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PharmaPoint: Hemophilia A and B - Global Drug Forecast and Market Analysis to 2026

Published By :

GlobalData

Published Date : Jul 2017

Category :

Pharmaceutical

No. of Pages : 404 Pages

PharmaPoint: Hemophilia A and B - Global Drug Forecast and Market Analysis to 2026

Summary

Hemophilia A and B are rare recessive X-linked genetic deficiencies in the blood clotting factors VIII and IX, respectively, and are characterized by the failure of blood to form normal clots after damage to veins and tissue. In the 7MM, patients with severe forms of the disease are increasingly treated on a prophylactic basis rather than on demand after bleeds. The frequent prophylactic infusions of rFVIII or rFIX begin from the first one or two years of life, often continuing through adulthood. However, the burden on patients and their families of maintaining the prophylactic treatment schedule is high.

Traditionally, the hemophilia market has been dominated by short-acting rFVIII or FIX concentrates. However, in recent years the hemophilia market has become increasingly competitive due to the approval of extended half-life products that can reduce the burden associated with prophylaxis. These improvements have mainly benefited hemophilia B patients, while a substantial unmet need remains for hemophilia A patients and patients with inhibitors who do not respond to standard treatments.

The hemophilia pipeline activity is strong and includes two candidates, Roches emicizumab (ACE-910) and Alnylams fitusiran, which do not exploit a replacement strategy but target different effectors of the coagulation cascade and are expected to cause a paradigm shift in the treatment of the disease.

Key Questions Answered

- What are the key drivers behind the increasing uptake and market penetration of the long-acting factor concentrates in hemophilia A and B, since their approval in 2014, and what is the trend in the use of long-acting factors in the forecast period?
- The hemophilia market is characterized by a number of unmet needs. What are the main unmet needs in this market? Will the pipeline drugs under development fulfil these unmet needs?
- What impact will the approval of Roches emicizumab have on the hemophilia market? What will the drugs peak sales be, and why?

Scope

- Overview of hemophilia, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and disease management.
- Annualized hemophilia therapeutics market revenue, cost of therapy per patient, and treatment usage patterns in nine patient segments and two treatment strategies (episodic and prophylactic treatment), forecast from 2016 to 2026.
- Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping, and implications for the hemophilia therapeutics market.
- Pipeline analysis: comprehensive data assessing emerging trends and mechanisms of action under development for hemophilia. The most promising candidate in Phase III development is profiled.
- Analysis of the current and future market competition in the global hemophilia market. Insightful review of the key industry drivers, restraints and, challenges. Each trend is independently researched to provide qualitative analysis of its implications.

