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 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
- Datamonitor Healthcare estimates that in 2017, there were 3.2 million prevalent cases of sickle cell anemia worldwide, and forecasts that number to increase to 3.5 million prevalent cases by 2026.
- Africa is estimated to have had 2.7 million prevalent cases of sickle cell anemia in 2017, accounting for 85% of worldwide cases. The approved drugs in the sickle cell anemia space predominantly target fetal hemoglobin, with the exception of Endari, which targets nicotinamide adenine dinucleotide. These drugs are administered via the oral route.
- The largest proportion of industry-sponsored drugs in active clinical development for sickle cell anemia are in Phase I, with two drugs in the NDA/BLA phase.
- 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 estimated PDUFA date for voxelotor, an expected PDUFA date as well as a CHMP opinion for crizanlizumab, topline Phase I/II trial results for CTX001, and topline Phase II trial results for olinciguat.
- The overall likelihood of approval of a Phase I hematology asset is 25.5%, and the average probability a drug advances from Phase III is 73%. Drugs, on average, take 8.9 years from Phase I to approval in the overall hematology space.
- There have been 16 licensing and asset acquisition deals involving sickle cell anemia drugs during 2014–19. The $3,520m coexclusive license agreement made in 2017 between Vertex Pharmaceuticals and CRISPR Therapeutics for the development and commercialization of potential hemoglobinopathy treatments, including treatments for sickle cell disease, was the largest deal.
- 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 EU 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 and Pfizer have the highest number of completed clinical trials for sickle cell anemia, with nine trials each.
- 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
13 MARKETED DRUGS
15 PIPELINE DRUGS
22 RECENT EVENTS AND ANALYST OPINION
22 Rivipansel for Sickle Cell Anemia (August 2, 2019)
23 Olinciguat for Sickle Cell Anemia (April 1, 2019)
24 RVT-1801 for Sickle Cell Anemia (December 3, 2018)
26 Voxelotor for Sickle Cell Anemia (December 3, 2018)
29 Zynteglo for Sickle Cell Anemia (December 3, 2018)
31 BCL11a shRNA(miR) for Sickle Cell Anemia (November 1, 2018)
34 KEY UPCOMING EVENTS
35 KEY REGULATORY EVENTS
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
35 Global Blood Therapeutics Leaves ASH With Momentum Behind Voxelotor In Sickle Cell Disease
36 PROBABILITY OF SUCCESS
37 LICENSING AND ASSET ACQUISITION DEALS
37 Emmaus Grants Taiba Rights To Endari In MENA Nations
37 How Brazilian Patients Might Access Sickle Cell Disease Drug Faster
39 PARENT PATENTS
40 CLINICAL TRIAL LANDSCAPE
41 Sponsors by status
42 Sponsors by phase
43 Recent events
44 BIBLIOGRAPHY
44 Prescription information
45 APPENDIX
LIST OF FIGURES
15 Figure 1: Overview of pipeline drugs for sickle cell anemia in the US
16 Figure 2: Pipeline drugs for sickle cell anemia, by company
16 Figure 3: Pipeline drugs for sickle cell anemia, by drug type
17 Figure 4: Pipeline drugs for sickle cell anemia, by classification
23 Figure 5: Rivipansel for Sickle Cell Anemia (August 2, 2019): Phase III – RESET
29 Figure 6: Voxelotor for Sickle Cell Anemia (December 3, 2018): Phase III – HOPE
34 Figure 7: Key upcoming events in sickle cell anemia
36 Figure 8: Probability of success in the sickle cell anemia pipeline
37 Figure 9: Licensing and asset acquisition deals in sickle cell anemia, 2014–19
39 Figure 10: Parent patents in sickle cell anemia
40 Figure 11: Clinical trials in sickle cell anemia
40 Figure 12: Top 10 drugs for clinical trials in sickle cell anemia
41 Figure 13: Top 10 companies for clinical trials in sickle cell anemia
41 Figure 14: Trial locations in sickle cell anemia
42 Figure 15: Sickle cell anemia trials status
43 Figure 16: Sickle cell anemia trial sponsors, by phase
LIST OF TABLES
11 Table 1: Prevalent cases of sickle cell anemia, 2017–26
14 Table 2: Marketed drugs for sickle cell anemia
18 Table 3: Pipeline drugs for sickle cell anemia in the US
22 Table 4: Rivipansel for Sickle Cell Anemia (August 2, 2019)
24 Table 5: Olinciguat for Sickle Cell Anemia (April 1, 2019)
25 Table 6: RVT-1801 for Sickle Cell Anemia (December 3, 2018)
27 Table 7: Voxelotor for Sickle Cell Anemia (December 3, 2018)
30 Table 8: Zynteglo for Sickle Cell Anemia (December 3, 2018)
32 Table 9: BCL11a shRNA(miR) for Sickle Cell Anemia (November 1, 2018)
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