Home / Therapeutic Area / Global Genetic Disorders Drugs Market to 2023 – A Rapidly Growing Treatment Landscape Driven by Targeted Complement System Inhibitors and Enzyme Replacement Therapies to Treat PNH and Lysosomal Storage Disease

Global Genetic Disorders Drugs Market to 2023 – A Rapidly Growing Treatment Landscape Driven by Targeted Complement System Inhibitors and Enzyme Replacement Therapies to Treat PNH and Lysosomal Storage Disease

Published: Feb 2018 | Published By: GBI Research

The genetic disorders therapy area consists of indications that arise because of an abnormality in a person’s DNA. This report focuses on the key therapy area indications of cystic fibrosis (CF), Duchenne muscular dystrophy (DMD), lysosomal storage disease (LSD) and paroxysmal nocturnal hemoglobinuria (PNH). Genetic disorders are frequently inherited from an individual’s parents but can also arise because of a new (de novo) mutation. The treatment landscape for genetic disorders is dominated commercially by orphan drugs for the treatment of rare genetic disorders. Overall, 1,390 products are being actively developed in the genetic disorders pipeline. The pipelines of CF, DMD, LSD and PNH contain 163, 109, 174 and 24 products, respectively. The pipeline is dominated by targeted therapies that aim to treat the underlying cause, which is often specific to the particular indication.

Scope

  • Global revenue from the genetic disorders market is forecast to increase from $19.6 billion in 2016 to $47.7 billion in 2023, at a compound annual growth rate of 13.55%. What is driving this growth?
  • The leading companies in terms of market share are Alexion, Sanofi, Shire and Vertex. Which of these are forecast to experience the largest growth?
  • There are new approvals and late-stage products set to enter the market during the forecast period. Which drugs will achieve blockbuster status?
  • The market has been dominated by complement system inhibitors and enzyme replacement therapies. To what extent will these classes of drugs and others dominate the market over the forecast period?
  • There are 1,390 genetic disorders products in the pipeline. What molecular targets are most abundant in the pipeline and what role will pipeline product approvals play in market growth?
  • Genetic disorders clinical trials have an overall attrition rate of around 93%, what can companies do to maximize their chance of success?

Reasons To Buy

  • Understand the current treatment landscape, with portfolios of key marketed products and a focus on historical and forecast sales patterns, and an overview of each drug’s mechanism of action.
  • Analyze the genetic disorders pipeline through a comprehensive review of the pipeline segmented by stage of development, molecule type and molecular target.
  • This review also provides a detailed look at products for the treatment of genetic disorders to provide an insight into the risk associated with attempting to bring pipeline products to market.
  • Predict growth in market size, with in-depth market forecasting from 2016 to 2023.
  • The forecasts will provide an understanding of how epidemiology trends, new drug entries, and patent expirations will influence market value.
  • Identify commercial opportunities in the genetic disorders deals landscape by analyzing trends in licensing and co-development deals.

1 Table of Contents 4
1.1 List of Tables 7
1.2 List of Figures 8

2 Introduction 11
2.1.1 Cystic Fibrosis 12
2.1.2 Duchenne Muscular Dystrophy 12
2.1.3 Lysosomal Storage Disease 12
2.1.4 Paroxysmal Nocturnal Hemoglobinuria 14
2.2 Symptoms 14
2.3 Diagnosis 15
2.3.1 Cystic Fibrosis 17
2.3.2 Duchenne Muscular Dystrophy 18
2.3.3 Lysosomal Storage Disease 19
2.3.4 Paroxysmal Nocturnal Hemoglobinuria 19
2.4 Etiology and Pathophysiology 20
2.4.1 Cystic Fibrosis 20
2.4.2 Duchenne Muscular Dystrophy 21
2.4.3 Lysosomal Storage Disease 22
2.4.4 Paroxysmal Nocturnal Hemoglobinuria 25
2.5 Epidemiology Patterns and Forecasts – Prevalence and Patient Segmentation 26
2.5.1 Cystic Fibrosis 27
2.5.2 Duchenne Muscular Dystrophy 28
2.5.3 Lysosomal Storage Disease 28
2.5.4 Paroxysmal Nocturnal Hemoglobinuria 29
2.6 Co-morbidities and Complications 30
2.6.1 Cystic Fibrosis 30
2.6.2 Duchenne Muscular Dystrophy 31
2.6.3 Lysosomal Storage Disease 31
2.6.4 Paroxysmal Nocturnal Hemoglobinuria 32
2.7 Prognosis 33
2.7.1 Cystic Fibrosis 33
2.7.2 Duchenne Muscular Dystrophy 34
2.7.3 Lysosomal Storage Disease 35
2.7.4 Paroxysmal Nocturnal Hemoglobinuria 37
2.8 Treatment 37
2.8.1 Cystic Fibrosis 37
2.8.2 Duchenne Muscular Dystrophy 39
2.8.3 Lysosomal Storage Disease 41
2.8.4 Paroxysmal Nocturnal Hemoglobinuria 42

