Sickle Cell Disease Drugs Market 2034: Clinical Trials, Prevalence, EMA, PDMA, FDA Approvals, Medication, Statistics, NICE Approval.Revenue and Companies by DelveInsight

Globe and Mail

07-07-2025

Sickle Cell Disease companies working in the treatment market are Cellectis, Sana Biotechnology, Global Blood Therapeutics, Inc., CSL Behring, Pfizer, Aruvant Sciences, Graphite Bio, Novartis, Agios Pharmaceuticals, Forma therapeutics, Vertex Pharmaceuticals, Global Blood Therapeutics, Inc., Alfasigma, Novo Nordisk, bluebird bio, ExCellThera, Gamida Cell, KM Biologics/Takeda, Editas Medicine, and others.
Sickle Cell Disease Market Summary
In 2023, the Sickle Cell Disease (SCD) market across the 6MM was valued at approximately USD 650 million, with the United States dominating at ~USD 603 million (92% share), followed by France and the UK. SCD is a hereditary blood disorder caused by mutations in the HBB gene, resulting in abnormal hemoglobin (HbS) that distorts red blood cells into a sickle shape. These rigid cells obstruct microvasculature, triggering vaso-occlusive crises, anemia, acute chest syndrome, stroke, organ damage, and heightened infection risk, severely impacting patient quality of life and survival. Historically, management relied on NSAIDs, transfusions, chelators, and supportive care, with hydroxyurea (DROXIA) being the primary disease-modifying agent for decades. Recent approvals of ENDARI (L-glutamine), ADAKVEO (crizanlizumab), and OXBRYTA (voxelotor) have expanded options, though challenges in adherence and reimbursement persist. New approvals like CASGEVY (exa-cel) and LYFGENIA (lovo-cel) are expected to drive market growth, alongside pipeline candidates such as EDIT-301, Mitapivat, Inclacumab, and Osivelotor. Despite advances, delays in diagnosis, economic burden, and complications continue to constrain outcomes. Multidisciplinary care, genetic counseling, early intervention, and patient education are critical, while gene therapy and gene editing hold promise for transformative treatment, aiming to mitigate SCD's substantial clinical and economic impact in the coming decade.
(Albany, USA) DelveInsight's ' Sickle Cell Disease Market Insights, Epidemiology, and Market Forecast-2034 ' report delivers an in-depth understanding of Sickle Cell Disease, historical and forecasted epidemiology as well as the Sickle Cell Disease market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.
The Sickle Cell Disease market report provides current treatment practices, emerging drugs, the market share of the individual therapies, and the current and forecasted Sickle Cell Disease market size from 2020 to 2034, segmented by seven major markets. The Report also covers current Sickle Cell Disease treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying potential of the Sickle Cell Disease market.
Some facts of the Sickle Cell Disease Market Report are:
According to DelveInsight, Sickle Cell Disease market size is expected to grow at a decent CAGR by 2034.
Leading Sickle Cell Disease companies working in the market are Cellectis, Sana Biotechnology, Global Blood Therapeutics, Inc., CSL Behring, Pfizer, Aruvant Sciences, Graphite Bio, Novartis, Agios Pharmaceuticals, Forma therapeutics, Vertex Pharmaceuticals, Global Blood Therapeutics, Inc., Alfasigma, Novo Nordisk, bluebird bio, ExCellThera, Gamida Cell, KM Biologics/Takeda, Editas Medicine, and others.
Key Sickle Cell Disease Therapies expected to launch in the market are BPX-501 T cells, Canakinumab, EPI01, CTX001, ADAKVEO, DROXIA, ENDARI, OXBRYTA, and many others.
In May 2025, Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company specializing in precision genetic medicines via base editing, announced it will share new findings from its BEACON Phase 1/2 clinical trial of BEAM-101 at the upcoming European Hematology Association 2025 Congress (EHA2025), scheduled for June 12–15, 2025, in Milan, Italy. BEAM-101 is an investigational ex vivo genetically modified cell therapy being developed to treat sickle cell disease (SCD), specifically in patients experiencing severe vaso-occlusive crises (VOCs).
In November 2024, BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a commercial-stage biopharmaceutical company focused on oncology and rare diseases, announced that an abstract featuring initial results from a Phase 1 trial of motixafortide—both as a monotherapy and in combination with natalizumab for CD34+ hematopoietic stem cell (HSC) mobilization in gene therapies for sickle cell disease (SCD)—has been accepted for oral presentation at the 66th American Society of Hematology (ASH) Annual Meeting & Exposition, taking place December 7-10, 2024, in San Diego, California. Conducted in collaboration with Washington University School of Medicine in St. Louis, this proof-of-concept study aims to explore alternative HSC mobilization approaches to enhance the treatment experience for SCD patients undergoing gene therapy.
In May 2024, Afimmune announced results of an Open-label Mechanistic Study to Assess the Pharmacokinetics, Pharmacodynamics and Safety of Orally Administered Epeleuton in Patients With Sickle Cell Disease
In April 2024, Pfizer announced results of an Open-label Extension Study to Evaluate the Long-term Safety of GBT021601 Administered to Participants With Sickle Cell Disease Who Have Participated in a GBT021601 Clinical Trial
In April 2024, Novo Nordisk A/S (Forma Therapeutics, Inc.) announced results of an adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral Etavopivat, a Pyruvate Kinase Activator in Patients With Sickle Cell Disease (HIBISCUS).
In July 2023, Novartis announced results of an Open-label, Multi-center, Phase IV, Rollover Study for Patients With Sickle Cell Disease Who Have Completed a Prior Novartis-Sponsored Crizanlizumab Study.
