$1,318.00
This Market Spotlight report covers the Sickle Cell Anemia market, comprising key marketed and pipeline drugs, clinical trials, recent events and analyst opinion, upcoming and regulatory events, probability of success, a 10-year disease prevalence forecast, and licensing and acquisition deals, as well as presenting drug-specific revenue forecasts.
This Market Spotlight report covers the Sickle Cell Anemia market, comprising key marketed and pipeline drugs, clinical trials, recent events and analyst opinion, upcoming and regulatory events, probability of success, a 10-year disease prevalence forecast, and licensing and acquisition deals, as well as presenting drug-specific revenue forecasts.
Key Takeaways
CONTENTS
6 OVERVIEW
7 KEY TAKEAWAYS
8 DISEASE BACKGROUND
9 TREATMENT
9 Bone marrow transplantation
9 Hydroxyurea
9 Blood transfusions
10 Prophylactic therapy
10 Pain management
11 EPIDEMIOLOGY
12 MARKETED DRUGS
14 PIPELINE DRUGS
21 RECENT EVENTS AND ANALYST OPINION
21 EDIT-301 for Sickle Cell Anemia (December 6, 2022)
23 Zynteglo for Sickle Cell Anemia (December 20, 2021)
24 Multiple Drugs for Sickle Cell Anemia (December 12, 2021)
27 SAR445136 for Sickle Cell Anemia (December 12, 2021)
29 KEY UPCOMING EVENTS
30 KEY REGULATORY EVENTS
30 Scottish HTA Requests More Data For Orphan Drugs
30 PRIME: How The EU Scheme Is Delivering On Promise Of Faster Drug Approvals
31 PROBABILITY OF SUCCESS
32 LICENSING AND ASSET ACQUISITION DEALS
32 Novo Nordisk’s Forma Buy Builds On Foothold In Rare Blood Disorders
32 Pfizer’s Buying Spree Continues With GBT, Gaining A Sickle Cell Disease Franchise
32 Novartis Dives Into In Vivo Gene-Editing With Precision Biosciences Deal
33 Sanofi Exit From Sickle Cell Gene Therapy Leaves Sangamo With Difficult Decision
34 REVENUE OPPORTUNITY
35 CLINICAL TRIAL LANDSCAPE
36 Sponsors by status
37 Sponsors by phase
38 Recent events
41 BIBLIOGRAPHY
42 APPENDIX
LIST OF FIGURES
14 Figure 1: Overview of pipeline drugs for sickle cell anemia in the US
14 Figure 2: Pipeline drugs for sickle cell anemia, by company
15 Figure 3: Pipeline drugs for sickle cell anemia, by drug type
15 Figure 4: Pipeline drugs for sickle cell anemia, by classification
23 Figure 5: EDIT-301 for Sickle Cell Anemia (December 6, 2022): Phase I/II – RUBY
26 Figure 6: Etavopivat for Sickle Cell Anemia (December 12, 2021): Phase I – SAD/MAD
27 Figure 7: Pyrukynd for Sickle Cell Anemia (December 11, 2021): Phase I – NHLBI
29 Figure 8: Key upcoming events in sickle cell anemia
31 Figure 9: Probability of success in the hematology pipeline
35 Figure 10: Clinical trials in sickle cell anemia
35 Figure 11: Top 10 drugs for clinical trials in sickle cell anemia
36 Figure 12: Top 10 companies for clinical trials in sickle cell anemia
36 Figure 13: Trial locations in sickle cell anemia
37 Figure 14: Sickle cell anemia trials status
38 Figure 15: Sickle cell anemia trial sponsors, by phase
LIST OF TABLES
13 Table 1: Marketed drugs for sickle cell anemia
16 Table 2: Pipeline drugs for sickle cell anemia in the US
21 Table 3: EDIT-301 for Sickle Cell Anemia (December 6, 2022)
23 Table 4: Zynteglo for Sickle Cell Anemia (December 20, 2021)
24 Table 5: Multiple Drugs for Sickle Cell Anemia (December 12, 2021)
27 Table 6: SAR445136 for Sickle Cell Anemia (December 12, 2021)
34 Table 7: Historical global sales, by drug ($m), 2017–21
34 Table 8: Forecasted global sales, by drug ($m), 2023–27
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