Market Spotlight: Sickle Cell Anemia
Market Spotlight: Sickle Cell Anemia
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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, patent information, 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 therapeutics market, comprising key marketed and pipeline drugs, clinical trials, recent events and analyst opinion, upcoming and regulatory events, patent information, 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 approximated 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 majority of industry-sponsored drugs in active clinical development for sickle cell anemia are spread evenly across Phase I and Phase II, with two drugs in Phase III.
- Therapies in development for sickle cell anemia focus on a wide variety of targets. All the pipeline drugs are administered via the intravenous or oral routes.
- High-impact upcoming events for drugs in the sickle cell anemia space comprise an expected CHMP opinion for Adakveo, topline Phase II trial results for olinciguat, topline Phase IIb trial results for IMR-687, and topline Phase III trial results for Brilinta.
- The overall likelihood of approval of a Phase I hematology asset is 27.1%, and the average probability a drug advances from Phase III is 75.2%. Drugs, on average, take 9.3 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 80% of trials in Phase I–II, and only 20% 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.
- Clinical trial activity in the sickle cell anemia space is dominated by completed trials. Global Blood Therapeutics has the highest number of completed clinical trials for sickle cell anemia, with 10 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 IMR-687 for Sickle Cell Anemia (February 14, 2020)
22 CTX001 for Sickle Cell Anemia (November 19, 2019)
23 Rivipansel for Sickle Cell Anemia (August 2, 2019)
25 Olinciguat for Sickle Cell Anemia (April 1, 2019)
26 ARU-1801 for Sickle Cell Anemia (December 3, 2018)
28 Oxbryta for Sickle Cell Anemia (December 3, 2018)
31 Zynteglo for Sickle Cell Anemia (December 3, 2018)
32 BCL11a shRNA(miR) for Sickle Cell Anemia (November 1, 2018)
34 KEY UPCOMING EVENTS
35 KEY REGULATORY EVENTS
35 Global Blood Therapeutics’ Oxbryta Approved Broadly For Sickle Cell Disease
35 FDA Approval For Novartis’s Sickle Cell Treatment Adakveo
35 Emmaus: ‘We Might Stand A Better Chance In Europe Without The EMA’
35 Global Blood Therapeutics’ Voxelotor On Track For 2020 Sickle Cell Shakeup
37 PROBABILITY OF SUCCESS
38 LICENSING AND ASSET ACQUISITION DEALS
38 Pfizer Ends Pact With GlycoMimetics After SCD Failure
38 Global Blood Partners With Syros For Sickle Cell, Beta Thalassemia Therapies
38 Vertex Partners With Molecular Templates To Enhance Stem Cell Candidates
39 CLINICAL TRIAL LANDSCAPE
40 Sponsors by status
41 Sponsors by phase
42 Recent events
43 BIBLIOGRAPHY
43 Prescription information
45 APPENDIX
LIST OF FIGURES
14 Figure 1: Overview of pipeline drugs for sickle cell anemia in the US
15 Figure 2: Pipeline drugs for sickle cell anemia, by company
15 Figure 3: Pipeline drugs for sickle cell anemia, by drug type
16 Figure 4: Pipeline drugs for sickle cell anemia, by classification
22 Figure 5: IMR-687 for Sickle Cell Anemia (February 14, 2020): Phase IIa – IMR-SCD-102
25 Figure 6: Rivipansel for Sickle Cell Anemia (August 2, 2019): Phase III – RESET
31 Figure 7: Oxbryta for Sickle Cell Anemia (December 3, 2018): Phase III – HOPE
34 Figure 8: Key upcoming events in sickle cell anemia
37 Figure 9: Probability of success in the sickle cell anemia pipeline
39 Figure 10: Clinical trials in sickle cell anemia
39 Figure 11: Top 10 drugs for clinical trials in sickle cell anemia
40 Figure 12: Top 10 companies for clinical trials in sickle cell anemia
40 Figure 13: Trial locations in sickle cell anemia
41 Figure 14: Sickle cell anemia trials status
42 Figure 15: Sickle cell anemia trial sponsors, by phase
LIST OF TABLES
13 Table 1: Marketed drugs for sickle cell anemia
17 Table 2: Pipeline drugs for sickle cell anemia in the US
20 Table 3: IMR-687 for Sickle Cell Anemia (February 14, 2020)
22 Table 4: CTX001 for Sickle Cell Anemia (November 19, 2019)
24 Table 5: Rivipansel for Sickle Cell Anemia (August 2, 2019)
26 Table 6: Olinciguat for Sickle Cell Anemia (April 1, 2019)
27 Table 7: ARU-1801 for Sickle Cell Anemia (December 3, 2018)
28 Table 8: Oxbryta for Sickle Cell Anemia (December 3, 2018)
31 Table 9: Zynteglo for Sickle Cell Anemia (December 3, 2018)
33 Table 10: BCL11a shRNA(miR) for Sickle Cell Anemia (November 1, 2018)
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