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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

List of Figures

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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  7. Reference to any product or service on the Site does not constitute an offer to sell or supply that product or service and does not mean that the product or service is available in all countries or that the name and description of any product in your country will be the same as that included on the Site.

  8. We have taken every care in the preparation of the Site. However, as certain technical matters may be beyond our control, we cannot guarantee that you will have uninterrupted or error-free access to the Site at all times.

  9. By submitting personal information via any of the online forms on the site, you consent to being contacted (either by phone or email) by a GBI Research representative.

  10. We make no warranty that the contents of the Site are free from infection by viruses or anything else which has contaminating or destructive properties and shall have no liability in respect thereof.

  11. We reserve the right at any time, without liability for doing so, to modify or discontinue, temporarily or permanently, any of the content, functions or services we offer on the Site (or any part of them) with or without notice. This includes removing information transmitted by you to us.

  12. You agree that the information you provide us with when you use the Site will be true and complete and that you will keep it true and complete.

Use of the Site
You agree not to use the services we provide via the Site to:

  1. Transmit any information or do anything that is unlawful, harmful, threatening, abusive, harassing, tortious, defamatory, vulgar, obscene, libellous, invasive of another's privacy, hateful, or racially, ethnically or otherwise objectionable;

  2. Impersonate any person or entity, or falsely state or otherwise misrepresent your affiliation with a person or entity;

  3. Forge headers or otherwise manipulate identifiers in order to disguise the origin of any information transmitted through the services

  4. Transmit any information that you do not have a right to transmit or which infringes another's rights;

  5. Transmit any unsolicited or unauthorised promotion (including without limitation "junk mail", "spam", "chain letters" or "pyramid schemes");

  6. Transmit any material that contains software viruses or any other computer code, files or programs designed to interrupt, damage, destroy or limit the functionality of any computer software or hardware or telecommunications equipment;

  7. Interfere with or disrupt the services or servers or networks connected to the services, or disobey any requirements, procedures, policies or regulations of networks connected to the services; or collect or store personal data about other users.

Amendment of the Terms and conditions

We reserve the right to amend these Terms without notice to you from time to time. Any such amendment shall be effective once the revised terms have been posted on the Site.

Applicable Law

We reserve the right to amend these Terms without notice to you from time to time. Any such amendment shall be effective once the revised terms have been posted on the Site.

Comments

If you have any comments or questions about the Site please contact us at clientservices@gbiresearch.com

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