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, 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 II. Therapies in development for sickle cell anemia focus on a wide variety of targets. Pipeline drugs are administered via the intravenous or oral routes, with two products also being tested in subcutaneous formulations.
- High-impact upcoming events in the sickle cell anemia space comprise an expected CHMP opinion for Endari, a pre-NDA meeting with the FDA for Voxelotor, topline Phase III trial results for Rivipansel, and topline Phase II trial results for Olinciguat.
- The overall likelihood of approval of a Phase I hematology asset is 29.4%, and the average probability a drug advances from Phase III is 75.9%. Drugs, on average, take 8.9 years from Phase I to approval in the overall hematology space.
- There have been 11 licensing and asset acquisition deals involving sickle cell anemia drugs during 2014–19. The $2,520m strategic research collaboration deal signed in 2015 between Vertex Pharmaceuticals and CRISPR Therapeutics for the discovery and development of drugs based on CRISPR-Cas9 technology 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 79% of trials in Phase I–II, and only 21% 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 eight trials each.
- Global Blood Therapeutics leads industry sponsors with the highest 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
21 RECENT EVENTS AND ANALYST OPINION
21 LentiGlobin for Sickle Cell Anemia (December 3, 2018)
22 RVT-1801 for Sickle Cell Anemia (December 3, 2018)
24 Voxelotor for Sickle Cell Anemia (December 3, 2018)
27 BCL11a shRNA(miR) for Sickle Cell Anemia (November 1, 2018)
29 Voxelotor for Sickle Cell Anemia (June 27, 2018)
30 Altemia for Sickle Cell Anemia (April 30, 2018)
32 LentiGlobin for Sickle Cell Anemia (December 9, 2017)
34 KEY UPCOMING EVENTS
35 KEY REGULATORY EVENTS
35 Global Blood Therapeutics Leaves ASH With Momentum Behind Voxelotor In Sickle Cell Disease
35 Modus CEO Says Rare Disease Priority Review Voucher To Be Key Catalyst
36 PROBABILITY OF SUCCESS
37 LICENSING AND ASSET ACQUISITION DEALS
37 How Brazilian Patients Might Access Sickle Cell Disease Drug Faster
37 GBT Picks Up Inclacumab From Roche’s Bargain Bin
38 EpiDestiny Inc. granted Novo Nordisk AS exclusive global rights to develop and sell its sickle cell disease candidate EPI01 (decitabine/tetrahydouridine)
39 PARENT PATENTS
40 CLINICAL TRIAL LANDSCAPE
41 Sponsors by status
42 Sponsors by phase
43 Recent events
43 Bluebird ‘Turns A Corner’ With Lentiglobin Gene Therapy, Tests Remain
44 BIBLIOGRAPHY
44 Prescription information
45 APPENDIX
LIST OF FIGURES
15 Figure 1: Pipeline drugs for sickle cell anemia in the US
15 Figure 2: Pipeline drugs for sickle cell anemia, by company
16 Figure 3: Pipeline drugs for sickle cell anemia, by drug type
16 Figure 4: Pipeline drugs for sickle cell anemia, by drug classification
27 Figure 5: Voxelotor for Sickle Cell Anemia (December 3, 2018): Phase III – HOPE
30 Figure 6: Voxelotor for Sickle Cell Anemia (June 27, 2018): Phase III – HOPE
32 Figure 7: Altemia for Sickle Cell Anemia (April 30, 2018): Phase II – dose finding (SCOT)
34 Figure 8: Key upcoming events in sickle cell anemia
36 Figure 9: Probability of success in the sickle cell anemia pipeline
37 Figure 10: Licensing and asset acquisition deals in sickle cell anemia, 2014–19
39 Figure 11: Parent patents in sickle cell anemia
40 Figure 12: Clinical trials in sickle cell anemia
40 Figure 13: Top 10 drugs for clinical trials in sickle cell anemia
41 Figure 14: Top 10 companies for clinical trials in sickle cell anemia
41 Figure 15: Trial locations in sickle cell anemia
42 Figure 16: Sickle cell anemia trials status
43 Figure 17: 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
17 Table 3: Pipeline drugs for sickle cell anemia in the US
21 Table 4: LentiGlobin for Sickle Cell Anemia (December 3, 2018)
23 Table 5: RVT-1801 for Sickle Cell Anemia (December 3, 2018)
25 Table 6: Voxelotor for Sickle Cell Anemia (December 3, 2018)
28 Table 7: BCL11a shRNA(miR) for Sickle Cell Anemia (November 1, 2018)
29 Table 8: Voxelotor for Sickle Cell Anemia (June 27, 2018)
31 Table 9: Altemia for Sickle Cell Anemia (April 30, 2018)
32 Table 10: LentiGlobin for Sickle Cell Anemia (December 9, 2017)
© Informa UK Ltd. This document is a licensed product and is not to be reproduced or redistributed
Do you have a subscription to Datamonitor Healthcare, Biomedtracker or Meddevicetracker? You may already have access to these reports, contact your account manager or email pharma@informabi.com for further help or assistance.
Sign up to the