Global Sickle Cell Disease Treatment Market Size, Share & Industry Trends Analysis Report By Treatment (Blood Transfusion, Pharmacotherapy, and Bone Marrow Transplant), By End-Use (Hospitals, Specialty Clinics), By Regional Outlook and Forecast, 2023 - 2030

The Global Sickle Cell Disease Treatment Market size is expected to reach $7.3 billion by 2030, rising at a market growth of 27.9% CAGR during the forecast period.

According to a study by NCBI in 2021, the disease can expose patients to up to 18 VOCs per year. The increasing prevalence of SCD and the efficacy of the products used to treat the disease in decreasing the risk of VOCs are boosting the product's usage among the patient population. Therefore, The Bone Marrow Transplant segment would capture 1/5thshare in the market by 2030, as the incidence of SCD disease has been rising more quickly. Vaso-occlusive crisis is a frequent and excruciating complication of sickle cell disease in adolescents and adults. By replacing the patient's diseased bone marrow with healthy donor marrow, this procedure can effectively eliminate the root cause of SCD, which is the production of abnormal hemoglobin. It causes episodes of acute, excruciating pain causing patients, including infants and adults, to seek urgent medical care.



The major strategies followed by the market participants are Partnerships as the key developmental strategy to keep pace with the changing demands of end users. For instance, In June, 2022, Novartis AG came into partnership with Precision BioSciences, Inc, to develop solutions for in vivo gene editing. Through the partnership, Novartis AG would be better positioned to provide effective treatment for patients with hemoglobinopathies. Additionally, In November, 2021, Emmaus Life Sciences, Inc. partnered with UpScript IP Holdings, LLC, to provide telehealth solutions for patients with sickle cell disease.

The Cardinal Matrix - Market Competition Analysis

Based on the analysis presented in the The Cardinal Matrix; Pfizer Inc. is the forerunner in the Market. Companies such as Novo Nordisk A/S, Novartis AG, Bristol Myers Squibb Company are some of the key innovators in the Market. In October, 2019, Novo Nordisk A/S signed a partnership with bluebird bio, Inc, to develop in vivo genome editing treatments for inherited diseases. Through the partnership, Novo Nordisk would be better positioned to develop disease-modifying medicines.

COVID-19 Impact Analysis

Workflows in the healthcare industry were hampered by the COVID-19 pandemic all over the world. Several industries, including several sub-domains of healthcare, were compelled to close temporarily due to the disease outbreak. A further factor limiting market expansion was the disruption of the sickle cell disease drug supply chain during COVID-19. Additionally, the lockdown had a detrimental effect on hospital admissions, affecting the market. In the later phases of the pandemic, the growth of the blood transfusion and bone marrow transplant industries returned to normal due to a rise in population vaccination, the implementation of stringent COVID-19 guidelines, and the lifting of travel restrictions. Therefore, the pandemic had a significant negative impact on the market.

Market Growth Factors

Development of new treatment methods

Earlier, blood transfusions and bone marrow transplants were the only treatments for SCD. The increasing prevalence and awareness of the disease boosted the demand for alternative, effective treatment types. Major players have increased their emphasis on introducing new products to treat this disease to meet market demand. Endari was the first branded drug to be approved in decades, before which there was a limited emphasis on innovation in devising a treatment for the disease. Sickle cell disease patients without a suitable donor may be cured by genetic therapies that alter their own hematopoietic stem cells. Such development increases the number of available treatments for SCD and may cover a large proportion of patient base, and thus, aids in the growth of the market.

Modernization of clinical trials and pharmaceutical research

Leaders in the pharmaceutical research industry are now exploring to implement efficient ways of doing the RD processes, for which machine learning appear to be a promising solution. The top pharmaceutical companies in the world are using machine learning to better the hit-and-miss process of discovering new treatments. In addition, many large pharmaceutical companies are investigating the potential of big data to speed up the drug discovery process. This would lead to smaller, quicker, and more affordable trials overall. For greater safety, ML can also be used for remote monitoring and real-time data access. Therefore, the advancement of clinical trials and R&D in the pharma sector with ML, which enables faster and more effective drug development, will propel the market growth.

Market Restraining Factors

Minimal treatment options available for SCD

Hydroxyurea and a few branded medications are the pharmacotherapy used to treat sickle cell disease. Numerous healthcare professionals recommend hydroxyurea as the first-line treatment for the disease. However, the absence of treatment options in countries and regions around the world stands out as a significant constraining factor. Despite its inclusion on the WHO Model List of Essential Medicines for Children, hydroxyurea is still unavailable in the region. In addition, the substance is considered too costly in Africa. In addition, the lack of awareness of the disease among the population in developing economies can hinder the development of the market during the forecast period.

Treatment Outlook

By treatment, the market is categorized into blood transfusion, pharmacotherapy, and bone marrow transplant. The bone marrow transplant segment covered a considerable revenue share in the market in 2022. The segment has experienced rapid expansion due to the emergence of new, branded pharmaceutical medications and growing government incentives to launch these drugs early. The government provides funding and designations such as orphan pharmaceuticals, fast track, and priority review, among others, to support sickle cell disease research.

