Emerging therapies for Sickle Cell Disease, including CASGEVY (exagamglogene autotemcel), ENDARI (L-glutamine), Mitapivat, Inclacumab, and others, are anticipated to drive significant growth in the Sickle Cell Disease market over the coming years.
DelveInsight has introduced a new report, “Sickle Cell Disease – Market Insights, Epidemiology, and Market Forecast–2034”, which provides comprehensive insights into the disorder, covering historical and projected epidemiology along with market trends across the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
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Some of the key facts of the Sickle cell disease Market Report:
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In 2023, the Sickle Cell Disease (SCD) market across the 6 major markets (6MM) was valued at nearly USD 650 million, with the United States contributing the majority share (~USD 603 million), followed by France and the United Kingdom. SCD is an inherited condition caused by mutations in the HBB gene, leading to sickle-shaped red blood cells that obstruct circulation and trigger painful vaso-occlusive crises. The disease primarily affects individuals of African, Middle Eastern, Mediterranean, and South Asian descent.
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Common complications include chronic anemia, stroke, organ damage, and infections. Current treatment approaches range from NSAIDs, blood transfusions, and iron chelators to FDA-approved drugs such as hydroxyurea (DROXIA), L-glutamine (ENDARI), crizanlizumab (ADAKVEO), and voxelotor (OXBRYTA)—the first oral therapy addressing the root cause of the disorder.
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Despite recent progress, challenges such as treatment adherence and reimbursement barriers persist. The approval of gene therapies CASGEVY (exa-cel) and LYFGENIA (Lovo-cel) marks a significant milestone and is expected to accelerate market growth. The development pipeline also includes promising candidates such as EDIT-301, Mitapivat, and Inclacumab.
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Market barriers remain, including delayed diagnosis, severe complications, high treatment costs, and limited awareness. Still, continued research and innovation are paving the way for improved disease management and potential curative options.
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In December 2023, CASGEVY became the first FDA-approved CRISPR/Cas9-based gene-editing therapy for SCD, targeting patients aged 12 and above with recurrent vaso-occlusive crises. The treatment involves harvesting hematopoietic stem cells, genetically modifying them to boost fetal hemoglobin production, and reinfusing them to reduce red cell sickling and complications.
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Key players such as Global Blood Therapeutics, Pfizer, Agios Pharmaceuticals, Editas Medicine, Forma Therapeutics, Novo Nordisk, Bausch Health, Bluebird Bio, and others are actively advancing novel therapies to reshape the treatment landscape. Emerging treatments like CASGEVY, ENDARI, Mitapivat, and Inclacumab are expected to drive substantial market expansion between 2024 and 2034.
Sickle cell disease Overview
SCD is a hereditary blood disorder caused by mutations in the HBB gene, which leads to the production of abnormal hemoglobin known as hemoglobin S. This results in red blood cells assuming a rigid, crescent or “sickle” shape, reducing their flexibility and lifespan.
SCD primarily affects individuals of African, Middle Eastern, Mediterranean, and South Asian descent. Globally, millions are affected, with significant prevalence in sub-Saharan Africa and the United States.
The sickle-shaped cells can block blood flow in small vessels, causing vaso-occlusive crises (painful episodes), chronic anemia, organ damage, and increased risk of infections. The shortened lifespan of these cells contributes to anemia.
Sickle cell disease Market Outlook
Sickle Cell Disease (SCD) is a group of inherited, lifelong disorders that affect hemoglobin. It is a chronic hemolytic condition in which hemoglobin within red blood cells polymerizes, causing them to assume a rigid, crescent or “sickle” shape (Hb S). This abnormal morphology leads to vaso-occlusive events and accelerated red blood cell destruction.
SCD mainly affects large blood vessels, but medium and small arteries can also be involved. The condition is inherited in an autosomal manner, either homozygously or as a compound heterozygote. Homozygous inheritance results in sickle cell anemia (SCA). Other genotypes include hemoglobin SC disease, sickle beta-plus thalassemia, sickle beta-zero thalassemia (comparable in severity to SCA), hemoglobin SD Punjab, and hemoglobin SO Arab, among others. The Hb S variant differs from normal hemoglobin (Hb A) by a single amino acid substitution in the beta-globin gene.
In the United States, newborns are routinely screened for Hb S. Infants are typically protected during the first six months by high levels of fetal hemoglobin (HbF). SCD symptoms usually appear in early childhood, with acute and chronic pain, especially vaso-occlusive crises, being the most prominent feature. These crises often cause severe bone pain due to bone marrow infarction.
Symptoms generally begin around four months of age, with painful episodes lasting hours to days. The frequency varies widely—some individuals experience one crisis per year, while others have multiple episodes. Severe crises frequently require hospitalization, all resulting from reduced oxygen delivery caused by the sickled red blood cells.
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Sickle cell disease marketed drugs
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CASGEVY (exagamglogene autotemcel): Vertex Pharmaceuticals/CRISPR Therapeutics
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ENDARI (L-glutamine): Emmaus Life Sciences
Sickle cell disease Emerging Drugs
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Mitapivat: Agios Pharmaceuticals
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Inclacumab: Pfizer
Scope of the Sickle cell disease Market Report
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Study Period: 2020-2034
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Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
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Key Sickle cell disease Companies: Global Blood Therapeutics, Pfizer, Agios Pharmaceuticals, Editas Medicine, Forma Therapeutics, Novo Nordisk, Bausch Health, Bluebird Bio, and others
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Key Sickle cell disease Therapies: CASGEVY (exagamglogene autotemcel), ENDARI (L-glutamine), Mitapivat, Inclacumab, and others
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Sickle cell disease Therapeutic Assessment: Sickle cell disease current marketed and Sickle cell disease emerging therapies
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Sickle cell disease Market Dynamics: Sickle cell disease market drivers and Sickle cell disease market barriers
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Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
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Sickle cell disease Unmet Needs, KOL’s views, Analyst’s views, Sickle cell disease Market Access and Reimbursement
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Key benefits of the Sickle cell disease Market Report:
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Sickle cell disease market report covers a descriptive overview and comprehensive insight of the Sickle cell disease Epidemiology and Sickle cell disease market in the 7MM (the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan).
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The Sickle cell disease market report provides insights into the current and emerging therapies.
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The Sickle cell disease market report provides a global historical and forecasted market covering drug outreach in 7MM.
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The Sickle cell disease market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Sickle cell disease market.
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Table of Contents
1. Report Introduction
2. Executive Summary
3. SWOT analysis
4. Sickle cell disease Patient Share (%) Overview at a Glance
5. Sickle cell disease Market Overview at a Glance
6. Sickle cell disease Disease Background and Overview
7. Sickle cell disease Epidemiology and Patient Population
8. Country-Specific Patient Population of Sickle cell disease
9. Sickle cell disease Current Treatment and Medical Practices
10. Unmet Needs
11. Sickle cell disease Emerging Therapies
12. Sickle cell disease Market Outlook
13. Country-Wise Sickle cell disease Market Analysis (2020–2034)
14. Market Access and Reimbursement of Therapies
15. Market drivers
16. Market barriers
17. Appendix
18. Sickle cell disease Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight
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Related Reports:
Sickle cell disease Pipeline Insights, DelveInsight
“Sickle cell disease Pipeline Insight, 2024” report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Sickle cell disease market. A detailed picture of the Sickle cell disease pipeline landscape is provided, which includes the disease overview and Sickle cell disease treatment guidelines.
About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.
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