The therapeutic strategies for hemophilia were revolutionized after the introduction of replacement factors during the late 1990s and early 2000s, which were followed by EHLs over the past decade. Currently, the hemophilia market is undergoing a third revolution, with an anticipated shift towards alternative coagulation promoters and gene therapy.
CONTENTS
8 OVERVIEW
8 Latest key takeaways
10 DISEASE BACKGROUND
10 Definition
10 Patient segmentation
10 Etiology
11 Von Willebrand disease
11 Hemophilia symptoms
12 VWD symptoms
12 Hemophilia and VWD complications
13 Diagnosis
13 Treatment options
14 TREATMENT
19 EPIDEMIOLOGY
19 Prevalence methodology
27 MARKETED DRUGS
27 Hemophilia A
33 Hemophilia B
36 Hemophilia A and B
38 Von Willebrand disease
40 PIPELINE DRUGS
41 Hemophilia A
44 Hemophilia B
46 Hemophilia A and B
48 Von Willebrand disease
51 KEY REGULATORY EVENTS
51 Hemophilia B Gene Therapy Among Five Drugs On Track For EU-Wide Approval
51 ICER Hemophilia Gene Therapy Reviews Show Clear ‘Net Health Benefit’ But Caution On Patient, Payer Fronts Given Upfront Costs
52 CSL’s Hemgenix Gene Therapy Does Not Need A REMS, FDA Tells National Hemophilia Foundation
52 UniQure/CSL Set To Launch First Hemophilia B Gene Therapy Next Year At Record Price
53 BioMarin Prepares Investors For A Possible US Hemophilia Gene Therapy Delay
53 Round Two: BioMarin Resubmits Hemophilia A Gene Therapy Roctavian In US
53 Novo Eyes Global-First Japan Approval For Concizumab
54 Priority Review Sets Up Sanofi’s ‘Best In Disease’ Hemophilia Drug For 2023 Launch
54 €1.5m Price Tag Could Spell EU Success For BioMarin’s Hemophilia Gene Therapy
55 Japan-Specific Approval For Hemlibra In Acquired Hemophilia A
55 Hemophilia Gene Therapies Should Have REMS For Approval, National Hemophilia Foundation Says
55 BioMarin Rolls Up Sleeves To Prepare For Roctavian’s EU Debut
56 Takeda Expands Hemophilia Play In India But Hemlibra Has Made Inroads
56 CSL/uniQure Hope To Be First In Hemophilia B Gene Therapy
56 As BioMarin Stumbles, Sanofi Gets Breakthrough Status For Hemlibra Challenger
57 BioMarin Delays Resubmission, But Roctavian’s Rocky Road Winding Down
57 Cevenfacta Among Nine Drugs To Win EU Thumbs Up
58 PROBABILITY OF SUCCESS
59 LICENSING AND ASSET ACQUISITION DEALS
59 2seventy Licenses LNP Tech From Genevant, Builds On Novo Nordisk Pact
60 CLINICAL TRIAL LANDSCAPE
61 Sponsors by status
62 Sponsors by phase
63 Recent events
69 DRUG ASSESSMENT MODEL
69 Replacement factor concentrates
70 Bypassing agents
71 Alternative coagulation promoters
71 Tissue factor pathway inhibitors
72 Small interference RNA therapy
73 Gene therapy for hemophilia A
76 Gene therapy for hemophilia B
81 MARKET DYNAMICS
82 FUTURE TRENDS
82 The hemophilia market will continue to expand over the forecast period
82 Hemlibra will continue to capture market share from replacement factors in the hemophilia A space
82 Pipeline alternative coagulation promoters will capture a limited amount of market share in the hemophilia B space
83 Uptake of gene therapy will be limited by high upfront costs and uncertainty over long-term effectiveness
84 CONSENSUS FORECASTS
89 RECENT EVENTS AND ANALYST OPINION
89 Fidanacogene Elaparvovec for Hemophilia B (December 29, 2022)
90 Fitusiran for Hemophilia A and B (July 10, 2022)
92 BIVV 001 for Hemophilia A (March 9, 2022)
93 Fitusiran for Hemophilia A and B (December 14, 2021)
95 Fitusiran for Hemophilia A and B (December 12, 2021)
96 Etranacogene Dezaparvovec for Hemophilia B (December 9, 2021)
98 SerpinPC for Hemophilia A and B (September 9, 2021)
100 Roctavian for Hemophilia A (July 19, 2021)
102 SIG-001 for Hemophilia A (July 9, 2021)
105 KEY UPCOMING EVENTS
106 KEY OPINION LEADER INSIGHTS
107 UNMET NEEDS
109 BIBLIOGRAPHY
112 APPENDIX
LIST OF FIGURES
11 Figure 1: Hemophilia etiology