End-Use Outlook

Based on end-use, the market is classified into hospitals, specialty clinics, and others. In 2022 the hospitals segment witnessed the largest revenue share in the market. The primary centres for disease diagnosis and treatment are hospitals. Most blood transfusions and bone marrow transplants are performed in hospitals. In addition, superior healthcare services, a large patient pool, and an enhanced healthcare infrastructure are some of the primary reasons for the segment's large market share.

Regional Outlook

Region wise, the market is analysed across North America, Europe, Asia Pacific, and LAMEA. In 2022 the North America region led the market by generating the highest revenue share. The expansion in the region can be attributed to the increased availability of SCD treatments and promising pipeline candidates. Strong government support in the United States will further promote market growth. The region's market is anticipated to grow faster than average due to the increasing incidence of sickle cell disease, the rising number of clinical trials, and the increasing number of product launches.

The market research report covers the analysis of key stakeholders of the market. Key companies profiled in the report include Novartis AG, Pfizer Inc., bluebird bio, Inc., CSL Limited, Emmaus Life Sciences, Inc., Bristol Myers Squibb Company, CRISPR Therapeutics AG, Agios Pharmaceuticals, Inc. (LES LABORATOIRES SERVIER SAS), Novo Nordisk A/S and Medunik USA (Duchesnay Inc.)

Strategies Deployed in the Market

Partnerships, Collaborations and Agreements:

• Jun-2022: Novartis AG came into partnership with Precision BioSciences, Inc., a biotechnology company based in the United States, to develop solutions for in vivo gene editing. Through the partnership, Novartis AG would be better positioned to provide effective treatment for patients with hemoglobinopathies.
• Nov-2021: Emmaus Life Sciences, Inc. partnered with UpScript IP Holdings, LLC, a pharmaceutical company, to provide telehealth solutions for patients with sickle cell disease. The partnership would allow Emmaus Life Sciences to provide patients with access to its Endari treatment.
• Jul-2021: Bluebird Bio, Inc. signed a partnership with National Resilience, Inc., a biotechnology company, to develop solutions for cell therapies. The partnership comes in line with Bluebird's ambition for the development of effective cell therapies.
• Apr-2021: CRISPR Therapeutics AG entered into a partnership with Vertex Pharmaceuticals Incorporated, a US-based biotechnology company, to market its CTX001 gene editing therapy for sickle cell disease treatment. The partnership would extend the global reach of CRISPR's CTX001 gene editing therapy.
• Feb-2021: Novartis AG announced a partnership with Bill & Melinda Gates Foundation, an American private foundation, to develop solutions for vivo gene therapy for sickle cell disease treatment. The partnership would pave the way for effective sickle cell disease treatment for patients around the world.

Product Launches and Product Expansions:

• Apr-2022: Emmaus Life Sciences, Inc. announced the launch of a full-service telehealth solution. The solutions feature improved access to Endari sickle disease treatment drugs. The launch extends Endari's reach to a wider population of patients with sickle cell disease.

Mergers & Acquisition:

• Oct-2022: Pfizer Inc. acquired Global Blood Therapeutics, Inc., a biopharmaceutical company. The acquisition strengthens Pfizer's capabilities to provide sickle cell disease treatment solutions.
• Sep-2022: Novo Nordisk A/S announced the acquisition of Forma Therapeutics, a biotech company specializing in sickle cell disease (SCD) treatment. The acquisition enhances Novo Nordisk's sickle cell disease treatment offerings.

Trials and Approvals:

• Jul-2023: The Oman Ministry of Health approved Emmaus Life Sciences' Endari, an Oral medicine used for the treatment of sickle cell disease.
• Dec-2021: Medunik USA received approval from The Food and Drug Administration (FDA) for new applications of its Siklos, a sickle cell anemia treatment solution.
• Nov-2020: Agios Pharmaceuticals, Inc. received approval from the U.S. Food and Drug Administration (FDA) for its mitapivat, a pyruvate kinase R (PKR) activator used for the treatment of sickle cell disease.
• Oct-2020: Novartis AG received approval from the European Commission (EC) for its Adakveo, a sickle cell disease treatment used for the prevention of recurrent vaso-occlusive crises (VOCs).

Scope of the Study

By Treatment

• Blood Transfusion
• Pharmacotherapy
• Bone Marrow Transplant

By End-Use

• Hospitals
• Specialty Clinics
• Others

By Geography

• North America
• US
• Canada
• Mexico
• Rest of North America

• Europe
• Germany
• UK
• France
• Russia
• Spain
• Italy
• Rest of Europe

• Asia Pacific
• China
• Japan
• India
• South Korea
• Singapore
• Malaysia
• Rest of Asia Pacific

• LAMEA
• Brazil
• Argentina
• UAE
• Saudi Arabia
• South Africa
• Nigeria
• Rest of LAMEA

Key Market Players

List of Companies Profiled in the Report:

• Novartis AG
• Pfizer Inc.
• bluebird bio, Inc.
• CSL Limited
• Emmaus Life Sciences, Inc.
• Bristol Myers Squibb Company
• CRISPR Therapeutics AG
• Agios Pharmaceuticals, Inc. (LES LABORATOIRES SERVIER SAS)
• Novo Nordisk A/S
• Medunik USA (Duchesnay Inc.)

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