Market Spotlight: Sickle Cell Anemia
Market Spotlight: Sickle Cell Anemia
$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.
Author: Informa Pharma Intelligence
Publisher: Datamonitor Healthcare
- Description
- Contents
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.
Key Takeaways
- Using an estimated global prevalence of 0.04%, Datamonitor Healthcare approximates that there were 3.3 million prevalent cases of sickle cell anemia worldwide in 2019. Africa is estimated to account for 85% of worldwide cases.
- The approved drugs in the sickle cell anemia space target selectins, fetal hemoglobin, nicotinamide adenine dinucleotide, and hemoglobin. These drugs are administered via the oral route, except for Adakveo, which is available as an intravenous formulation.
- The largest proportion of industry-sponsored drugs in active clinical development for sickle cell anemia are in Phase I. Therapies in development for sickle cell anemia focus on a wide variety of targets. The majority of the pipeline drugs are administered via the intravenous route, with the remainder being oral and subcutaneous formulations.
- High-impact upcoming events for drugs in the sickle cell anemia space comprise topline Phase I/II trial results for SAR445136, topline Phase IIb trial results for IMR-687, and an expected CHMP opinion for Oxbryta.
- The overall likelihood of approval of a Phase I hematology asset is 24.8%, and the average probability a drug advances from Phase III is 75.5%. Drugs, on average, take 9.7 years from Phase I to approval in the overall hematology space.
- The distribution of clinical trials across Phase I–IV indicates that the majority of trials for sickle cell anemia have been in the early and mid-phases of development, with 77% of trials in Phase I–II, and only 23% in Phase III–IV.
- The US has a substantial lead in the number of sickle cell anemia clinical trials globally. The UK leads the major European markets, while Lebanon has the top spot in Asia.
- Global Blood Therapeutics has the highest number of ongoing clinical trials for sickle cell anemia, with 12 trials. Global Blood Therapeutics leads industry sponsors with the highest overall number of clinical trials for sickle cell anemia, followed by Novartis
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
20 RECENT EVENTS AND ANALYST OPINION
20 Zynteglo for Sickle Cell Anemia (April 20, 2021)
21 Zynteglo for Sickle Cell Anemia (March 10, 2021)
23 Zynteglo for Sickle Cell Anemia (February 16, 2021)
24 IMR-687 for Sickle Cell Anemia (January 6, 2021)
26 Multiple Drugs for Sickle Cell Anemia (December 7, 2020)
28 IMR-687 for Sickle Cell Anemia (June 12, 2020)
30 IMR-687 for Sickle Cell Anemia (February 14, 2020)
33 KEY UPCOMING EVENTS
34 KEY REGULATORY EVENTS
34 Bluebird Sickle Cell Gene Therapy Hangs In Balance
34 European Medicines Agency Grants PRIME Designation To ARU-1801
34 New Therapeutic Targets & A Rich Year For Approvals: EMA’s PRIME In 2020
35 PROBABILITY OF SUCCESS
36 LICENSING AND ASSET ACQUISITION DEALS
36 Vertex’s Price Tag For Taking More Control Of CRISPR-Partnered Candidate: At Least $900m
36 GBT Bucks Gene Therapy Trend With Sanofi Sickle Cell Deal
37 REVENUE OPPORTUNITY
38 CLINICAL TRIAL LANDSCAPE
39 Sponsors by status
40 Sponsors by phase
41 Recent events
43 BIBLIOGRAPHY
44 APPENDIX
LIST OF FIGURES
14 Figure 1: Overview of pipeline drugs for sickle cell anemia in the US
14 Figure 2: Pipeline drugs for sickle cell anemia, by company
15 Figure 3: Pipeline drugs for sickle cell anemia, by drug type
15 Figure 4: Pipeline drugs for sickle cell anemia, by classification
26 Figure 5: IMR-687 for Sickle Cell Anemia (January 6, 2021): Phase IIa – IMR-SCD-102
30 Figure 6: IMR-687 for Sickle Cell Anemia (June 12, 2020): Phase IIa – IMR-SCD-102
32 Figure 7: IMR-687 for Sickle Cell Anemia (February 14, 2020): Phase IIa – IMR-SCD-102
33 Figure 8: Key upcoming events in sickle cell anemia
35 Figure 9: Probability of success in the hematology pipeline
38 Figure 10: Clinical trials in sickle cell anemia
38 Figure 11: Top 10 drugs for clinical trials in sickle cell anemia
39 Figure 12: Top 10 companies for clinical trials in sickle cell anemia
39 Figure 13: Trial locations in sickle cell anemia
40 Figure 14: Sickle cell anemia trials status
41 Figure 15: Sickle cell anemia trial sponsors, by phase
LIST OF TABLES
13 Table 1: Marketed drugs for sickle cell anemia
16 Table 2: Pipeline drugs for sickle cell anemia in the US
20 Table 3: Zynteglo for Sickle Cell Anemia (April 20, 2021)
22 Table 4: Zynteglo for Sickle Cell Anemia (March 10, 2021)
23 Table 5: Zynteglo for Sickle Cell Anemia (February 16, 2021)
25 Table 6: IMR-687 for Sickle Cell Anemia (January 6, 2021)
26 Table 7: Multiple Drugs for Sickle Cell Anemia (December 7, 2020)
29 Table 8: IMR-687 for Sickle Cell Anemia (June 12, 2020)
30 Table 9: IMR-687 for Sickle Cell Anemia (February 14, 2020)
37 Table 10: Historical global sales, by drug ($m), 2016–20
37 Table 11: Forecasted global sales, by drug ($m), 2021–25
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