3 Key Marketed Products 44
3.1 Overview 44
3.2 Soliris (eculizumab) – Alexion 44
3.3 Orkambi (lumacaftor/ivacaftor) – Vertex 46
3.4 Cerezyme (imiglucerase) – Sanofi Genzyme 47
3.5 Myozyme/Lumizyme (alglucosidase alfa) – Sanofi Genzyme 49
3.6 Fabrazyme (agalsidase beta) – Sanofi Genzyme 51
3.7 Pulmozyme (dornase alfa) – Roche 53
3.8 Kalydeco (ivacaftor) – Vertex 54
3.9 Elaprase (idursulfase) – Shire 56
3.10 Vimizim (elosulfase alfa) – Biomarin 58
3.11 Cerdelga (eliglustat tartrate) – Sanofi Genzyme 60
3.12 Exondys 51 (eterplirsen) – Sarepta 61
3.13 Conclusion 62

4 Pipeline Landscape Assessment 63
4.1 Overview 63
4.2 Pipeline Development Landscape 63
4.3 Molecule Types in the Pipeline 66
4.4 Molecular Targets in the Pipeline 67
4.5 Clinical Trials Landscape 69
4.5.1 Clinical Trial Failure Rates 69
4.5.2 Clinical Trial Duration 73
4.5.3 Clinical Trial Size 77
4.5.4 Cumulative Clinical Trial Size 81
4.6 Assessment of Key Pipeline Products 84
4.6.1 Epidiolex (cannabidiol) – GW Pharmaceuticals 84
4.6.2 ALXN-1210 (ravulizumab) – Alexion 86
4.6.3 Lanadelumab – Shire 87
4.6.4 Burosumab – Ultragenyx and Kyowa Hakko Kirin 88
4.6.5 GBT440 (voxelotor) – Global Blood Therapeutics 90
4.6.6 Ivacaftor and Tezacaftor – Vertex 91

5 Multi-scenario Market Forecast to 2023 93
5.1 Overall Market Size 93
5.2 Generic Penetration 95
5.3 Revenue Forecast by Molecular Target 97
5.3.1 Immune/Inflammatory Mediators 97
5.3.2 Enzyme Replacement/Modifying Therapies 98
5.3.3 Ion Channel Modulators 99
5.3.4 G Protein-Coupled Receptor Modulators 100
5.3.5 Metabolic Enzyme Mediators 101

6 Company Analysis and Positioning 102
6.1 Revenue and Market Share Analysis by Company 104
6.1.1 Alexion – How will Alexion overcome the threat posed by the emergence of Soliris biosimilars? 108
6.1.2 Sanofi – How will revenue be affected by the expiry of key patents? 109
6.1.3 Vertex – Will Vertex continue to focus heavily on the treatment of CF? 110
6.1.4 Shire – How will late-stage pipeline drug Lanadelumab affect Shire’s revenue? 111
6.1.5 Amgen – Will Amgen develop more products for genetic diseases following the success of Repatha? 112
6.1.6 GW Pharmaceuticals – How will Epidiolex drive sales for GW? 113
6.1.7 Pfizer – How will Pfizer’s small genetic disorders pipeline affect long-term sales? 114
6.1.8 BioMarin – To what extent will BioMarin continue to focus exclusively on the treatment of genetic disorders? 115
6.1.9 Sarepta – Will Sarepta emerge as a key player in the genetic disorders landscape? 116
6.2 Company Landscape 117
6.3 Marketed and Pipeline Portfolio Analysis 118

7 Strategic Consolidations 121
7.1 Licensing Deals 121
7.1.1 Deals by Region, Year and Value 121
7.1.2 Deals by Stage of Development and Value 124
7.1.3 Deals by Molecule Type, Molecular Target and Value 125
7.1.4 Table for Licensing Deals with Disclosed Values 126
7.2 Co-development Deals 130
7.2.1 Deals by Region, Year and Value 130
7.2.2 Deals by Stage of Development and Value 132
7.2.3 Deals by Molecule Type, Molecular Target and Value 133
7.2.4 Table for Co-development Deals with Disclosed Values 134

