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Market Spotlight: Sickle Cell Anemia

  • ID: 4421373
  • Report
  • November 2020
  • Region: Global
  • 38 pages
  • Datamonitor Healthcare
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FEATURED COMPANIES

  • CRISPR Therapeutics
  • Global Blood Therapeutics
  • Novartis
  • Pfizer
  • Vertex Pharmaceuticals
  • MORE
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 majority 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 topline Phase III trial results for Brilinta.

The overall likelihood of approval of a Phase I hematology asset is 25.9%, and the average probability a drug advances from Phase III is 74.8%. Drugs, on average, take 9.3 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 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 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
Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • CRISPR Therapeutics
  • Global Blood Therapeutics
  • Novartis
  • Pfizer
  • Vertex Pharmaceuticals
  • MORE
OVERVIEW

KEY TAKEAWAYS

DISEASE BACKGROUND

TREATMENT
  • Bone marrow transplantation
  • Hydroxyurea
  • Blood transfusions
  • Prophylactic therapy
  • Pain management
EPIDEMIOLOGY

MARKETED DRUGS

PIPELINE DRUGS

RECENT EVENTS AND ANALYST OPINION
  • 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
  • Global Blood Therapeutics' Oxbryta Approved Broadly For Sickle Cell Disease
  • FDA Approval For Novartis’s Sickle Cell Treatment Adakveo
PROBABILITY OF SUCCESS

LICENSING AND ASSET ACQUISITION DEALS
  • Pfizer Ends Pact With GlycoMimetics After SCD Failure
  • Global Blood Partners With Syros For Sickle Cell, Beta Thalassemia Therapies
  • Vertex Partners With Molecular Templates To Enhance Stem Cell Candidates
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 (June 12, 2020): Phase IIa - IMR-SCD-102
  • Figure 6: IMR-687 for Sickle Cell Anemia (February 14, 2020): Phase IIa - IMR-SCD-102
  • Figure 7: Rivipansel for Sickle Cell Anemia (August 2, 2019): Phase III - RESET
  • Figure 8: Key upcoming events in sickle cell anemia
  • Figure 9: Probability of success in the hematology pipeline
  • Figure 10: Clinical trials in sickle cell anemia
  • Figure 11: Top 10 drugs for clinical trials in sickle cell anemia
  • Figure 12: Top 10 companies for clinical trials in sickle cell anemia
  • Figure 13: Trial locations in sickle cell anemia
  • Figure 14: Sickle cell anemia trials status
  • Figure 15: 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: IMR-687 for Sickle Cell Anemia (June 12, 2020)
  • Table 4: IMR-687 for Sickle Cell Anemia (February 14, 2020)
  • Table 5: CTX001 for Sickle Cell Anemia (November 19, 2019)
  • Table 6: Rivipansel for Sickle Cell Anemia (August 2, 2019)
Note: Product cover images may vary from those shown
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  • CRISPR Therapeutics
  • Global Blood Therapeutics
  • Novartis
  • Pfizer
  • Vertex Pharmaceuticals
Note: Product cover images may vary from those shown
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