Reasons to buy

The report will enable you to -
- Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.
- Develop business strategies by understanding the trends shaping and driving the global hemophilia market.
- Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global hemophilia market in the future.
- Formulate effective sales and marketing strategies by understanding the competitive landscape and by analysing the performance of various competitors.
- Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
- Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments, and strategic partnerships.
1 Table of Contents
1 Table of Contents 2
1.1 List of Tables 7
1.2 List of Figures 11
2 Executive Summary 12
2.1 Sales for Hemophilia A and B Recombinant Therapies by Region, 2016-2026 13
2.2 Long-Acting rFVIIIs Have Not Lived Up to Expectations 14
2.3 Inhibitor Segment to Devalue Post-Alternative Coagulation Promoter Launch 16
2.4 Roches Emicizumab to Radically Impact Hemophilia A and Inhibitor Segments 16
2.5 Treatment Cost to Remain a Major Unmet Need 16
2.6 New Comers to Take Over Market Space From Established Companies 17
2.7 What Do Physicians Think? 18
3 Introduction 19
3.1 Catalyst 19
3.2 Related Reports 20
3.3 Upcoming Related Reports 21
4 Disease Overview 22
4.1 Etiology and Pathophysiology 22
4.1.1 Etiology 22
4.1.2 Pathophysiology 23
4.2 Classification 25
4.3 Symptoms 27
4.3.1 Hemophilia A and B 27
4.3.2 Inhibitors 27
4.4 Prognosis and Quality of Life 28
5 Epidemiology 30
5.1 Disease Background 30
5.2 Risk Factors and Comorbidities 30
5.3 Global and Historical Trends 31
5.4 Forecast Methodology 33
5.4.1 Sources 33
5.4.2 Forecast Assumptions and Methods 35
5.4.3 Diagnosed Prevalent Cases 36
5.4.4 Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B by Severity 37
5.4.5 Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B by Inhibitors 40
5.5 Epidemiological Forecast for Hemophilia A (2016-2026) 41
5.5.1 Diagnosed Prevalent Cases of Hemophilia A 41
5.5.2 Age-Specific Diagnosed Prevalent Cases of Hemophilia A 41
5.5.3 Sex-Specific Diagnosed Prevalent Cases of Hemophilia A 42
5.5.4 Diagnosed Prevalent Cases of Hemophilia A by Severity 43
5.5.5 Diagnosed Prevalent Cases of Hemophilia A with Inhibitors 44
5.6 Epidemiological Forecast for Hemophilia B (2016-2026) 44
5.6.1 Diagnosed Prevalent Cases of Hemophilia B 44
5.6.2 Age-Specific Diagnosed Prevalent Cases of Hemophilia B 45
5.6.3 Sex -Specific Diagnosed Prevalent Cases of Hemophilia B 46
5.6.4 Diagnosed Prevalent Cases of Hemophilia B by Severity 46
5.6.5 Diagnosed Prevalent Cases of Hemophilia B with Inhibitors 47
5.7 Epidemiological Forecast for Hemophilia A and Hemophilia B (2016-2026) 47
5.7.1 Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B 48
5.7.2 Age-Specific Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B 49
5.7.3 Sex -Specific Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B 50
5.7.4 Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B by Severity 50
5.7.5 Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B with Inhibitors 51
5.8 Discussion 51
5.8.1 Epidemiological Forecast Insight 52
5.8.2 Limitations of the Analysis 52
5.8.3 Strengths of the Analysis 53
6 Disease Management 54
6.1 Treatment Overview 54
6.1.1 Treatment Guidelines 54
6.1.2 Clinical Practice 55
6.2 US 71
6.2.1 Clinical Practice 71
6.3 France 75
6.3.1 Clinical Practice 75
6.4 Germany 77
6.4.1 Clinical Practice 77
6.5 Italy 81
6.5.1 Clinical Practice 81
6.6 Spain 85
6.6.1 Clinical Practice 85
6.7 UK 88
6.7.1 Clinical Practice 88
6.8 Japan 91
6.8.1 Clinical Practice 91
7 Competitive Assessment 97
7.1 Overview 97
7.2 Product Profiles - Hemophilia A 101
7.2.1 Short Acting rFVIII 101
7.2.2 Long Acting rFVIII 138