8 Appendix 138
8.1 References 138
8.2 Figures of All Clinical Stage Pipeline Products 147
8.3 Abbreviations 156
8.4 Disease List 157
8.5 Methodology 158
8.5.1 Coverage 158
8.5.2 Secondary Research 158
8.5.3 Market Size and Revenue Forecasts 159
8.5.4 Pipeline Analysis 159
8.5.5 Competitive Landscape 160
8.6 Contact Us 160
8.7 Disclaimer 160

Table 1: Genetic Disorders, Common Symptoms of Genetic Disorders 14

Table 2: Genetic Disorders, Affected Areas of LSDs 15

Table 3: Genetic Disorders, Classification of Lysosomal Storage Diseases, 2017, Part 1 23

Table 4: Genetic Disorders, Classification of Lysosomal Storage Diseases, 2017, Part 2 25

Table 5: Genetic Disorders, Global, Epidemiology of Genetic Disorders, 2017 27

Table 6: Genetic Disorders, CF Severity Classification, 2017 33

Table 7: Stages of Duchenne Muscular Dystrophy, 2017 35

Table 8: Genetic Disorders, Global, CF Treatment Plan, 2017 38

Table 9: Genetic Disorders, Global, DMD Treatment Plan, 2017 39

Table 10: Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Soliris, 2017 45

Table 11: Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Orkambi, 2017 46

Table 12: Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Cerezyme, 2017 48

Table 13: Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Myozyme/Lumizyme, 2017 50

Table 14: Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Fabrazyme, 2017 51

Table 15: Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Pulmozyme, 2017 53

Table 16: Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Kalydeco, 2017 55

Table 17: Genetic Disorders Market, Global, CFTR Gene Mutations Responsive to Kalydeco, 2017 55

Table 18: Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Elaprase, 2017 57

Table 19: Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Vimizim, 2017 58

Table 20: Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Cerdelga, 2017 60

Table 21: Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Cerdelga, 2017 61

Table 22: Genetic Disorders Market, Global, Annual Revenue Forecast for Key Products ($bn), 2016–2023 94

Table 23: Genetic Disorders Market, Global, Usage of Generics Across Key Indications, 2017 96

Table 24: Genetic Disorders Market, Global, Forecast Revenue by Company, 2016–2023 105

Table 25: Genetic Disorders Market, Global, Licensing Deals, 2006–2017 126

Table 26: Genetic Disorders Market Therapeutics Market, Global, Co-development Deals, 2006–2017 134

Figure 1: Genetic Disorders, CF Testing Algorithm 18

Figure 2: Epidemiology Patterns for Cystic Fibrosis, 2016–2023 27

Figure 3: Epidemiology Patterns for Duchenne Muscular Dystrophy, 2016–2023 28

Figure 4: Epidemiology Patterns for Lysosomal Storage Disease, 2016–2023 29

Figure 5: Epidemiology Patterns for Paroxysmal Nocturnal Hemoglobinuria, 2016–2023 29

Figure 6: Genetic Disorders, Progression of CF 34

Figure 7: Genetic Disorders, Schema for Initiation of Glucocorticoid Treatment in Duchenne Muscular Dystrophy, 2017 40

Figure 8: Genetic Disorders, Global, Key Marketed Products and Approved Indications, 2017 44

Figure 9: Genetic Disorders Market, Global, Annual Revenue for Soliris ($bn), 2007–2023 46

Figure 10: Genetic Disorders Market, Global, Annual Revenue for Orkambi ($bn), 2015–2023 47

Figure 11: Genetic Disorders Market, Global, Annual Revenue for Cerezyme ($bn), 2006–2023 49

Figure 12: Genetic Disorders Market, Global, Annual Revenue for Myozyme/Lumizyme ($bn), 2006–2023 51

Figure 13: Genetic Disorders Market, Global, Annual Revenue for Fabrazyme ($bn), 2006–2023 53

Figure 14: Genetic Disorders Market, Global, Annual Revenue for Pulmozyme ($bn), 2006–2023 54

Figure 15: Genetic Disorders Market, Global, Annual Revenue for Kalydeco ($bn), 2012–2023 56

Figure 16: Genetic Disorders Market, Global, Annual Revenue for Elaprase ($bn), 2006–2023 58

Figure 17: Genetic Disorders Market, Global, Annual Revenue for Vimizim ($bn), 2014–2023 59

Figure 18: Genetic Disorders Market, Global, Annual Revenue for Cerdelga ($bn), 2014–2023 61

