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Sickle Cell Disease Treatment Market Set for Robust Growth to US$ 9.97 Billion by 2033, Led by North America's 37% Market Share | DataM Intelligence

12-02-2025 07:43 AM CET | Health & Medicine

Press release from: DataM intelligence 4 Market Research LLP

Sickle Cell Disease Treatment Market

Sickle Cell Disease Treatment Market

The Global Sickle Cell Disease Treatment Market reached approximately USD 2.99 billion in 2024 and is expected to reach about USD 9.97 billion by 2033, growing at a CAGR of around 14.8% during the forecast period from 2025 to 2033, according to the DataM Intelligence report.

This significant growth is driven by the increasing prevalence of sickle cell disease, advancements in gene therapies and novel treatment modalities, rising healthcare expenditure, and supportive government initiatives.

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United States: Key Industry Developments
✅ September 2025: Pfizer Inc. voluntarily discontinued Oxbryta (voxelotor) worldwide after assessing that benefits no longer outweigh risks, recalling all lots and halting clinical trials, disrupting the sickle cell disease treatment market.​

✅ Early 2025: Market faced ongoing disruption from Oxbryta discontinuation, amid rising prevalence with nearly 100,000 U.S. cases, driving focus on alternative novel therapeutics and gene therapies.​

Europe: Key Industry Developments
✅ November 2023: UK's MHRA approved Casgevy (exagamglogene autotemcel) by Vertex and CRISPR Therapeutics as the first CRISPR-based gene therapy for sickle cell disease. Early 2025 rollout planned by NHS for 50 patients annually in London, Manchester, and Birmingham using lentiviral vectors.​

✅ 2025: NHS advances access to approved gene therapies like Casgevy, delivering functional beta-globin genes to stem cells, with 88% of trial patients resolving vaso-occlusive events within 6-18 months post-treatment.​

Key Merges and Acquisitions(2025):
✅ Vertex Pharmaceuticals/CRISPR Therapeutics receive EU approval for Casgevy: In November 2025, Vertex Pharmaceuticals and CRISPR Therapeutics received full marketing authorization from the European Commission for Casgevy (exagamglogene autotemcel), a one-time gene therapy for severe sickle cell disease (SCD) and beta-thalassemia, marking a critical commercial milestone in Europe.

✅ FDA approves first-in-class treatment for SCD: In October 2025, the U.S. FDA approved a new oral small molecule therapeutic for the treatment of SCD in patients aged 12 and older, offering a novel mechanism of action to prevent vaso-occlusive crises (VOCs).

✅ Sobi receives FDA approval for Doptelet in pediatric ITP: In July 2025, Sobi received FDA approval for Doptelet. While this treats immune thrombocytopenia (ITP), it highlights successful pediatric label expansions in hematological disorders, a key strategic pathway for SCD treatments.

✅ AstraZeneca presents positive data for Lynparza: In September 2025, AstraZeneca presented oncology data. The broader pharmaceutical conference circuit in Q3 saw heavy emphasis on gene therapies and hematology innovations, setting the stage for future SCD developments.

Market Segmentation Analysis:
-By Disease Type: Hemoglobin SS (HbSS) Leads with Around 60%
Hemoglobin SS (HbSS) is the dominant disease type segment in the sickle cell disease treatment market, holding about 60% market share due to its higher prevalence and severity among sickle cell disease patients. Hemoglobin SC (HbSC) and Hemoglobin (HbS) Beta Thalassemia follow as significant segments, representing milder variants of the disease. Others include less common genotypes that complicate disease management but account for smaller shares.

-By Treatment Type: Blood Transfusions Lead with About 49.5%
Blood transfusions hold the highest market share of approximately 49.5% in 2024, as they are a critical, widely used treatment to replace abnormal sickle-shaped red blood cells with healthy ones. Pharmacotherapy, including drugs like Hydroxyurea, Crizanlizumab, and Voxelotor, is a major segment due to its cost-effectiveness and symptom management role. Bone marrow transplantation, while more curative, holds a smaller share but is growing rapidly with advances in gene therapy.

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Growth Drivers:
-Advancements in gene therapies, including CRISPR and lentiviral vectors, offer curative potential and reduce reliance on lifelong treatments.​

-Expanded healthcare access and infrastructure enable earlier diagnoses and comprehensive management for more patients.​

-Rising prevalence of sickle cell disease, particularly in sub-Saharan Africa and parts of Asia, increases demand for effective therapies.​

-Growing investments in R&D by pharmaceutical companies, governments, and venture capital fuel innovative treatment development.​

-Newborn screening programs and improved diagnostic tools facilitate early interventions and better prognoses.​

-Regulatory support, such as fast-track FDA approvals, and increased awareness accelerate therapy availability and adoption.

Regional Insights:
-North America commands the highest market share in the sickle cell disease treatment market at approximately 37-38.7%, driven by advanced healthcare infrastructure, high disease awareness, robust regulatory support from the FDA, and the presence of major pharmaceutical players like Pfizer developing innovative therapies such as gene-editing treatments.​

-Europe holds the next largest share, estimated around 20-25% based on its significant position behind North America, fueled by government funding, increasing patient prevalence, and key market players expanding operations in the region.​

-Asia Pacific follows with a growing but smaller share of about 15-20%, propelled by rising disease prevalence, enhanced government initiatives, improved patient awareness, and faster adoption rates compared to other regions.

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Key Players:
Novartis AG | Vertex Pharmaceuticals Incorporated | bluebird bio, Inc. | Emmaus Medical, Inc. | Bristol-Myers Squibb Company | CHIESI FARMACEUTICI S.p.A. | Teva Pharmaceutical Industries Ltd.

Key Highlights (Top 5 Key Players) for Sickle Cell Disease Treatment Market:
-Novartis AG is a major player in the sickle cell disease treatment market, contributing significantly to the global market revenues, with a strong presence in North America and ongoing development in gene therapies.

-Vertex Pharmaceuticals Incorporated also holds a key position, known for its innovative treatment approaches and robust market revenue generation within this sector.

-bluebird bio, Inc. is notable for advancements in gene therapy treatments for sickle cell disease, contributing substantially to the market revenue.

-Emmaus Medical, Inc. is an important player with a reported quarterly revenue of approximately USD 3.38 million as of Q3 2025, reflecting its growing impact in the treatment landscape.

-Bristol-Myers Squibb Company is another key player, benefiting from its pharmaceutical portfolio and market penetration in sickle cell disease therapies.

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