn boys and is characterized by progressive muscle degeneration, loss of ambulation, and death by the late 20s. There are no marketed pharmacological therapies that are indicated for DMD. The current standard of care treatment involves generic corticosteroids which are used to marginally prolong muscle function but do not offer preventative treatment. The anticipated launch of PTC Therapeutics’ Translarna in 2015 in the US and 5EU, followed by the 6MM launch of Exon-skipping therapies- BioMarin/Prosensa’s drisapersen and Sarepta Therapeutics’ eteplirsen, in 2015 and 2016, respectively, are set to change the treatment landscape and drive growth in the DMD market. GlobalData estimates that the uptake of Translarna and exon-skipping therapies will be very high due to the lack of effective disease-modifying therapies for DMD. GlobalData estimates that the DMD market was valued at $8.2m across the 6MM in 2014, and it is expected to sharply increase to approximately $990.0m in 2019, at a Compound Annual Growth Rate (CAGR) of 160.5%.

Highlights

Key Questions Answered

- How will the DMD market landscape change within the 2014-2019 forecast periods in the 6MM?
- What are the most promising late-stage pipeline drugs and how will their launch shape the future treatment landscape in the DMD market?
- How do the clinical and commercial attributes of late-stage pipeline drugs compare to one another and against existing treatment options?
- Which patient population(s) are most likely to be targeted by late-stage pipeline drugs?
- What are the significant unmet needs and remaining opportunities in the DMD market?

Key Findings

- Due to strong demand from patient community and clinicians, high uptake is expected of pipeline drugs for DMD that are anticipated to launch during the 2014-2019 forecast period.
- Mutation-specific therapies are expected to emerge as the major R&D breakthrough and future market drivers for DMD.
- With ongoing research, large pharmaceutical companies, small biotechs, and research institutions have discovered a multitude of therapeutic strategies to treat DMD. There are 19 ongoing clinical trials in DMD with 11 molecules in Phase II and eight molecules in Phase I.
- The lack of consensus over clinical trial design and endpoints for the development of DMD therapies continues to be a barrier to clinical development of new drugs.
- The clinical stage pipeline is mainly focussed on ambulatory DMD patients with specific mutations, leaving a large unaddressed population and vast opportunity for developers to deliver new therapies, and for continued growth in the DMD market beyond 2019.

Scope

- Overview of DMD, including epidemiology, etiology, pathophysiology, symptoms and current treatment options
- Annualized DMD therapeutics market revenue, annual cost of therapies and forecasts for five years to 2019.
- Key topics covered include strategic product assessment, market characterization, unmet needs, R&D strategies, clinical trial design and implications for the DMD therapeutics market.
- Pipeline analysis: comprehensive data split across different phases, emerging trends and mechanisms of action under development, including nonsense mutation readthrough inducer, exon-skipping therapies, synthetic electron transporter, PDE5 inhibitor.
- Analysis of the current and future market competition in the US and five major EU DMD therapeutics market. Clinical and commercial benchmarking of promising pipeline products versus standard of care treatments and competitive assessment of all therapies. Insightful review of the key industry drivers, restraints and challenges.

Reasons to buy

- Identify the unmet needs and remaining opportunities in the DMD therapeutics market.
- Develop business strategies by understanding the trends shaping and driving the US and five major EU DMD therapeutics market.
- Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
- Assess the clinical and commercial viability of promising pipeline products.
- Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies.
- Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various emerging therapies.
- Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.
- Drive revenues by understanding the key trends, innovative products and technologies, market and segments likely to impact the US and five major EU DMD therapeutics market in future.

