Sections
Title | Starting Page | Number of Pages |
---|
Table of Contents | 5 | 6 |
List of Tables | 8 | 1 |
List of Figures | 9 | 2 |
Introduction | 11 | 10 |
Disease Introduction | 11 | 1 |
Epidemiology | 12 | 1 |
Symptoms | 12 | 1 |
Etiology and Pathophysiology | 13 | 1 |
Hemophilia A | 13 | 1 |
Hemophilia B | 13 | 1 |
Diagnosis | 14 | 1 |
Diagnosis of Inhibitors | 14 | 1 |
Disease stages | 15 | 1 |
Prognosis | 15 | 1 |
Treatment Options | 15 | 1 |
Management of Bleeding with Factor Replacement Therapy | 16 | 2 |
Antifibrinolytic Medication | 18 | 1 |
Treatment of Inhibitors | 18 | 1 |
High-Dose Clotting Factor Concentrates | 18 | 1 |
Bypassing Agents | 18 | 1 |
Immune Tolerance Induction Therapy | 18 | 1 |
Gene Therapy | 19 | 1 |
Hemophilia A | 19 | 1 |
Hemophilia B | 19 | 2 |
Marketed Products | 21 | 21 |
Overview | 21 | 1 |
Marketed Products Hemophilia A | 21 | 1 |
Advate (octacog alfa) Baxalta | 21 | 2 |
Adynovate (rurioctocog alfa pegol) Baxalta | 23 | 1 |
Xyntha/ReFacto AF (moroctocog alfa) Pfizer | 24 | 1 |
Kogenate FS (octacog alfa) Bayer | 25 | 1 |
Kovaltry (octacog alfa) Bayer | 26 | 2 |
Eloctate (efmoroctocog Alfa) Biogen | 28 | 1 |
Nuwiq (simoctocog alfa) Octapharma | 29 | 1 |
NovoEight (turoctocog alfa) Novo Nordisk | 30 | 2 |
GreenGene F (Beroctocog Alfa) Green Cross | 32 | 1 |
Marketed Products Hemophilia B | 33 | 1 |
Alprolix (Eftrenonacog alfa) Biogen | 33 | 1 |
BeneFix (nonacog alfa) Pfizer | 34 | 2 |
Rixubis (nonacog gamma) Baxter | 36 | 1 |
Idelvion (albutrepenonacog alfa) CSL Behring | 36 | 2 |
Marketed Products Hemophilia A and B | 38 | 1 |
Feiba NF (anti-inhibitor coagulant complex) Baxter | 38 | 1 |
Byclot (freeze-dried-activated human blood coagulation factor VII concentrate containing factor X) Kaketsuken | 39 | 1 |
Comparative Efficacy and Safety of Marketed Products | 40 | 2 |
Pipeline Analysis | 42 | 21 |
Overview | 42 | 1 |
Pipeline by Stage of Development, Molecule Type, Route of Administration and Program Type | 42 | 2 |
Pipeline by Molecular Target | 44 | 1 |
Promising Pipeline Candidates Hemophilia A | 45 | 1 |
BAX-801 (susoctocog alfa) Baxalta | 45 | 2 |
CSL-627/NBP-601 (Lonoctocog alfa) CSL Behring | 47 | 2 |
Emicizumab (ACE-910/RG6013) Roche | 49 | 2 |
N8-GP (turoctocog Alfa Pegol) Novo Nordisk | 51 | 2 |
BAY94-9027 (damoctocog alfa pegol) Bayer | 53 | 2 |
Promising Pipeline Candidates Hemophilia B | 55 | 1 |
N9-GP/NN-7999 (nonacog beta pegol) Novo Nordisk | 55 | 2 |
Promising Pipeline Candidates Hemophilia A and B | 57 | 1 |
BAX-817 (recombinant activated factor VII BI) Baxalta | 57 | 1 |
CSL-689 (recombinant activated VII-FP) CSL Behring | 58 | 1 |
Comparative Efficacy and Safety of Pipeline Products | 59 | 1 |
Product Competitiveness Framework | 60 | 3 |
Clinical Trial Analysis | 63 | 10 |
Failure Rate | 63 | 1 |
Overall Failure Rate | 63 | 1 |
Failure Rate by Phase and Molecule Type | 64 | 1 |
Failure Rate by Phase and Molecular Target | 64 | 1 |
Clinical Trial Duration | 65 | 1 |
Clinical Trial Duration by Molecule Type | 65 | 1 |
Clinical Trial Duration by Molecular Target | 66 | 1 |
Clinical Trial Size | 67 | 1 |
Patient Enrollment per Product by Molecule Type, Molecular Target and Stage of Development | 67 | 2 |
Patient Enrollment per Trial by Molecule Type, Molecular Target and Stage of Development | 69 | 2 |