7.2.3 Plasma-Derived FVIII Concentrates 156
7.2.4 Desmopressin Acetate 158
7.3 Product Profiles - Hemophilia B 159
7.3.1 Short Acting rFIX 159
7.3.2 Long Acting FIX 174
7.3.3 Plasma-Derived FIX Concentrates 190
7.4 Product Profiles - Hemophilia A and B with Inhibitors 191
7.4.1 NovoSeven RT (Eptacog Alfa) 191
7.4.2 Feiba NF (Activated Prothrombin Complex Concentrate) 197
8 Unmet Needs and Opportunities 202
8.1 Overview 202
8.2 Reduction in Risk of Inhibitor Development in Previously Untreated Patients 203
8.2.1 Unmet Need 203
8.2.2 Gap Analysis 204
8.2.3 Opportunity 205
8.3 More Effective Treatments for Patients with Inhibitors 205
8.3.1 Unmet Need 205
8.3.2 Gap Analysis 205
8.3.3 Opportunity 206
8.4 Longer-Lasting Agents and More Convenient Administration Routes 207
8.4.1 Unmet Need 207
8.4.2 Gap Analysis 209
8.4.3 Opportunity 209
8.5 Treatments to Cure the Disease 210
8.5.1 Unmet Need 210
8.5.2 Gap Analysis 210
8.5.3 Opportunity 211
8.6 Decreasing the Costs Associated with Prophylaxis 211
8.6.1 Unmet Need 211
8.6.2 Gap Analysis 213
8.6.3 Opportunity 213
8.7 Individualized Prophylaxis 213
8.7.1 Unmet Need 213
8.7.2 Gap Analysis 214
8.7.3 Opportunity 214
9 Pipeline Assessment 215
9.1 Overview 215
9.2 Late-Stage Development Candidates - Hemophilia A 219
9.2.1 BAY 94-9027 (Damoctocog Alfa Pegol) 220
9.2.2 N8-GP (Turoctocog Alfa Pegol) 226
9.2.3 Emicizumab (ACE-910, RG-6013) 232
9.2.4 Fitusiran (ALN-AT3, SAR-439774) 242
9.3 Late-Stage Development Candidates - Hemophilia B 249
9.4 Late-Stage Development Candidates - Hemophilia A and B Patients with Inhibitors 250
9.4.1 LR-769 (rhFVIIa) 252
9.4.2 Obizur (Susoctocog Alfa) 256
9.4.3 CSL-689 (rVIIa-FP) 261
9.4.4 BAX-817 (rFVIIa BI) 267
9.5 Promising Approaches in Early-Stage Development 269
9.5.1 Alternative Coagulation Promoters - Tissue Factor Pathway Inhibitors 270
9.5.2 Gene Therapies 272
9.5.3 Replacement Strategies 278
9.5.4 Strategies to Prevent and Manage Inhibitors 280
9.5.5 Other Drugs in Development 280
10 Current and Future Players 282
10.1 Overview 282
10.2 Trends in Corporate Strategy 286
10.3 Company Profiles 287
10.3.1 Bayer 287
10.3.2 Bioverativ 289
10.3.3 CSL Behring 292
10.3.4 Novo Nordisk 294
10.3.5 Pfizer 297
10.3.6 Roche 299
10.3.7 Shire 301
11 Market Outlook 305
11.1 Global Markets 305
11.1.1 Forecast 305
11.1.2 Drivers and Barriers - Global Market 307
11.2 US 309
11.2.1 Forecast 310
11.2.2 Key Events 313
11.2.3 Drivers and Barriers 313
11.3 5EU 315
11.3.1 Forecast 315
11.3.2 Key Events 319
11.3.3 Drivers and Barriers - 5EU 319
11.3.4 Drivers and Barriers - France 321
11.3.5 Drivers and Barriers - Germany 321
11.3.6 Drivers and Barriers - Italy 323
11.3.7 Drivers and Barriers - Spain 324
11.3.8 Drivers and Barriers - UK 325
11.4 Japan 327
11.4.1 Forecast 327
11.4.2 Key Events 330
11.4.3 Drivers and Barriers 330
12 Appendix 333
12.1 Bibliography 333
12.2 Abbreviations 368
12.3 Methodology 373
12.4 Forecasting Methodology 373
12.4.1 Diagnosed Hemophilia Patients 373
12.4.2 Percent Drug-Treated Patients 374
12.4.3 Drugs Included in Each Therapeutic Class 374
12.4.4 Launch and Patent Expiry Dates 376
12.4.5 General Pricing Assumptions 378
12.4.6 Individual Drug Assumptions 379
12.4.7 Pricing of Pipeline Agents 394
12.5 Primary Research - KOLs Interviewed for this Report 395
12.6 Primary Research - Prescriber Survey 398
12.7 Real-World Data 399
12.8 About the Authors 399
12.8.1 Analyst 399
12.8.2 Reviewer 400
12.8.3 Therapy Area Director 400
12.8.4 Epidemiologists 400
12.8.5 Reviewers 401
12.8.6 Global Director of Therapy Analysis and Epidemiology 401
12.8.7 Global Head and EVP of Healthcare Operations and Strategy 402
12.9 About GlobalData 403
12.10 Contact Us 403
12.11 Disclaimer 404