Table Of Contents

OpportunityAnalyzer: Duchenne Muscular Dystrophy - Opportunity and Market Analysis to 2019
1 Table of Contents
1 Table of Contents 8
1.1 List of Tables 12
1.2 List of Figures 15
2 Introduction 16
2.1 Catalyst 16
2.2 Related Reports 17
3 Disease Overview 18
3.1 Etiology and Pathophysiology 18
3.1.1 Etiology 18
3.1.2 Pathophysiology 19
3.2 Prognosis and Symptoms 20
3.3 Diagnosis 21
4 Epidemiology 23
4.1 Disease Background 23
4.2 Risk Factors and Comorbidities 24
4.3 Global Trends 25
4.4 Forecast Methodology 27
4.4.1 Sources Used 33
4.4.2 Sources Not Used 34
4.4.3 Forecast Assumptions and Methods 35
4.5 Epidemiological Forecast for DMD (2013-2023) 40
4.5.1 Diagnosed Prevalent Cases of DMD (All Ages) 40
4.5.2 Diagnosed Prevalent Cases of DMD (Ages 5-13 Years) 42
4.5.3 Age-Specific Diagnosed Prevalent Cases of DMD (All Ages) 44
4.5.4 Age-Standardized Diagnosed Prevalence of DMD 45
4.6 Epidemiological Forecast for Nonsense Mutations in DMD (2013-2023) 46
4.7 Epidemiological Forecast for the Applicability of Exon-Skipping Therapies in DMD (2013-2023) 48
4.7.1 Diagnosed Prevalent Cases of DMD Eligible for an Exon 51 Skipping Therapy (All Ages) 48
4.7.2 Diagnosed Prevalent Cases of DMD Eligible for an Exon 45 Skipping Therapy (All Ages) 49
4.7.3 Diagnosed Prevalent Cases of DMD Eligible for an Exon 53 Skipping Therapy (All Ages) 51
4.7.4 Diagnosed Prevalent Cases of DMD Eligible for an Exon 44 Skipping Therapy (All Ages) 52
4.8 Epidemiological Forecast for BMD (2013-2023) 54
4.8.1 Diagnosed Prevalent Cases of BMD (All Ages) 54
4.8.2 Age-Specific Diagnosed Prevalent Cases of BMD (All Ages) 55
4.8.3 Age-Standardized Diagnosed Prevalence of BMD 57
4.9 Discussion 58
4.9.1 Epidemiological Forecast Insight 58
4.9.2 Limitations of the Analysis 59
4.9.3 Strengths of the Analysis 60
5 Current Treatment Options 61
5.1 Overview 61
5.1.1 Duchenne Muscular Dystrophy 61
5.1.2 Becker Muscular Dystrophy 62
5.2 Product Profiles - Major Brands 63
5.2.1 Corticosteroids (Prednisone and Deflazacort - Numerous Generic and Brand Names) 63
5.2.2 Translarna (ataluren) 66
6 Unmet Needs Assessment and Oppportunity Analysis 72
6.1 Overview 72
6.2 Need for Disease-Modifying Therapies 73
6.2.1 Unmet Need 73
6.2.2 Gap Analysis 74
6.2.3 Opportunity 74
6.3 Delayed Diagnosis 75
6.3.1 Unmet Need 75
6.3.2 Gap Analysis 76
6.3.3 Opportunity 76
6.4 High Variation in Design of DMD Clinical Trials 77
6.4.1 Unmet Need 77
6.4.2 Gap Analysis 78
6.4.3 Opportunity 78
6.5 Need for Regulatory Pathway for Approval of Follow-On Exon-Skipping Molecules 79
6.5.1 Unmet Need 79
6.5.2 Gap Analysis 79
6.5.3 Opportunity 80
7 RandD Strategies 81
7.1 Overview 81
7.1.1 Follow-On and Multi Exon-Skipping Molecules 81
7.1.2 Forming Alliances with Patient Advocacy Groups 82
7.1.3 Partnering with Pharmaceutical Companies to Increase Marketing Resources 83
7.2 Current Clinical Trial Design 84
7.2.1 Current Methods for Assessing Clinical Efficacy and Their Limitations 85
7.2.2 Current Trial Design of Key Pipeline Products 87
7.2.3 Patient Exclusion Issues in Current Trial Designs 88
7.3 Future Clinical Trial Design 89
7.3.1 Dystrophin-Positive Biomarkers as Surrogate Clinical Endpoints 89
7.3.2 Need for Measuring Clinical Outcome and Endpoints Based on Disease Onset 90
7.3.3 Maximizing Inclusion of Patient Populations in Studies 91
8 Pipeline Assessment 92
8.1 Overview 92
8.2 Promising Drugs in Clinical Development 93
8.2.1 Drisapersen 94
8.2.2 Eteplirsen 97
8.2.3 Catena/Raxone (idebenone) 101
8.2.4 Tadalafil 105
8.3 Innovative Early-Stage Approaches 108
8.3.1 Follow-On Exon-Skipping Therapies 110
8.3.2 Utrophin Activation 111
8.3.3 Myostatin Inhibition 112
8.3.4 Gene Therapy 112
8.3.5 Targeting Cellular Pathways and Other Early-Stage Approaches 113
9 Pipeline Valuation Analysis 115
9.1 Clinical Benchmark of Key Pipeline Drugs 115
9.2 Commercial Benchmark of Key Pipeline Drugs 117
9.3 Competitive Assessment 119
9.4 Top-Line Five-Year Forecast 122
9.4.1 US 126
9.4.2 5EU 127
10 Appendix 128
10.1 Bibliography 128
10.2 Abbreviations 137
10.3 Methodology 140
10.4 Forecasting Methodology 141
10.4.1 Diagnosed DMD Patients 141
10.4.2 Percent Drug-Treated Patients 142
10.4.3 Drugs Included in Each Therapeutic Class 142
10.4.4 Launch and Patent Expiry Dates 143
10.4.5 General Pricing Assumptions 143
10.4.6 Individual Drug Assumptions 144
10.4.7 Generic Erosion 148
10.4.8 Pricing of Pipeline Agents 148
10.5 Physicians and Specialists Included in this Study 150
10.6 About the Authors 151
10.6.1 Author 151
10.6.2 Reviewers 151
10.6.3 Epidemiologist 152
10.6.4 Global Head of Healthcare 152
10.7 About GlobalData 153
10.8 Disclaimer 153

