Sections
Title | Starting Page | Number of Pages |
---|
Table of Contents | 4 | 8 |
List of Tables | 7 | 3 |
List of Figures | 10 | 2 |
Neurometabolic Disorders Market to 2017 - Introduction | 12 | 2 |
GBI Research Report Guidance | 13 | 1 |
Neurometabolic Disorders Market to 2017 - Global Market Overview | 14 | 6 |
Introduction | 14 | 1 |
Market Revenue | 14 | 2 |
Annual Cost of Therapy | 16 | 1 |
Treatment Usage Pattern | 17 | 1 |
Diseased Population | 18 | 1 |
Treatment Seeking Population | 18 | 1 |
Diagnosed Population | 18 | 1 |
Prescription Population | 19 | 1 |
Neurometabolic Disorders Market to 2017 - Geographical Landscape | 20 | 15 |
The US | 21 | 1 |
Revenue | 21 | 2 |
Annual Cost of Therapy | 23 | 1 |
Treatment Usage Pattern | 24 | 1 |
Diseased Population | 25 | 1 |
Treatment Seeking Population | 25 | 1 |
Diagnosed Population | 25 | 1 |
Prescription Population | 26 | 1 |
Top Five Countries of Europe | 26 | 1 |
Revenue | 26 | 2 |
Annual Cost of Therapy | 28 | 1 |
Treatment Usage Pattern | 29 | 1 |
Diseased Population | 30 | 1 |
Treatment Seeking Population | 30 | 1 |
Diagnosed Population | 30 | 1 |
Prescription Population | 30 | 1 |
Japan | 31 | 1 |
Revenue | 31 | 1 |
Annual Cost of Therapy | 32 | 1 |
Treatment Usage Pattern | 33 | 1 |
Diseased Population | 34 | 1 |
Treatment Seeking Population | 34 | 1 |
Diagnosed Population | 34 | 1 |
Prescription Population | 34 | 1 |
Neurometabolic Disorders Market to 2017 - Therapeutic Landscape | 35 | 47 |
Neurometabolic Disorders Market to 2017 - Gaucher s Disease | 35 | 1 |
Introduction | 35 | 1 |
Revenue | 36 | 2 |
Revenue by Geography | 38 | 2 |
Annual Cost of Therapy | 40 | 1 |
Treatment Usage Pattern | 41 | 1 |
Diseased Population | 41 | 1 |
Treatment Seeking Population | 42 | 1 |
Diagnosed Population | 42 | 1 |
Prescription Population | 42 | 1 |
Treatment Algorithm | 43 | 1 |
Gaucher s Type 1 Disease Therapeutics Market Drivers | 44 | 1 |
Promising Pipeline Molecules in Late stage of Development | 44 | 1 |
Increase in Awareness Levels among Gaucher s Patients | 44 | 1 |
Gaucher s Type 1 Disease Therapeutics Market Restraints | 45 | 1 |
Low Diagnosis Rate | 45 | 1 |
High Treatment Cost | 45 | 1 |
Neurometabolic Disorders Market to 2017 - Niemann-Pick Type C Disease | 45 | 1 |
Introduction | 45 | 2 |
Revenue | 47 | 1 |
Revenue by Geography | 48 | 2 |
Annual Cost of Therapy | 50 | 1 |
Treatment Usage Pattern | 51 | 1 |
Diseased Population | 51 | 1 |
Treatment Seeking Population | 52 | 1 |
Diagnosed Population | 52 | 1 |
Prescription Population | 52 | 1 |
Treatment Algorithm | 53 | 1 |
Niemann-Pick Type C Market Drivers | 54 | 1 |
Increase in Disease Awareness | 54 | 1 |
Increase in Drug Uptake in Europe and Other Countries | 55 | 1 |
Niemann-Pick Type C Market Restraints | 55 | 1 |
Lack of Approval and Reimbursement of Zavesca in the US and Japan | 55 | 1 |
Misdiagnosis | 55 | 1 |
Neurometabolic Disorders Market to 2017 - Pompe Disease | 56 | 1 |
Introduction | 56 | 1 |
