Sickle Cell Disease Treatment Market Set for Robust Growth Through 2034, Driven by Recent Clinical Pipeline Developments | DelveInsight

July 01 20:46 2025
Sickle Cell Disease Treatment Market Set for Robust Growth Through 2034, Driven by Recent Clinical Pipeline Developments | DelveInsight
Sickle Cell Disease Market Insight, Epidemiology And Market Forecast – 2034
The sickle cell disease treatment market is experiencing significant expansion, driven by increasing disease awareness, rising prevalence, and breakthrough therapies from key pharmaceutical players, including Global Blood Therapeutics/Pfizer, Vertex Pharmaceuticals/CRISPR Therapeutics, Agios Pharmaceuticals, Editas Medicine, Forma Therapeutics, Novo Nordisk, Bausch Health, Bluebird Bio, Emmaus Life Sciences, and other emerging biotechnology companies advancing innovative treatment approaches.

DelveInsight’s “Sickle Cell Disease Market Insight, Epidemiology And Market Forecast – 2034” report delivers an in-depth understanding of the sickle cell disease treatment market, historical and forecasted epidemiology, as well as market trends across the US, EU4 (Germany, France, Italy, and Spain) and the United Kingdom. The report comprehensively evaluates the current treatment landscape, emerging therapies, market share of individual treatments, and sickle cell disease market forecasts through 2034, providing crucial insights for stakeholders in the sickle cell disease therapeutic area.

According to DelveInsight’s analysis, the sickle cell disease treatment market is projected to grow substantially during the forecast period (2025-2034), driven by enhanced understanding of disease mechanisms, gene therapy innovations, and breakthrough CRISPR-based treatment approaches.

The sickle cell disease market was valued at approximately USD 650 million in 2023 across the 6MM countries and is expected to increase throughout the forecast period. The United States accounted for the highest market share in 2023, representing approximately USD 603 million, or nearly 92% of the total market share compared to EU4 and the UK. Among EU4 and the UK, France accounted for the largest market share, while Spain accounted for the smallest share.

Download the Sickle Cell Disease Market report to understand which factors are driving the Sickle Cell Disease therapeutic market @ Sickle Cell Disease Market Trends.

The DelveInsight’s report further categorizes the sickle cell disease patient population into several main groups for epidemiological analysis. In the US, among the sickle cell disease type-specific prevalent cases, sickle cell anemia (hemoglobin S/S or hemoglobin S/BO-thalassemia) was identified as the major subtype of sickle cell disease. The report provides comprehensive segmentation, including total prevalent cases of sickle cell disease trait, total prevalent cases of sickle cell disease, diagnosed cases, age-specific prevalent cases, and type-specific prevalence across all studied markets.

Discover evolving trends in the Sickle Cell Disease patient pool forecasts @ Sickle Cell Disease Epidemiology Analysis.

The sickle cell disease treatment landscape has been revolutionized by recent FDA approvals of breakthrough gene therapies. A significant milestone occurred in December 2023 when CASGEVY (exagamglogene autotemcel), developed by Vertex Pharmaceuticals and CRISPR Therapeutics, received FDA approval as the first CRISPR/Cas9 genome-edited cell therapy for treating sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises. Additionally, LYFGENIA (Lovo-cel) from bluebird bio received approval, marking another major advancement in gene therapy approaches for this devastating genetic disorder.

The current treatment paradigm includes several FDA-approved therapies addressing different aspects of sickle cell disease management. ENDARI (L-glutamine) from Emmaus Life Sciences provides oral pharmaceutical-grade L-glutamine to reduce severe complications in adult and pediatric patients aged 5 and older. OXBRYTA (voxelotor) became the first-in-class oral therapy targeting the underlying cause of sickle cell disease with convenient oral administration, while ADAKVEO (crizanlizumab) offers targeted therapy to reduce vaso-occlusive crises.

According to the DelveInsight report, the sickle cell disease therapies pipeline demonstrates robust potential with several novel treatments in late-stage development. Agios Pharmaceuticals’ Mitapivat, a first-in-class oral small molecule allosteric activator of the pyruvate kinase enzyme, has successfully met its primary endpoint in the Phase II portion of the global RISE UP study, demonstrating hemoglobin response for patients in both the 50 mg and 100 mg twice daily mitapivat arms. Pfizer’s Inclacumab (PF-07940370) is currently in Phase III development following the company’s acquisition of Global Blood Therapeutics, while Novo Nordisk’s etavopivat and other pipeline candidates including EDIT-301 (Reni-cel) and Osivelotor (GBT-601) are expected to be major contributors to market growth.

