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, probability of success, a 10-year disease prevalence forecast, and licensing and acquisition deals.
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, as well as presenting drug-specific revenue forecasts.
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 expected topline results from the Hibiscus study for etavopivat, topline Phase I/II trial results for GPH101, topline Phase II trial results for MGTA-145 and etavopivat, and topline Phase IIa trial results for VIT-2763.
- The overall likelihood of approval of a Phase I hematology asset is 24.5%, and the average probability a drug advances from Phase III is 75.2%. Drugs, on average, take 9.9 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 75% of trials in Phase I–II, and only 25% 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 16 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
21 RECENT EVENTS AND ANALYST OPINION
21 Zynteglo for Sickle Cell Anemia (December 20, 2021)
22 Multiple Drugs for Sickle Cell Anemia (December 12, 2021)
24 SAR445136 for Sickle Cell Anemia (December 12, 2021)
26 Zynteglo for Sickle Cell Anemia (June 7, 2021)
27 Zynteglo for Sickle Cell Anemia (April 20, 2021)
28 Zynteglo for Sickle Cell Anemia (March 10, 2021)
30 Zynteglo for Sickle Cell Anemia (February 16, 2021)
31 IMR-687 for Sickle Cell Anemia (January 6, 2021)
33 Multiple Drugs for Sickle Cell Anemia (December 7, 2020)
36 KEY UPCOMING EVENTS
37 KEY REGULATORY EVENTS
37 PRIME: How The EU Scheme Is Delivering On Promise Of Faster Drug Approvals
37 Accelerated Approval Makes Big Splash In 2021 Novel Agents, Despite Crackdown On ‘Dangling’ Cancer Claims
37 FDA Puts Partial Clinical Hold On Bluebird Bio’s Lovotibeglogene Autotemcel
38 Novel Sickle Cell Treatment Among 13 New Drugs On Track For EU Approval
38 Biotech Drives Base Editing Into Clinic As Big Pharma Looks On
38 New Sickle Cell Treatment Wins English Funding Approval
39 Safety Thumbs Up For Zynteglo Prompts Bluebird To Lift Marketing Suspension
39 UK MHRA Awards Bluebird’s LentiGlobin An Innovation Passport
40 PROBABILITY OF SUCCESS
41 LICENSING AND ASSET ACQUISITION DEALS
41 Sanofi Exit From Sickle Cell Gene Therapy Leaves Sangamo With Difficult Decision
41 Vertex Links Up Again With Arbor For Gene Editing
42 REVENUE OPPORTUNITY
43 CLINICAL TRIAL LANDSCAPE
44 Sponsors by status
45 Sponsors by phase
46 Recent events
48 BIBLIOGRAPHY
49 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
24 Figure 5: Etavopivat for Sickle Cell Anemia (December 12, 2021): Phase I – SAD/MAD
24 Figure 6: Pyrukynd for Sickle Cell Anemia (December 11, 2021): Phase I – NHLBI
33 Figure 7: IMR-687 for Sickle Cell Anemia (January 6, 2021): Phase IIa – IMR-SCD-102
36 Figure 8: Key upcoming events in sickle cell anemia
40 Figure 9: Probability of success in the hematology pipeline
43 Figure 10: Clinical trials in sickle cell anemia
43 Figure 11: Top 10 drugs for clinical trials in sickle cell anemia
44 Figure 12: Top 10 companies for clinical trials in sickle cell anemia
44 Figure 13: Trial locations in sickle cell anemia
45 Figure 14: Sickle cell anemia trials status
46 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: Zynteglo for Sickle Cell Anemia (December 20, 2021)
22 Table 4: Multiple Drugs for Sickle Cell Anemia (December 12, 2021)
25 Table 5: SAR445136 for Sickle Cell Anemia (December 12, 2021)
26 Table 6: Zynteglo for Sickle Cell Anemia (June 7, 2021)
27 Table 7: Zynteglo for Sickle Cell Anemia (April 20, 2021)
29 Table 8: Zynteglo for Sickle Cell Anemia (March 10, 2021)
30 Table 9: Zynteglo for Sickle Cell Anemia (February 16, 2021)
32 Table 10: IMR-687 for Sickle Cell Anemia (January 6, 2021)
33 Table 11: Multiple Drugs for Sickle Cell Anemia (December 7, 2020)
42 Table 12: Historical global sales, by drug ($m), 2017–21
42 Table 13: Forecasted global sales, by drug ($m), 2022–26
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