1.1 List of Tables
Table 1: Hemophilia A and B: Key Metrics in the Seven Major Markets, 2016-2026 12
Table 2: Classification of Hemophilia A and B 26
Table 3: Symptoms of Hemophilia A and B 28
Table 4: Relationship of Bleeding Severity with Clotting Factor Level. 30
Table 5: Comorbidities for Hemophilia 31
Table 6: 7MM, Diagnosed Prevalent Cases of Hemophilia A, Both Sexes, All Ages, N, Selected Years 2016-2026. 41
Table 7: 7MM, Diagnosed Prevalent Cases of Hemophilia B, Both Sexes, All Ages, N, Selected Years 2016-2026. 44
Table 8: 7MM, Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B, Both Sexes, All Ages, N, Selected Years 2016-2026. 48
Table 9: 7MM Treatment Guidelines for Hemophilia 55
Table 10: Most Prescribed Hemophilia A and B Drugs in the Global Markets, 2016 60
Table 11: Definition of Therapeutic Protocols in Hemophilia 62
Table 12: Country Profile - US, 2016 71
Table 13: Country Profile - France, 2016 75
Table 14: Country Profile - Germany, 2016 77
Table 15: Country Profile - Italy, 2016 81
Table 16: Country Profile - Spain, 2016 85
Table 17: Country Profile - UK, 2016 88
Table 18: Country Profile - Japan, 2016 91
Table 19: Leading Replacement Therapies for Hemophilia A and B and Patients with Inhibitors, 2017 101
Table 20: Product Profile - Recombinate 102
Table 21: Summary of the Results of the Pivotal Trial of Recombinate 103
Table 22: Recombinate SWOT Analysis, 2017 104
Table 23: Product Profile - Kogenate FS 105
Table 24: Summary of the Results from the Trial that Led to the Approval of Kogenate FS 108
Table 25: Summary of the Results from the Trial that Studies Kogenate FS in Pediatric Prophylaxis 109
Table 26: Kogenate FS SWOT Analysis, 2017 109
Table 27: Product Profile -Helixate FS 111
Table 28: Helixate FS SWOT Analysis, 2017 113
Table 29: Product Profile - Advate 113
Table 30: Summary of the Results from the Part 2 Phase II/III Pivotal Trial of Advate 117
Table 31: Summary of the Results from the Phase IV Prophylaxis Study of Advate 118
Table 32: Advate SWOT Analysis, 2017 119
Table 33: Product Profile - Kovaltry 120
Table 34: Summary of the LEOPOLD I and II Trials of Kovaltry 123
Table 35: Summary of the LEOPOLD Kids Trial of Kovaltry 124
Table 36: Kovaltry SWOT Analysis, 2017 125
Table 37: Product Profile - Xyntha/ReFacto AF 126
Table 38: Summary of the Pivotal Trial of Xyntha 128
Table 39: Xyntha/ReFacto AF SWOT Analysis, 2017 128
Table 40: Product Profile - NovoEight 129
Table 41: Summary of the Results of The Guardian 1 Trial of NovoEight 131
Table 42: Summary of the Results of The Guardian 3 Trial of NovoEight 132
Table 43: NovoEight SWOT Analysis, 2017 133
Table 44: Product Profile - Nuwiq 134
Table 45: Summary of the Results from the GENA-01, GENA-08, and GENA-03 Trials of Nuwiq 136
Table 46: Nuwiq SWOT Analysis, 2017 137
Table 47: Product Profile - Eloctate 140
Table 48: Summary of the Results of the A-LONG Trial of Eloctate 143
Table 49: Eloctate SWOT Analysis, 2017 145
Table 50: Product Profile - Adynovate 146
Table 51: Summary of the Results of the PROLONG-ATE Trial of Adynovate 148
Table 52: Summary of the Results of the BAX855 Pediatric Trial of Adynovate 149
Table 53: Adynovate SWOT Analysis, 2017 151
Table 54: Product Profile - Afstyla 152
Table 55: Summary of the Results of the AFFINITY and Pediatric Trials of Afstyla 154