22 Figure 2: Trends in diagnosed prevalent cases of hemophilia A, 2018–27
24 Figure 3: Trends in diagnosed prevalent cases of hemophilia B, 2018–27
26 Figure 4: Trends in diagnosed cases of Von Willebrand disease, 2018–27
40 Figure 5: Overview of pipeline drugs for hemophilia in the US
40 Figure 6: Pipeline drugs for hemophilia, by company
41 Figure 7: Pipeline drugs for hemophilia, by drug type
41 Figure 8: Pipeline drugs for hemophilia, by classification
58 Figure 9: Probability of success in the hemophilia pipeline
60 Figure 10: Clinical trials in hemophilia
60 Figure 11: Top 10 drugs for clinical trials in hemophilia
61 Figure 12: Top 10 companies for clinical trials in hemophilia
61 Figure 13: Trial locations in hemophilia
62 Figure 14: Hemophilia trials status
63 Figure 15: Hemophilia trials sponsors, by phase
69 Figure 16: Datamonitor Healthcare’s drug assessment summary for hemophilia
81 Figure 17: Market dynamics in hemophilia
82 Figure 18: Future trends in hemophilia
92 Figure 19: Fitusiran for Hemophilia A and B (July 10, 2022): Phase III – ATLAS-PPX
95 Figure 20: Fitusiran for Hemophilia A and B (December 14, 2021): Phase III – ATLAS-A/B
96 Figure 21: Fitusiran for Hemophilia A and B (December 12, 2021): Phase III – ATLAS-INH
98 Figure 22: Etranacogene Dezaparvovec for Hemophilia B (December 9, 2021): Phase III – HOPE-B
100 Figure 23: SerpinPC for Hemophilia A and B (September 9, 2021): Phase I/IIa – AP-0101
102 Figure 24: Roctavian for Hemophilia A (July 19, 2021): Phase III – GENEr8-1 (6E13 vg/kg)
105 Figure 25: Key upcoming events in hemophilia
108 Figure 26: Unmet needs in hemophilia, physician survey results
LIST OF TABLES
15 Table 1: Drug classes for hemophilia A, B, and VWD
18 Table 2: Medical and Scientific Advisory Council (MASAC) recommendations for hemophilia A, B, and VWD
19 Table 3: Gender distributions of diagnosed prevalent cases of hemophilia A, hemophilia B, and Von Willebrand disease
20 Table 4: Diagnosed prevalent cases of hemophilia A, 2018–27
23 Table 5: Diagnosed prevalent cases of hemophilia B, 2018–27
25 Table 6: Diagnosed prevalent cases of Von Willebrand disease, 2018–27
28 Table 7: Marketed drugs for hemophilia A
34 Table 8: Marketed drugs for hemophilia B
37 Table 9: Marketed drugs for hemophilia A and B
39 Table 10: Marketed drugs for Von Willebrand disease
42 Table 11: Pipeline drugs for hemophilia A in the US
45 Table 12: Pipeline drugs for hemophilia B in the US
47 Table 13: Pipeline drugs for both hemophilia A and B in the US
49 Table 14: Pipeline drugs for Von Willebrand disease in the US
78 Table 15: Summary of safety and efficacy results for gene therapies for hemophilia A and B
85 Table 16: Historical global sales, by drug ($m), 2017–21
87 Table 17: Forecasted global sales, by drug ($m), 2023–27
89 Table 18: Fidanacogene Elaparvovec for Hemophilia B (December 29, 2022)
90 Table 19: Fitusiran for Hemophilia A and B (July 10, 2022)
93 Table 20: BIVV 001 for Hemophilia A (March 9, 2022)
94 Table 21: Fitusiran for Hemophilia A and B (December 14, 2021)
95 Table 22: Fitusiran for Hemophilia A and B (December 12, 2021)
97 Table 23: Etranacogene Dezaparvovec for Hemophilia B (December 9, 2021)
99 Table 24: SerpinPC for Hemophilia A and B (September 9, 2021)
101 Table 25: Roctavian for Hemophilia A (July 19, 2021)
103 Table 26: SIG-001 for Hemophilia A (July 9, 2021)
© Pharma Intelligence UK Ltd. This document is a licensed product and is not to be reproduced or redistributed
Do you have a subscription to Datamonitor Healthcare, Biomedtracker or Meddevicetracker? You may already have access to these reports, contact your account manager or email pharma@informabi.com for further help or assistance.
Sign up to the Pharma Intelligence Report Store Newsletter to get the latest blogs, news, reports and discounts!