Sickle Cell Disease Overview
Sickle cell disease (SCD) is a hereditary blood disorder characterized by the production of abnormal hemoglobin, known as hemoglobin S. This defect causes red blood cells to assume a rigid, sickle-like shape, which impedes their ability to flow smoothly through blood vessels. Consequently, these misshapen cells can obstruct blood flow, leading to severe pain, organ damage, and an increased risk of infection.
Sickle cell disease is most prevalent among individuals of African, Mediterranean, Middle Eastern, and Indian ancestry. Symptoms typically appear in early childhood and include episodes of pain (called sickle cell crises), chronic anemia, fatigue, swelling in the hands and feet, and delayed growth. Complications can be severe, including stroke, acute chest syndrome, and organ failure.
Management of Sickle cell disease involves both preventive and therapeutic strategies. Preventive measures include regular vaccinations, antibiotics to prevent infections, and hydroxyurea, a medication that reduces the frequency of pain crises and the need for blood transfusions. Pain management, blood transfusions, and bone marrow transplants are critical therapeutic options for managing acute and chronic complications.
Recent advancements in gene therapy and CRISPR technology hold promise for more effective treatments and potential cures for Sickle cell disease. Ongoing research and clinical trials aim to improve the quality of life for patients and reduce the burden of this debilitating disease.
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Sickle Cell Disease Market
The Sickle Cell Disease market outlook of the report helps to build a detailed comprehension of the historical, current, and forecasted Sickle Cell Disease market trends by analyzing the impact of current Sickle Cell Disease therapies on the market and unmet needs, and drivers, barriers, and demand for better technology.
This segment gives a thorough detail of the Sickle Cell Disease market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated Sickle Cell Disease market data are presented with relevant tables and graphs to give a clear view of the market at first sight.
According to DelveInsight, the Sickle Cell Disease market in 7MM is expected to witness a major change in the study period 2020-2034.
Sickle Cell Disease Epidemiology
The Sickle Cell Disease epidemiology section provides insights into the historical and current Sickle Cell Disease patient pool and forecasted trends for seven individual major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the Sickle Cell Disease market report also provides the diagnosed patient pool, trends, and assumptions.
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Sickle Cell Disease Drugs Uptake
This section focuses on the uptake rate of the potential Sickle Cell Disease drugs recently launched in the Sickle Cell Disease market or expected to be launched in 2020-2034. The analysis covers the Sickle Cell Disease market uptake by drugs, patient uptake by therapies, and sales of each drug.
Sickle Cell Disease Drugs Uptake helps in understanding the drugs with the most rapid uptake and the reasons behind the maximal use of new drugs and allows the comparison of the drugs based on Sickle Cell Disease market share and size, which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.
Sickle Cell Disease Pipeline Development Activities
The Sickle Cell Disease report provides insights into different therapeutic candidates in Phase II, and Phase III stages. It also analyses Sickle Cell Disease key players involved in developing targeted therapeutics.
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Sickle Cell Disease Therapeutics Assessment
Major key companies are working proactively in the Sickle Cell Disease Therapeutics market to develop novel therapies which will drive the Sickle Cell Disease treatment markets in the upcoming years are Cellectis, Sana Biotechnology, Global Blood Therapeutics, Inc., CSL Behring, Pfizer, Aruvant Sciences, Graphite Bio, Novartis, Agios Pharmaceuticals, Forma therapeutics, Vertex Pharmaceuticals, Global Blood Therapeutics, Inc., Alfasigma, Novo Nordisk, bluebird bio, ExCellThera, Gamida Cell, KM Biologics/Takeda, Editas Medicine, and others.
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Sickle Cell Disease Report Key Insights
1. Sickle Cell Disease Patient Population
2. Sickle Cell Disease Market Size and Trends
3. Key Cross Competition in the Sickle Cell Disease Market
4. Sickle Cell Disease Market Dynamics (Key Drivers and Barriers)
5. Sickle Cell Disease Market Opportunities
6. Sickle Cell Disease Therapeutic Approaches
7. Sickle Cell Disease Pipeline Analysis
8. Sickle Cell Disease Current Treatment Practices/Algorithm
9. Impact of Emerging Therapies on the Sickle Cell Disease Market
Table of Contents
1. Key Insights
2. Executive Summary
3. Sickle Cell Disease Competitive Intelligence Analysis
4. Sickle Cell Disease Market Overview at a Glance
5. Sickle Cell Disease Disease Background and Overview
6. Sickle Cell Disease Patient Journey
7. Sickle Cell Disease Epidemiology and Patient Population
8. Sickle Cell Disease Treatment Algorithm, Current Treatment, and Medical Practices
9. Sickle Cell Disease Unmet Needs
10. Key Endpoints of Sickle Cell Disease Treatment
11. Sickle Cell Disease Marketed Products
12. Sickle Cell Disease Emerging Therapies
13. Sickle Cell Disease Seven Major Market Analysis
14. Attribute Analysis
15. Sickle Cell Disease Market Outlook (7 major markets)
16. Sickle Cell Disease Access and Reimbursement Overview
17. KOL Views on the Sickle Cell Disease Market
18. Sickle Cell Disease Market Drivers
19. Sickle Cell Disease Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
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