Table 56: Afstyla SWOT Analysis, 2017 156
Table 57: Marketed Plasma-Derived Replacement Therapies for Hemophilia A Patients, 2017 158
Table 58: Product Profile - BeneFIX 160
Table 59: Summary of the Results of the Phase III Trial of BeneFIX 161
Table 60: Summary of the Results that Proved the Effectiveness of BeneFIX as Prophylaxis Regimen 162
Table 61: Summary of the Results of the Phase III BeneFIX 1010 Study 162
Table 62: BeneFIX SWOT Analysis, 2017 164
Table 63: Product Profile - Rixubis 165
Table 64: Summary of the Results of the Pivotal Trial of Rixubis 167
Table 65: Summary of the Results of the Pediatric Trial of Rixubis 167
Table 66: Rixubis SWOT Analysis, 2017 169
Table 67: Product Profile - IXinity 170
Table 68: Summary of the Results of the Pivotal Trial of IXinity 173
Table 69: IXinity SWOT Analysis, 2017 174
Table 70: Product Profile - Alprolix 175
Table 71: Summary of the Results of the B-LONG Trial of Alprolix 178
Table 72: Summary of the Results of the Kids B-Long Trial of Alprolix 179
Table 73: Alprolix SWOT Analysis, 2017 180
Table 74: Product Profile - Idelvion 181
Table 75: Summary of the Results of the CSL654_3001 Trial of Idelvion 183
Table 76: Summary of the Results of the CSL654_3002 Trial of Idelvion 184
Table 77: Idelvion SWOT Analysis, 2017 184
Table 78: Product Profile - Rebynin 185
Table 79: Results of the Paradigm 2 Trial of Rebynin 187
Table 80: Results of the Pediatric Paradigm 5 Trial of Rebynin 188
Table 81: Rebynin SWOT Analysis, 2017 189
Table 82: Marketed Plasma-Derived Replacement Therapies for Hemophilia B Patients, 2017 190
Table 83: Product Profile - NovoSeven RT 192
Table 84: Summary of the Results of the Phase III Trial of NovoSeven 195
Table 85: NovoSeven RT SWOT Analysis, 2017 196
Table 86: Product Profile - Feiba NF 198
Table 87: Summary of the Results of the PROOF Trial of Feiba NF 200
Table 88: Feiba NF SWOT Analysis, 2017 200
Table 89: Unmet Needs and Opportunities in Hemophilia 203
Table 90: Product Profile - BAY 94-9027 221
Table 91: Efficacy of BAY 94-9027 in the PROTECT VIII study 223
Table 92: BAY 94-9027 SWOT Analysis, 2017 226
Table 93: Product Profile - N8-GP 227
Table 94: Efficacy of N8-GP in the Pathfinder 2 and 5Trials 229
Table 95: N8-GP SWOT Analysis, 2017 232
Table 96: Product Profile - Emicizumab 233
Table 97: Efficacy of Emicizumab in a Japanese Phase I/II Study 239
Table 98: Emicizumab SWOT Analysis, 2016 242
Table 99: Product Profile - Fitusiran 243
Table 100 : Efficacy of Fitusiran in the Part-D Phase I Study 246
Table 101: Fitusiran SWOT Analysis, 2017 249
Table 102: Product Profile - LR-769 252
Table 103: LR-769 SWOT Analysis, 2017 256
Table 104: Product Profile - Obizur 257
Table 105: Obizur SWOT Analysis, 2017 261
Table 106: Product Profile - CSL-689 262
Table 107: CSL-689 SWOT Analysis, 2017 266
Table 108: Product Profile - BAX-817 267
Table 109: BAX-817 SWOT Analysis, 2017 269
Table 110: TFPI inhibitors - Early-Stage Pipeline, 2017 270
Table 111: Hemophilia A and B Gene Therapies - Early-Stage Pipeline, 2017 272
Table 112: Hemophilia A and B Recombinant Replacement Therapies - Early-Stage Pipeline, 2017 278
Table 113: Drugs in Development for Hemophilia, 2016 280
Table 114: Key Companies in the Hemophilia A and B Recombinant Therapies Market, 2016-2026 286