Figure 19: Genetic Disorders Market, Global, Annual Revenue for Exondys 51 ($bn), 2016–2023 62

Figure 20: Genetic Disorders Market, Global, Overall Pharmaceutical Industry Pipeline by Genetic Disorders, 2017 63

Figure 21: Genetic Disorders Market, Global, Genetic Disorders Pipeline by Stage of Development and Molecule Type, 2017 64

Figure 22: Genetic Disorders Market, Global, Pipeline for Key Genetic Disorders Indications by Stage of Development, 2017 65

Figure 23: Genetic Disorders Market, Global, Pipeline for Key Genetic Disorders Indications by Molecule Type, 2017 66

Figure 24: Genetic Disorders Market, Global, Genetic Disorders Pipeline by Molecular Target, 2017 67

Figure 25: Genetic Disorders Market, Global, Pipeline for Key Genetic Disorders Indications by Molecular Target, 2017 68

Figure 26: Genetic Disorders Market, Global, Clinical Trial Attrition Rates by Stage of Development (%), 2006–2017 69

Figure 27: Genetic Disorders Market, Global, Clinical Trial Attrition Rates by Stage of Development and Indication (%), 2006–2017 70

Figure 28: Genetic Disorders Market, Global, Clinical Trial Attrition Rates by Stage of Development and Molecule Type (%), 2006–2017 71

Figure 29: Genetic Disorders Market, Global, Clinical Trial Attrition Rates by Stage of Development and Molecular Target (%), 2006–2017 72

Figure 30: Genetic Disorders Market, Global, Clinical Trial Duration by Stage of Development (months), 2006–2017 73

Figure 31: Genetic Disorders Market, Global, Clinical Trial Duration by Stage of Development and Indication (months), 2006–2017 74

Figure 32: Genetic Disorders Market, Global, Clinical Trial Duration by Stage of Development and Molecule Type (months), 2006–2017 75

Figure 33: Genetic Disorders Market, Global, Clinical Trial Duration by Stage of Development and Molecular Target (months), 2006–2017 76

Figure 34: Genetic Disorders Market, Global, Clinical Trial Size by Stage of Development (participants), 2006–2017 77

Figure 35: Genetic Disorders Market, Global, Clinical Trial Size by Stage of Development and Indication (participants), 2006–2017 78

Figure 36: Genetic Disorders Market, Global, Clinical Trial Size by Stage of Development and Molecule Type (participants), 2006–2017 79

Figure 37: Genetic Disorders Market, Global, Clinical Trial Size by Stage of Development and Molecular Target (participants), 2006–2017 80

Figure 38: Genetic Disorders Market, Global, Clinical Program Size by Stage of Development (participants), 2006–2017 81

Figure 39: Genetic Disorders Market, Global, Clinical Program Size by Stage of Development and Indication (participants), 2006–2017 82

Figure 40: Genetic Disorders Market, Global, Clinical Program Size by Stage of Development and Molecule Type (participants), 2006–2017 83

Figure 41: Genetic Disorders Market, Global, Clinical Program Size by Stage of Development and Molecular Target (participants), 2006–2017 84

Figure 42: Genetic Disorders Market, Global, Revenue Forecast for Epidiolex ($m), 2018–2023 86

Figure 43: Genetic Disorders Market, Global, Revenue Forecast for ALXN-1210 (ravulizumab) ($m), 2019–2023 87

Figure 44: Genetic Disorders Market, Global, Revenue Forecast for Lanadelumab ($m), 2018–2023 88

Figure 45: Genetic Disorders Market, Global, Revenue Forecast for Burosumab ($m), 2018–2023 90

Figure 46: Genetic Disorders Market, Global, Revenue Forecast for GBT440 (voxelotor) ($m), 2020–2023 91

Figure 47: Genetic Disorders Market, Global, Revenue Forecast for Ivacaftor and Tezacaftor ($m), 2018–2023 92

Figure 48: Genetic Disorders Market, Global, Market Size ($bn), 2016–2023 93

Figure 49: Genetic Disorders Market, Global, Annual Revenue Forecast for Premium and Generic Products ($bn), 2016–2023 95

Figure 50: Genetic Disorders Market, Global, Annual Revenue Forecast for Immune/Inflammatory Mediators ($bn), 2016–2023 97

Figure 51: Genetic Disorders Market, Global, Annual Revenue Forecast for Enzyme Replacement/Modifying Therapies ($bn), 2016–2023 98

Figure 52: Genetic Disorders Market, Global, Annual Revenue Forecast for Ion Channel Modulators ($bn), 2016–2023 99