1.1 List of Tables
Table 1: Symptoms of DMD 21
Table 2: Risk Factors and Comorbidities for DMD and BMD 25
Table 3: Global Estimates for the Diagnosed Prevalence of DMD and BMD 27
Table 4: MD STARnet Case Definition Criteria for DMD and BMD 29
Table 5: Sources of Epidemiological Data Used for the Forecast for the Diagnosed Prevalent Cases of DMD in the 6MM 30
Table 6: Sources of Epidemiological Data Used for the Forecast for the Diagnosed Prevalent Cases of BMD in the 6MM 30
Table 7: Sources of Epidemiological Data Used for the Forecast for the Diagnosed Prevalent Cases of DMD with Nonsense Mutations in the 6MM 31
Table 8: Sources of Epidemiological Data Used for the Forecast for the Diagnosed Prevalent Cases of DMD Eligible for Exon-Skipping Therapies (Exons 51, 45, 53, or 44) in the 6MM 32
Table 9: 6MM, Diagnosed Prevalent Cases of DMD, Males, All Ages, N, Select Years 2013-2023 41
Table 10: 6MM, Diagnosed Prevalent Cases of DMD, Males, Ages 5-13 Years, N, Select Years 2013-2023 43
Table 11: 6MM, Age-Specific Diagnosed Prevalent Cases of DMD, Men, N (Row %), 2013 44
Table 12: 6MM, Diagnosed Prevalent Cases of DMD with Nonsense Mutations, Males, All Ages, N, Select Years 2013-2023 47
Table 13: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 51 Skipping Therapy, Males, All Ages, N, Select Years 2013-2023 48
Table 14: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 45 Skipping Therapy, Males, All Ages, N, 2013-2023 50
Table 15: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 53 Skipping Therapy, Males, All Ages, N, Select Years 2013-2023 51
Table 16: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 44 Skipping Therapy, Males, All Ages, N, Select Years 2013-2023 53
Table 17: 6MM, Diagnosed Prevalent Cases of BMD, Males, All Ages, N, Select Years 2013-2023 54
Table 18: 6MM, Age-Specific Diagnosed Prevalent Cases of BMD, Men, N (Row %), 2013 56
Table 19: Current Treatment Options for Duchenne Muscular Dystrophy 62
Table 20: Product Profile - Corticosteroids (Generics - Prednisone, Deflazacort) 64
Table 21: Corticosteroids SWOT Analysis, 2014 66
Table 22: Product Profile - Translarna 68
Table 23: Translarna SWOT Analysis, 2014 71
Table 24: Unmet Need and Opportunity in Duchenne Muscular Dystrophy 73
Table 25: Clinical Trial Design of Key Pipeline Drugs for DMD, January 2015 87
Table 26: Clinical Outcome Measures Based on Age and Onset of DMD 90
Table 27: DMD - Late-Stage Pipeline, February 2015 94
Table 28: Product Profile - Drisapersen 95
Table 29: Drisapersen SWOT Analysis, 2014 97
Table 30: Product Profile - Eteplirsen 99
Table 31: Eteplirsen SWOT Analysis, 2014 101
Table 32: Product Profile - Catena 103
Table 33: Catena SWOT Analysis, 2014 105
Table 34: Product Profile - Tadalafil 107
Table 35: Tadalafil SWOT Analysis, 2014 108
Table 36: Early-Stage Pipeline Products in DMD, February 2015 109
Table 37: Clinical Benchmark of Key Pipeline Drugs for DMD 117
Table 38: Commercial Benchmark of Key Pipeline Drugs for DMD 118
Table 39: Top-Line Sales Forecasts ($m) for DMD, 2014-2019 123
Table 40: Key Events Impacting Sales for DMD, 2014-2019 125
Table 41: DMD Market - Drivers and Barriers, 2014-2019 126
Table 42: Key Launch Dates, DMD, 2014-2019 143
Table 43: Key Patent Expiries, 2014-2019 143