Table 115: Bayers Hemophilia Portfolio Assessment, 2015 288
Table 116: Bayer SWOT Analysis, 2017 289
Table 117: Bioverativ/Sobis Hemophilia Portfolio Assessment, 2017 290
Table 118: Bioverativ SWOT Analysis, 2017 292
Table 119: CSL Behrings Hemophilia Recombinant Portfolio Assessment, 2017 293
Table 120: CSL Behring SWOT Analysis, 2017 294
Table 121: Novo Nordisks Hemophilia Recombinant Portfolio Assessment, 2017 295
Table 122: Novo Nordisk SWOT Analysis, 2017 297
Table 123: Pfizers Hemophilia Recombinant Portfolio Assessment, 2017 298
Table 124: Pfizer SWOT Analysis, 2017 299
Table 125: Roches Hemophilia Recombinant Portfolio Assessment, 2017 300
Table 126: Roche SWOT Analysis, 2017 301
Table 127: Shires Hemophilia Recombinant Portfolio Assessment, 2017 303
Table 128: Shire SWOT Analysis, 2017 304
Table 129: Global Hemophilia A and B Market - Drivers and Barriers, 2016-2026 308
Table 130: Key Events Impacting Sales for Hemophilia A and B in the US, 2016-2026 313
Table 131: US Hemophilia A and B Market - Drivers and Barriers, 2016-2026 313
Table 132: Key Events Impacting Sales for Hemophilia A and B Therapeutics in the 5EU, 2016-2026 319
Table 133: 5EU Hemophilia A and B Market - Drivers and Barriers, 2016-2026 319
Table 134: French Hemophilia A and B Market - Drivers and Barriers, 2016-2026 321
Table 135: German Hemophilia A and B Market - Drivers and Barriers, 2016-2026 321
Table 136: Italian Hemophilia A and B Market - Drivers and Barriers, 2016-2026 323
Table 137: Spanish Hemophilia A and B Market - Drivers and Barriers, 2016-2026 324
Table 138: UK Hemophilia A and B Market - Drivers and Barriers, 2016-2026 325
Table 139: Key Events Impacting Sales for Hemophilia A and B in Japan, 2016-2026 330
Table 140: Japanese Hemophilia A and B Market - Drivers and Barriers, 2016-2026 330
Table 141: Key Launch Dates 376
Table 142: Key Historical and Projected Patent Expiry Dates 377
Table 143: Average Body Weight Across the 7MM 379
Table 144: Average Cost of Therapy of Plasma-Derived FVIII Concentrates in the 7MM 380
Table 145: Average Cost of Therapy of Recombinate in the 7MM 381
Table 146: Average Cost of Therapy of Kogenate FS in the 7MM 381
Table 147: Average Cost of Therapy of Helixate FS in the 7MM 382
Table 148: Average Cost of Therapy of Advate in the 7MM 383
Table 149: Average Cost of Therapy of Kovaltry in the 7MM 384
Table 150: Average Cost of Therapy of Xyntha in the 7MM 384
Table 151: Average Cost of Therapy of NovoEight in the 7MM 385
Table 152: Average Cost of Therapy of Nuwiq in the 7MM 386
Table 153: Average Cost of Therapy of Eloctate in the 7MM 386
Table 154: Average Cost of Therapy of Adynovate in the 7MM 387
Table 155: Average Cost of Therapy of Afstyla in the 7MM 388
Table 156: Average Cost of Therapy of Plasma-derived FIX Concentrates in the 7MM 389
Table 157: Average Cost of Therapy of BeneFIX in the 7MM 389
Table 158: Average Cost of Therapy of Rixubis in the 7MM 390
Table 159: Average Cost of Therapy of IXinity in the 7MM 391
Table 160: Average Cost of Therapy of Alprolix in the 7MM 392
Table 161: Average Cost of Therapy of Idelvion in the 7MM 392
Table 162: Average Cost of Therapy of NovoSeven in the 7MM 393
Table 163: Average Cost of Therapy of Feiba in the 7MM 394
Table 164: Average Cost of Therapy of DDAVP in the 7MM 394
Table 165: Physicians Surveyed 399

