Back to Report Store Home

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: Dec-2017
  • Report Code: GBIHC464MR
  • Report Format: pdf

Description

Table of Contents

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

License Details
Electronic PDF copy delivered via email. Report can be used by individual purchaser only.
Single User Licence
$4995
Electronic PDF copy delivered via email. Report can be shared by unlimited users within one corporate location, e.g. a regional office.
Site Licence
$9990
Electronic PDF copy delivered via email. Report can be shared globally by unlimited users within the purchasing corporation e.g. all employees of a single company.
Global Licence
$14985
Email to a colleague Enquire Before Buying
Prefer to speak to a team member? Call us on:
+44 161 359 5414
Or click here to contact us
Secure Payments
Cards