Market Spotlight: Sickle Cell Anemia

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Report
45 Pages
February 2021
Region: Global
Citeline
ID: 4421373

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.

Key Takeaways

Using an estimated global prevalence of 0.04%, the publisher 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 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 topline Phase IIb trial results for IMR-687, and an expected CHMP opinion for Oxbryta.
The overall likelihood of approval of a Phase I hematology asset is 24.8%, and the average probability a drug advances from Phase III is 75.5%. Drugs, on average, take 9.2 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 77% of trials in Phase I–II, and only 23% 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

OVERVIEWKEY TAKEAWAYSDISEASE BACKGROUND

TREATMENT

Bone marrow transplantation
Hydroxyurea
Blood transfusions
Prophylactic therapy
Pain management

EPIDEMIOLOGYMARKETED DRUGSPIPELINE DRUGS

RECENT EVENTS AND ANALYST OPINION

Zynteglo for Sickle Cell Anemia (February 16, 2021)
IMR-687 for Sickle Cell Anemia (January 6, 2021)
Multiple Drugs for Sickle Cell Anemia (December 7, 2020)
IMR-687 for Sickle Cell Anemia (June 12, 2020)
IMR-687 for Sickle Cell Anemia (February 14, 2020)
CTX001 for Sickle Cell Anemia (November 19, 2019)
Rivipansel for Sickle Cell Anemia (August 2, 2019)

KEY UPCOMING EVENTS

KEY REGULATORY EVENTS

New Therapeutic Targets & A Rich Year For Approvals: EMA’s PRIME In 2020

PROBABILITY OF SUCCESS

LICENSING AND ASSET ACQUISITION DEALS

Pfizer Ends Pact With GlycoMimetics After SCD Failure

CLINICAL TRIAL LANDSCAPE

Sponsors by status
Sponsors by phase
Recent events

BIBLIOGRAPHY

Prescription information

APPENDIX

LIST OF FIGURES

Figure 1: Overview of pipeline drugs for sickle cell anemia in the US
Figure 2: Pipeline drugs for sickle cell anemia, by company
Figure 3: Pipeline drugs for sickle cell anemia, by drug type
Figure 4: Pipeline drugs for sickle cell anemia, by classification
Figure 5: IMR-687 for Sickle Cell Anemia (January 6, 2021): Phase IIa - IMR-SCD-102
Figure 6: IMR-687 for Sickle Cell Anemia (June 12, 2020): Phase IIa - IMR-SCD-102
Figure 7: IMR-687 for Sickle Cell Anemia (February 14, 2020): Phase IIa - IMR-SCD-102
Figure 8: Rivipansel for Sickle Cell Anemia (August 2, 2019): Phase III - RESET
Figure 9: Key upcoming events in sickle cell anemia
Figure 10: Probability of success in the hematology pipeline
Figure 11: Clinical trials in sickle cell anemia
Figure 12: Top 10 drugs for clinical trials in sickle cell anemia
Figure 13: Top 10 companies for clinical trials in sickle cell anemia
Figure 14: Trial locations in sickle cell anemia
Figure 15: Sickle cell anemia trials status
Figure 16: Sickle cell anemia trial sponsors, by phase

LIST OF TABLES

Table 1: Marketed drugs for sickle cell anemia
Table 2: Pipeline drugs for sickle cell anemia in the US
Table 3: Zynteglo for Sickle Cell Anemia (February 16, 2021)
Table 4: IMR-687 for Sickle Cell Anemia (January 6, 2021)
Table 5: Multiple Drugs for Sickle Cell Anemia (December 7, 2020)
Table 6: IMR-687 for Sickle Cell Anemia (June 12, 2020)
Table 7: IMR-687 for Sickle Cell Anemia (February 14, 2020)
Table 8: CTX001 for Sickle Cell Anemia (November 19, 2019)
Table 9: Rivipansel for Sickle Cell Anemia (August 2, 2019)

Table of Contents

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