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, as well as presenting drug-specific revenue forecasts.
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 2021. 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 II, with one drug in the NDA/BLA Phase.
- 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, topline Phase IIa trial results for VIT-2763, topline Phase II/III trial results for GBT601, estimated regulatory filings in the US and EU for crovalimab, and an estimated CHMP opinion and European approval decision for exa-cel, as well as an expected US FDA approval for Pyrukynd.
- The overall likelihood of approval of a Phase I hematology asset is 23.8%, and the average probability a drug advances from Phase III is 74.2%. 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 74% of trials in Phase I–II, and only 26% 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.
- Pfizer has the highest number of ongoing clinical trials for sickle cell anemia, with 17 trials.
- Pfizer 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 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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