CSL-654 (Hemophilia A and B)：市場予測と分析（～2022）...市場調査レポートについてご紹介

1 Table of Contents
1 Table of Contents 4
1.1 List of Tables 7
1.2 List of Figures 7
2 Introduction 8
2.1 Catalyst 8
2.2 Related Reports 9
2.3 Upcoming Related Reports 11
3 Disease Overview 12
3.1 Etiology and Pathophysiology 12
3.1.1 Etiology 12
3.1.2 Pathophysiology 13
3.1.3 Prognosis and Quality of Life 15
3.2 Symptoms 17
3.2.1 Hemophilia A and B 17
3.2.2 Inhibitors 18
4 Disease Management 19
4.1 Treatment Overview 19
4.1.1 Hemophilia A and B Diagnosis 20
4.1.2 Hemophilia A and B Treatment 20
4.1.3 Inhibitors Diagnosis 22
4.1.4 Inhibitors Treatment 22
5 Competitive Assessment 24
5.1 Overview 24
5.2 Strategic Competitive Assessment 25
6 Opportunity and Unmet Need 29
6.1 Overview 29
6.2 Opportunities Analysis 30
6.2.1 Longer-Lasting Recombinant Factors to Reduce the Frequency of Prophylactic Injections 30
6.2.2 Lower-Cost Recombinant Replacement Therapies 31
6.2.3 Lower Risk of Inhibitor Formation in Previously Untreated Patients 32
6.2.4 More Convenient Drug Administration 32
6.2.5 Oral Formulation of Recombinant Replacement Therapies 33
6.3 Unmet Needs Gap Analysis 34
6.3.1 Long-Acting FVIII Replacement Therapies 34
6.3.2 Gene Therapy Treatments for Hemophilia A and B 34
7 Pipeline Assessment 35
7.1 Overview 35
7.2 Promising Drugs in Clinical Development – Hemophilia A 35
7.3 Promising Drugs in Clinical Development – Hemophilia B 37
7.4 Promising Drugs in Clinical Development – Hemophilia A and B Patients with Inhibitors 39
8 CSL-654 (rIX-FP) 40
8.1 Overview 40
8.2 Efficacy 41
8.3 Safety 41
8.4 Dosing and Formulation 41
8.5 Potential Clinical Positioning 42
8.6 Potential Commercial Positioning 42
8.7 Pricing and Reimbursement 42
8.8 SWOT Analysis 43
8.9 Forecast 43
9 Appendix 45
9.1 Bibliography 45
9.2 Abbreviations 48
9.3 Methodology 51
9.4 Forecasting Methodology 51
9.4.1 Diagnosed Hemophilia Patients 51
9.4.2 Percent Drug-Treated Patients 52
9.4.3 General Pricing Assumptions 52
9.4.4 Pricing of Pipeline Agents 53
9.5 Physicians and Specialists Included in this Study 54
9.6 About the Authors 56
9.6.1 Authors 56
9.6.2 Global Head of Healthcare 57
9.7 About GlobalData 58
9.8 Disclaimer 58