Figure 53: Genetic Disorders Market, Global, Annual Revenue Forecast for G Protein-Coupled Receptor Modulators ($bn), 2016–2023 100

Figure 54: Genetic Disorders Market, Global, Annual Revenue Forecast for Metabolic Enzyme Mediators ($bn), 2016–2023 101

Figure 55: Genetic Disorders Therapeutics Market, Global, Company Analysis Matrix, 2016–2023 102

Figure 56: Genetic Disorders Market, Global, Cluster by Growth and Market Share, 2016–2023 103

Figure 57: Genetic Disorders Market, Global, Forecast Market Share by Company (%), 2016–2023 106

Figure 58: Genetic Disorders Market, Global, Companies by Compound Annual Growth Rate (%), 2016–2023 107

Figure 59: Genetic Disorders Market, Global, Revenues by Product Type, 2016–2023 108

Figure 60: Genetic Disorders Market, Global, Alexion Annual Revenue Forecast ($bn), 2016–2023 109

Figure 61: Genetic Disorders Market, Global, Sanofi Annual Revenue Forecast ($bn), 2016–2023 110

Figure 62: Genetic Disorders Market, Global, Vertex Annual Revenue Forecast ($bn), 2016–2023 111

Figure 63: Genetic Disorders Market, Global, Shire Annual Revenue Forecast ($bn), 2016–2023 112

Figure 64: Genetic Disorders Market, Global, Amgen Annual Revenue Forecast ($bn), 2016–2023 113

Figure 65: Genetic Disorders Market, Global, GW Pharmaceuticals Annual Revenue Forecast ($bn), 2016–2023 114

Figure 66: Genetic Disorders Market, Global, Pfizer Annual Revenue Forecast ($bn), 2016–2023 115

Figure 67: Genetic Disorders Market, Global, BioMarin Annual Revenue Forecast ($bn), 2016–2023 116

Figure 68: Genetic Disorders Market, Global, Sarepta Annual Revenue Forecast ($bn), 2016–2023 117

Figure 69: Genetic Disorders Market, Global, Companies by Type, 2017 118

Figure 70: Genetic Disorders Market, Global, Pipeline, High-Activity and Late-Stage Developers by Level of Genetic Disorders Specialization, 2017 119

Figure 71: Genetic Disorders Market, Global, Proportion of Total Company Revenue Attributed to Genetic Disorders, 2016–2023 120

Figure 72: Genetic Disorders Market, Global, Licensing Deals by Region, Year and Value, 2006–2017 122

Figure 73: Genetic Disorders Market, Global, Licensing Deals by Indication, 2006–2017 123

Figure 74: Genetic Disorders Market, Global, Licensing Deals by Stage of Development and Value, 2006–2016 124

Figure 75: Genetic Disorders Market, Global, Licensing Deals by Molecule Type and Molecular Target, 2006–2017 125

Figure 76: Genetic Disorders Market, Global, Co-development Deals by Region, Year and Value, 2006–2017 130

Figure 77: Genetic Disorders Market, Global, Co-development Deals by Indication, 2006–2017 131

Figure 78: Genetic Disorders Market, Global, Co-development Deals by Stage of Development and Value, 2006–2017 132

Figure 79: Genetic Disorders Market, Global, Co-development Deals by Molecule Type and Molecular Target, 2006–2017 133

Figure 80: Genetic Disorders, Global, Table of all Clinical Stage Pipeline Products, 2017, Part 1 147

Figure 81: Genetic Disorders, Global, Table of all Clinical Stage Pipeline Products, 2017, Part 2 148

Figure 82: Genetic Disorders, Global, Table of all Clinical Stage Pipeline Products, 2017, Part 3 149

Figure 83: Genetic Disorders, Global, Table of all Clinical Stage Pipeline Products, 2017, Part 4 150

Figure 84: Genetic Disorders, Global, Table of all Clinical Stage Pipeline Products, 2017, Part 5 151

Figure 85: Genetic Disorders, Global, Table of all Clinical Stage Pipeline Products, 2017, Part 6 152

Figure 86: Genetic Disorders, Global, Table of all Clinical Stage Pipeline Products, 2017, Part 7 153

Figure 87: Genetic Disorders, Global, Table of all Clinical Stage Pipeline Products, 2017, Part 8 154

Figure 88: Genetic Disorders, Global, Table of all Clinical Stage Pipeline Products, 2017, Part 9 155

Figure 89: Genetic Disorders, Global, Table of all Clinical Stage Pipeline Products, 2017, Part 10 156

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