Recent developments highlight the dynamic nature of the sickle cell disease treatment landscape. In June 2025, Beam Therapeutics’ BEAM-101 received FDA orphan drug designation for SCD. BEAM-101 is a precision genetic medicine currently in a Phase 1/2 clinical trial (BEACON) for patients with severe sickle cell disease. Sanofi’s Rilzabrutinib also received FDA orphan drug designation for SCD in the same month. Rilzabrutinib is an oral Bruton’s tyrosine kinase (BTK) inhibitor targeting the reduction of vaso-occlusive crises through immune modulation, demonstrating continued regulatory support for innovative therapeutic approaches.

Furthermore, in March 2025, the FLT1 gene is identified by a group of scientists as a potential new target for gene editing in SCD. Editing FLT1 could increase fetal hemoglobin, which is associated with improved outcomes for sickle cell disease patients.

Discover recent advancements in the Sickle Cell Disease treatment landscape @ Sickle Cell Disease Recent Developments.

The sickle cell disease treatment market remains highly competitive, with major pharmaceutical companies and emerging biotechnology firms advancing various therapeutic approaches. Key players include Global Blood Therapeutics (acquired by Pfizer), Vertex Pharmaceuticals collaborating with CRISPR Therapeutics, Agios Pharmaceuticals, Editas Medicine, Forma Therapeutics (partnered with Novo Nordisk), Bausch Health, Bluebird Bio, and Emmaus Life Sciences, all contributing to a diverse pipeline spanning small molecules, gene therapies, and cell-based treatments.

Looking ahead, the sickle cell disease market is expected to witness continued innovation driven by advances in gene editing technologies, improved understanding of disease pathophysiology, and personalized medicine approaches. While significant progress has been made with the approval of gene therapies, challenges remain in addressing treatment accessibility, affordability, and managing the complex logistics of cell-based therapies. The market outlook indicates positive growth potential, with pipeline candidates such as Etavopivat, Inclacumab, Reni-cel, and Osivelotor expected to drive market expansion during the forecast period.

DelveInsight’s analysis underscores the substantial opportunities that remain for developing more accessible, cost-effective treatment options that can provide durable clinical benefits and improved quality of life for sickle cell disease patients worldwide. As gene therapy technologies mature and novel therapeutic mechanisms are explored, the sickle cell disease market is positioned for remarkable transformation and expansion.

Table of Contents

1. Key Insights

2. Report Introduction

3. Sickle Cell Disease Market Overview at a Glance

4. Sickle Cell Disease Market: Future Perspective

5. Executive Summary of Sickle Cell Disease

6. Key Events

7. Sickle Cell Disease Background and Overview

8. Sickle Cell Disease Market Epidemiology and Patient Population

9. Sickle Cell Disease Patient Journey

10. Sickle Cell Disease Marketed Drugs

11. Sickle Cell Disease Emerging Drugs

12. Sickle Cell Disease: Seven Major Market Analysis

13. Key Opinion Leaders’ Views

14. Sickle Cell Disease Market Unmet Needs

15. SWOT Analysis

16. Reimbursement and Market Access

17. Appendix

18. DelveInsight Capabilities

19. Disclaimer

20. About DelveInsight

Related Reports

Sickle Cell Disease Pipeline Insight

Sickle Cell Disease pipeline insight provides comprehensive insights about the Sickle Cell Disease pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the Sickle Cell Disease companies, including Pfizer, Editas Medicine, Roche, Quercis Pharma, Graphite Bio, Beam Therapeutics Inc., GlaxoSmithKline, Agios Pharmaceuticals, BRL Medicine, Oryzon Genomics, CorrectSequence Therapeutics, GluBio Therapeutics, and Base Therapeutics, among others.

About DelveInsight

DelveInsight is a leading market research and consulting firm specializing in disease-specific insights and therapeutic market analysis. Their reports integrate real-world data, clinical trial findings, and expert interviews to deliver comprehensive industry intelligence.

Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Arpit Anand
Email: Send Email
Phone: +14699457679
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: Nevada
Country: United States
Website: https://www.delveinsight.com/consulting/due-diligence-services

view more articles

About Article Author