CONTENTS

5 OVERVIEW
6 KEY TAKEAWAYS
7 DISEASE BACKGROUND
8 TREATMENT
8 Bone marrow transplantation
8 Hydroxyurea
8 Blood transfusions
9 Prophylactic therapy
9 Pain management
10 EPIDEMIOLOGY
12 MARKETED DRUGS
12 Approvals by country
13 PIPELINE DRUGS
20 KEY UPCOMING EVENTS
21 KEY REGULATORY EVENTS
21 US FDA approves sickle cell therapy, Endari
22 LICENSING AND ASSET ACQUISITION DEALS
22 Generex signs LOI to acquire 51% of Emmaus Life Sciences
22 Novartis buys Selexys as competitors stumble in sickle cell
23 PARENT PATENTS
24 CLINICAL TRIAL LANDSCAPE
25 Sponsors by status
26 Sponsors by phase
27 Phase III trial of Mast Therapeutics product in sickle cell disease failed
28 BIBLIOGRAPHY
28 Prescription Information:
29 APPENDIX

LIST OF FIGURES
13 Figure 1: 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 drug classification
20 Figure 5: Key upcoming events in sickle cell anemia
22 Figure 6: Licensing and asset acquisition deals in sickle cell anemia
23 Figure 7: Parent patents in sickle cell anemia
24 Figure 8: Clinical trials in sickle cell anemia
24 Figure 9: Top 10 drugs for clinical trials in sickle cell anemia
25 Figure 10: Top 10 companies for clinical trials in sickle cell anemia
25 Figure 11: Trial locations in sickle cell anemia
26 Figure 12: Sickle cell anemia trials status
27 Figure 13: Sickle cell anemia trial sponsors, by phase

LIST OF TABLES
10 Table 1: Prevalent cases of sickle cell anemia, 2016-25
12 Table 2: Marketed drugs for sickle cell anemia
12 Table 3: Approvals by country for sickle cell anemia
17 Table 4: Pipeline drugs for sickle cell anemia in the US