Revenue | 56 | 2 |
Revenue by Geography | 58 | 2 |
Annual Cost of Therapy | 60 | 1 |
Treatment Usage Pattern | 61 | 1 |
Diseased Population | 62 | 1 |
Treatment Seeking Population | 62 | 1 |
Diagnosed Population | 62 | 1 |
Prescription Population | 63 | 1 |
Treatment Algorithm | 63 | 1 |
Pompe Disease Therapeutics Market Drivers | 64 | 1 |
Approval of Lumizyme and Strong Growth in Sales of Marketed Drugs | 64 | 1 |
Increase in Disease Awareness and Increased Support for Medical Research | 64 | 1 |
Pompe Disease Therapeutics Market Restraints | 65 | 1 |
Poor Diagnosis and Treatment Seeking Rate | 65 | 1 |
High Cost of Therapy | 65 | 1 |
Neurometabolic Disorders Market to 2017 - Fabry Disease | 65 | 1 |
Introduction | 65 | 1 |
Revenue | 65 | 2 |
Revenue by Geography | 67 | 2 |
Annual Cost of Therapy | 69 | 1 |
Treatment Usage Pattern | 70 | 1 |
Diseased Population | 71 | 1 |
Treatment Seeking Population | 71 | 1 |
Diagnosed Population | 71 | 1 |
Prescription Population | 71 | 1 |
Fabry Disease Therapeutics Market Drivers | 72 | 1 |
Promising Late Stage Pipeline Molecules (Pharmacological Chaperones) for the Treatment of Fabry Disease | 72 | 1 |
Increase in Awareness Levels of Disease Progression and Etiology | 72 | 1 |
Fabry Disease Therapeutics Market Restraints | 73 | 1 |
Misdiagnosis of Fabry Disease | 73 | 1 |
High Annual Cost of the Treatment | 73 | 1 |
Neurometabolic Disorders Market to 2017 - Mucopolysaccharidosis VI | 73 | 1 |
Introduction | 73 | 1 |
Revenue | 74 | 1 |
Revenue by Geography | 75 | 1 |
Annual Cost of Therapy | 76 | 1 |
Treatment Usage Pattern | 77 | 1 |
Diseased Population | 78 | 1 |
Treatment Seeking Population | 78 | 1 |
Diagnosed Population | 78 | 1 |
Prescription Population | 78 | 1 |
Treatment Algorithm | 79 | 1 |
Mucopolysaccharidosis VI Therapeutics Market Drivers | 80 | 1 |
Strong Performance by Existing Branded Drugs | 80 | 1 |
Increase in Disease Awareness | 80 | 1 |
Mucopolysaccharidosis VI Therapeutics Market Restraints | 81 | 1 |
Poor Diagnosis Rate | 81 | 1 |
Neurometabolic Disorders Market to 2017 - Major Marketed Products | 82 | 10 |
Gaucher s Disease | 82 | 1 |
Cerezyme | 82 | 1 |
Overview | 82 | 1 |
Mechanism of Action | 82 | 1 |
Efficacy Details | 82 | 1 |
Safety Issues | 83 | 1 |
VPRIV | 83 | 1 |
Overview | 83 | 1 |
Mechanism of Action | 84 | 1 |
Efficacy Details | 84 | 1 |
Safety Issues | 84 | 1 |
Gaucher s Disease and Niemann-Pick Type C | 85 | 1 |
Zavesca | 85 | 1 |
Overview | 85 | 1 |
Mechanism of Action | 85 | 1 |
Efficacy Details | 85 | 1 |
Safety Issues | 86 | 1 |
Pompe Disease | 86 | 1 |
Myozyme/Lumizyme | 86 | 1 |
Overview | 86 | 1 |
Mechanism of Action | 86 | 1 |
Efficacy Details | 87 | 1 |
Safety Issues | 88 | 1 |
Fabry Disease | 88 | 1 |
Fabrazyme | 88 | 1 |
Overview | 88 | 1 |
Mechanism of Action | 88 | 1 |
Efficacy Details | 88 | 1 |
Safety Issues | 89 | 1 |
Replagal | 89 | 1 |
Overview | 89 | 1 |
Mechanism of Action | 89 | 1 |
Efficacy Details | 89 | 1 |