1.2 List of Figures
Figure 1: Sales for Hemophilia A and B Therapeutics by Drug Class, 2016-2026 14
Figure 2: Competitive Assessment of Late-Stage Pipeline Agents, 2016-2026 15
Figure 3: Key Company Portfolio Positioning in Hemophilia A and B, 2016-2026 18
Figure 4: 7MM, Age-Standardized Diagnosed Prevalence of Hemophilia A (Cases per 100,000 Population), All Ages, 2016 32
Figure 5: 7MM, Age-Standardized Diagnosed Prevalence of Hemophilia B (Cases per 100,000 Population), All Ages, 2016 33
Figure 6: 7MM, Sources Used, Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B 34
Figure 7: 7MM, Sources Used, Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B by Severity 34
Figure 8: 7MM, Sources Used, Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B with Inhibitors 35
Figure 9: 7MM, Age-Specific Diagnosed Prevalent Cases of Hemophilia A, Both Sexes, All Ages, N, 2016 42
Figure 10: 7MM, Sex-Specific Diagnosed Prevalent Cases of Hemophilia A, Both Sexes, All Ages, N, 2016 43
Figure 11: 7MM, Diagnosed Prevalent Cases of Hemophilia A by Severity, Both Sexes, All Ages, N, 2016 43
Figure 12: 7MM, Diagnosed Prevalent Cases of Hemophilia A with Inhibitors, Both Sexes, All Ages, N, 2016 44
Figure 13: 7MM, Age-Specific Diagnosed Prevalent Cases of Hemophilia B, Both Sexes, All Ages, 2016 45
Figure 14: 7MM, Sex-Specific Diagnosed Prevalent Cases of Hemophilia B, Both Sexes, All Ages, N, 2016 46
Figure 15: 7MM, Diagnosed Prevalent Cases of Hemophilia B by Severity, Both Sexes, All Ages, N, 2016 47
Figure 16: 7MM, Diagnosed Prevalent Cases of Hemophilia B with Inhibitors, Both Sexes, All Ages, N, 2016 47
Figure 17: 7MM, Age-Specific Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B, Both Sexes, All Ages, N, 2016 49
Figure 18: 7MM, Sex-Specific Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B, All Ages, N, 2016 50
Figure 19: 7MM, Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B by Severity, Both Sexes, All Ages, N, 2016 51
Figure 20: 7MM, Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B with Inhibitors, Both Sexes, All Ages, N, 2016 51
Figure 21: Treatment Flowchart for Severe Patients with Inhibitors 69
Figure 22: Competitive Assessment of Late-Stage Pipeline Agents, 2016-2026 218
Figure 23: Overview of the Development Pipeline in Hemophilia, 2016-2026 219
Figure 24: Hemophilia A - Phase III Pipeline, 2017 220
Figure 25: BAY94-9027s Clinical Development in Hemophilia A 223
Figure 26: Clinical and Commercial Positioning of BAY94-9027 225
Figure 27: N8-GPs Clinical Development in Hemophilia A 228
Figure 28: Clinical and Commercial Positioning of N8-GP 230
Figure 29: Emicizumabs Clinical Development in Hemophilia A 238
Figure 30: Clinical and Commercial Positioning of Emicizumab 241
Figure 31: Clinical and Commercial Positioning of Fitusiran 248
Figure 32: Hemophilia A and B with Inhibitors - Phase III Pipeline, 2017 251
Figure 33: LR-769s Clinical Development in Patients with Inhibitors 254
Figure 34: Clinical and Commercial Positioning of LR-769 255
Figure 35: Obizurs Clinical Development in Patients with Inhibitors 259
Figure 36: Clinical and Commercial Positioning of Obizur 260
Figure 37: CSL-689s Clinical Development in Patients with Inhibitors 264
Figure 38: Clinical and Commercial Positioning of CSL-689 266
Figure 39: Clinical and Commercial Positioning of BAX-817 269
Figure 40: Company Portfolio Analysis in Hemophilia A and B, 2016-2026 284
Figure 41: Global Sales of Hemophilia Therapeutics by Company, 2016-2026 285
Figure 42: Global Sales for Hemophilia A and B by Drug Class, 2016-2026 306
Figure 43: Global Sales for Hemophilia A and B Therapeutics by Region, 2016-2026 307
Figure 44: Sales for Hemophilia A and B Therapeutics in the US by Drug Class, 2016-2026 312
Figure 45: Sales for Hemophilia A and B Therapeutics in the 5EU by Drug Class, 2016-2026 317
Figure 46: Sales for Hemophilia A and B Therapeutics in the 5EU by Country, 2016-2026 318
Figure 47: Sales for Hemophilia A and B Therapeutics in Japan by Drug Class, 2016-2026 329

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