Competitive Clinical Trials Metrics Analysis | 71 | 2 |
Multi-scenario Forecast | 73 | 20 |
Geographical Markets | 73 | 1 |
Asia-Pacific Market | 73 | 3 |
India | 76 | 1 |
Treatment Usage Patterns | 76 | 1 |
Annual Cost of Therapy | 77 | 1 |
Market Size | 78 | 2 |
China | 80 | 1 |
Treatment Usage Patterns | 80 | 1 |
Annual Cost of Therapy | 81 | 1 |
Market Size | 82 | 1 |
Australia | 83 | 1 |
Treatment Usage Patterns | 83 | 1 |
Annual Cost of Therapy | 84 | 1 |
Market Size | 85 | 2 |
South Korea | 87 | 1 |
Treatment Usage Patterns | 87 | 1 |
Annual Cost of Therapy | 88 | 1 |
Market Size | 89 | 1 |
Japan | 90 | 1 |
Treatment Usage Patterns | 90 | 1 |
Annual Cost of Therapy | 90 | 1 |
Market Size | 91 | 2 |
Drivers and Barriers | 93 | 2 |
Drivers | 93 | 1 |
Increasing Awareness | 93 | 1 |
Promising Late-Stage Pipeline Products with Potential to Change Treatment Paradigm | 93 | 1 |
Continued Uptake of Recently Approved Therapies | 93 | 1 |
Increasing Usage of Prophylactic Therapies | 94 | 1 |
Barriers | 94 | 1 |
Low Diagnosis and Low Treatment Rate | 94 | 1 |
Low Access to Factor Replacement Therapies | 94 | 1 |
Limitation of Premium Pricing | 94 | 1 |
Deals and Strategic Consolidations | 95 | 11 |
Licensing Deals | 95 | 1 |
Deals by Region and Value | 95 | 1 |
Number of Disclosed and Undisclosed Deals by Year, Aggregate Deal Value | 95 | 1 |
Deal Value by Stage of Development, Molecule Type, and Molecular Target | 96 | 3 |
Key Licensing Deals | 99 | 1 |
Licensing Agreement between Baxter and Archemix | 99 | 1 |
Licensing Agreement between Dimension Therapeutics and Bayer | 99 | 1 |
Licensing Agreement between Baxter and Xenetic | 99 | 1 |
Licensing Agreement between Bayer and CSL Behring | 100 | 1 |
Co-development Deals | 100 | 1 |
Deals by Region and Value | 100 | 1 |
Number of Disclosed and Undisclosed Deals by Year, Aggregate Deal Value | 101 | 1 |
Deal Value by Stage of Development, Molecule Type, and Molecular Target | 101 | 3 |
Key Co-development Deals | 104 | 1 |
Co-development Agreement between Biogen, Fondazione Telethon and Ospedale San Raffaele | 104 | 1 |
Co-development Agreement between Spark Therapeutics and Pfizer | 104 | 1 |
Co-development Agreement between Catalyst and ISU Abxis | 105 | 1 |
Co-development Agreement between Baxter and Chatham | 105 | 1 |
Appendix | 106 | 21 |
All Pipeline Drugs by Stage of Development | 106 | 4 |
Discovery | 106 | 1 |
Preclinical | 106 | 2 |
Phase I | 108 | 1 |
Phase II | 108 | 1 |
Phase III | 109 | 1 |
Pre-registration | 109 | 1 |
Market Forecasts to 2021 | 110 | 4 |
Asia-Pacific | 110 | 1 |
India | 110 | 1 |
China | 111 | 1 |
Australia | 112 | 1 |
South Korea | 112 | 1 |
Japan | 113 | 1 |
Bibliography | 114 | 8 |
Abbreviations | 122 | 1 |
Research Methodology | 122 | 4 |
Secondary Research | 122 | 1 |
Marketed Product Profiles | 123 | 1 |
Late-Stage Pipeline Candidates | 123 | 1 |
Comparative Efficacy and Safety Heatmap for Marketed and Pipeline Products | 123 | 1 |
Product Competitiveness Framework | 123 | 1 |
Pipeline Analysis | 123 | 1 |
Overall Pipeline | 123 | 1 |
Clinical Trials | 124 | 1 |
Failure Rate | 124 | 1 |
Clinical Trial Size | 124 | 1 |
Clinical Trial Duration | 124 | 1 |
Clinical Trial Endpoint Analysis | 124 | 1 |
Forecasting Model | 124 | 1 |
Deals Data Analysis | 125 | 1 |
Contact Us | 126 | 1 |
Disclaimer | 126 | 1 |