Safety Issues | 90 | 1 |
Mucopolysaccharidosis VI | 91 | 1 |
Naglazyme | 91 | 1 |
Overview | 91 | 1 |
Mechanism of Action | 91 | 1 |
Efficacy Details | 91 | 1 |
Safety Issues | 91 | 1 |
Neurometabolic Disorders Market to 2017 - Product Pipeline Analysis | 92 | 11 |
Gaucher s Disease - Research and Development Pipeline | 94 | 1 |
Overview | 94 | 1 |
Pipeline by Phase | 94 | 1 |
Niemann-Pick Type C: Research and Development Pipeline | 95 | 1 |
Overview | 95 | 1 |
Pipeline by Phase | 95 | 1 |
Pompe Disease: Research and Development Pipeline | 96 | 1 |
Overview | 96 | 1 |
Pipeline by Phase | 96 | 1 |
Fabry Disease: Research and Development Pipeline | 97 | 1 |
Overview | 97 | 1 |
Pipeline by Phase | 97 | 1 |
Most Promising Molecules | 98 | 1 |
Uplyso | 98 | 1 |
Overview | 98 | 1 |
Mechanism of Action | 98 | 1 |
Research and Development Brief | 98 | 1 |
Discovery/Preclinical Trial Details | 99 | 1 |
Genz-112638 | 99 | 1 |
Overview | 99 | 1 |
Mechanism of Action | 99 | 1 |
Phase III Trials | 100 | 1 |
Phase II Trials | 100 | 1 |
Amigal | 101 | 1 |
Overview | 101 | 1 |
Mechanism of Action | 101 | 1 |
Phase III Trials | 101 | 1 |
Phase II Trials | 101 | 1 |
Phase I Trials | 102 | 1 |
Other Developmental Activities | 102 | 1 |
Neurometabolic Disorders Market to 2017- Competitive Landscape | 103 | 25 |
Overview | 103 | 2 |
Company Profiles | 105 | 1 |
Genzyme Corporation | 105 | 1 |
Overview | 105 | 1 |
SWOT Analysis | 105 | 1 |
Deals | 106 | 7 |
Shire | 113 | 1 |
Overview | 113 | 1 |
SWOT Analysis | 113 | 1 |
Deals | 114 | 6 |
BioMarin Pharmaceutical Inc. | 120 | 1 |
Overview | 120 | 1 |
SWOT Analysis | 121 | 1 |
Deals | 122 | 2 |
Actelion | 124 | 1 |
Overview | 124 | 1 |
SWOT Analysis | 125 | 1 |
Deals | 126 | 2 |
Neurometabolic Disorders Market to 2017 - Strategic Consolidations | 128 | 7 |
Overview | 128 | 1 |
Sanofi S.A. Completes Acquisition of Genzyme | 129 | 1 |
Deal Summary | 129 | 1 |
Deal Rationale | 129 | 1 |
Pfizer Enters into a Licensing Agreement with Protalix Biotherapeutics | 130 | 1 |
GlaxoSmithKline Enters into Licensing Agreement with Amicus Therapeutics for Amigal | 130 | 1 |
Amicus Therapeutics Terminates Licensing Agreement with Shire | 131 | 1 |
Deals by Geography | 131 | 1 |
Deals by Licensing Type | 132 | 1 |
Deals by Value | 133 | 1 |
Deals by Phase | 134 | 1 |
NDA Filed | 134 | 1 |
Taliglucerase alpha | 134 | 1 |
Phase III | 134 | 1 |
Amigal | 134 | 1 |
Neurometabolic Disorders Market to 2017 - Appendix | 135 | 8 |
Market Definitions | 135 | 1 |
Abbreviations | 135 | 1 |
Research Methodology | 136 | 2 |
Coverage | 136 | 1 |
Secondary Research | 137 | 1 |
Primary Research | 137 | 1 |
Therapeutic Landscape | 138 | 3 |
Epidemiology-based Forecasting | 138 | 2 |
Market Size by Geography | 140 | 1 |
Geographical Landscape | 141 | 1 |
Pipeline Analysis | 141 | 1 |
Competitive Landscape | 141 | 1 |
Expert Panel Validation | 141 | 1 |
Contact Us | 141 | 1 |
Disclaimer | 141 | 1 |
Sources | 142 | 1 |