1.2 List of Figures
Figure 1: Schematic Representation of Dystrophin and Associated Proteins in Muscle. 19
Figure 2: Patient Flow for the Epidemiological Forecast of DMD in the 6MM 28
Figure 3: 6MM, Diagnosed Prevalent Cases of DMD, Males, All Ages, N, 2013-2023 42
Figure 4: 6MM, Diagnosed Prevalent Cases of DMD, Males, Ages 5-13 Years, N, 2013-2023 43
Figure 5: 6MM, Age-Specific Diagnosed Prevalent Cases of DMD, Men, N, 2013 45
Figure 6: 6MM, Age-Standardized Diagnosed Prevalence of DMD (Cases per 100,000 Population), All Ages, Men, 2013 46
Figure 7: 6MM, Diagnosed Prevalent Cases of DMD with Nonsense Mutations, Males, All Ages, N, 2013-2023 47
Figure 8: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 51 Skipping Therapy, Males, All Ages, N, 2013-2023 49
Figure 9: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 45 Skipping Therapy, Males, All Ages, N, 2013-2023 50
Figure 10: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 53 Skipping Therapy, Males, All Ages, N, 2013-2023 52
Figure 11: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 44 Skipping Therapy, Males, All Ages, N, 2013-2023 53
Figure 12: 6MM, Diagnosed Prevalent Cases of BMD, Males, All Ages, N, 2013-2023 55
Figure 13: 6MM, Age-Specific Diagnosed Prevalent Cases of BMD, Men, N, 2013 57
Figure 14: 6MM, Age-Standardized Diagnosed Prevalence of BMD (Cases per 100,000 Population), All Ages, Men, 2013 58
Figure 15: Competitive Assessment of Key Pipeline Drugs for DMD, 2014-2019 119
Figure 16: Sales for DMD by Region, 2014-2019 124

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