U.S. Sickle Cell Disease Treatment Market Size, Share & COVID-19 Impact Analysis, By Treatment Modality [Bone Marrow Transplant, Blood Transfusion, Pharmacotherapy {Hydroxyurea, Branded Products (Endari, Adakveo, Oxbryta, Zynteglo, PYRUKYND (Mitapivat), CTX001, Inclacumab, MGTA-145, Vamifeport (VIT-2763), ALXN1820, FT-4202, and GBT021601)}], By End-user (Hospitals, Specialty Clinics, and Others), and Country Forecast, 2023-2030

The U.S. sickle cell disease treatment market size was worth USD 1.44 billion in 2022 and is projected to grow at a CAGR of 17.3% during the forecast period.

A sickle cell disease is a group of blood disorders that is inherited. It includes hemoglobin sickle cell disease, hemoglobin SS disease, sickle cell anemia, and others, causing red blood cell deformation. Its treatment includes blood transfusions, bone-marrow transplants, and medications. The prevalence of sickle cell disease has been increasing significantly.

• For instance, as per data published by the Centers for Disease Control and Prevention in 2020, around 100,000 Americans had sickle cell disease.

The rising prevalence of the disease has created awareness among the population about effective treatment of the condition. Moreover, the increasing market players’ focus on effective drug development has led to an introduction of novel drugs in the U.S. market. These factors are fueling the U.S. sickle cell disease treatment market growth.

During the COVID-19 pandemic in 2020, the U.S. market remained unaffected. This was due to the new approvals and commercialization of new drugs in the pharmacotherapy segment during 2019-2020.

LATEST TRENDS

Increasing Emphasis on R&D for Sickle Cell Disease Treatment

The burden of sickle cell disease has been significantly increasing. Citing this, the government and other organizations are emphasizing improving the accessibility to disease treatment. The government body in the U.S. has been working on creating awareness about the disease and available treatment options in order to manage the disease in the country. 

• For instance, the American Society of Hematology Foundation works to create awareness regarding early diagnosis and treatment of SCD. Moreover, the organization is also involved in raising funds for R&D of effective treatment and to train hematologists to effectively manage the disease.

Such initiatives have been fueling the funding and financial support in the R&D for effective sickle cell disease treatment development.

• For instance, the National Institute of Health (NIH) Cure Sickle Cell Initiative funded more than USD 366.0 million in 2020 and USD 168.0 million in 2021 in sickle cell disease research. Other NIH funding in sickle cell disease research has increased from USD 80.0 million in 2008 to USD 142.0 million in 2020.

DRIVING FACTORS

Approval of Advanced Pharmacotherapy Drugs to Fuel the Market Growth

Patients with sickle cell disease often experience episodes of acute pain caused by vaso-occlusive crisis (VOC), one of the most common complications of the disease. For instance, the American Society of Hematology (ASH) published a study in 2019 including 16,092 patients. The duration of the study was from January 2001 to June 2018. Out of the total patients, around 14.0% of the patients suffered at least 1 VOC in a year. Moreover, as per the same study, 21.2% of the patients suffered at least 2 VOCs in a year.

Market players have increased their focus on the launch of innovative drugs to control the disease severity and prevent the complications associated with the disease.

• In February 2022, Agios Pharmaceuticals, Inc. received U.S. FDA approval for its product PYRUKYND (mitapivat) indicated for hemolytic anemia (symptom of sickle cell anemia) and pyruvate kinase (PK) deficiency in adults.

The increasing approval of such pharmacotherapy drugs and rising demand for these drugs for effectively managing the disease is fueling the market growth.

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Novo Nordisk A/S’ FT-4202 is currently in phase II clinical trial and the estimated completion of the phase II clinical trial is in July 2025. FT-4202 is anticipated to be commercialized in 2028. Similarly, Dianthus Therapeutics’ MGTA-145 is in the phase II clinical trial, and its estimated commercialization is in 2027. Moreover, Pfizer Inc.’s Inclacumab is in phase III clinical trials and is expected to launch in 2025.

RESTRAINING FACTORS

High Treatment Cost is Hampering the Market Growth

A significant increase in the prevalence of sickle disease has fueled the demand for effective treatment options. Pharmacotherapy drugs have proven to be quite effective in sickle cell disease management. However, treatment through these drugs is costly.

• For instance, the annual cost of treatment with Novartis AG’s Adakveo is around USD 88,000, per patient.

Such high cost of these drugs limits their adoption among the population, negatively impacting the market growth.  

SEGMENTATION

By Treatment Modality Analysis

Based on treatment modality, the U.S. market is classified into bone marrow transplant, blood transfusion, and pharmacotherapy. The pharmacotherapy segment is bifurcated into hydroxyurea and branded products. The branded products are further sub-segmented into Adakveo, Oxbryta, Endari, Zynteglo, CTX001, PYRUKYND (Mitapivat), Inclacumab, Vamifeport (VIT-2763), ALXN1820, MGTA-145, FT-4202, and GBT021601.

The pharmacotherapy segment is expected grow at the fastest CAGR during the forecast period. The approval and commercialization of pipeline drugs is expected to fuel the segment’s growth during the forecast period. For instance, Global Blood Therapeutics’ pipeline drug Inclacumab, which is currently undergoing phase III clinical trials, is expected to launch in 2025 in the U.S.  

By End-user Analysis

On the basis of end-user, the U.S. market is trifurcated into specialty clinics, hospitals, and others.

The hospitals segment dominated the market in 2022. Bone marrow transplant and blood transfusion procedures are mostly conducted in hospitals. This factor is responsible for the segment’s dominance. Moreover, the increasing hospital admissions due to VOC episodes has also been fueling the segment’s growth.

KEY INDUSTRY PLAYERS

Market players such as Global Blood Therapeutics Inc. (Pfizer Inc.), Novartis AG, and Emmaus Medical, Inc. accounted for a significant U.S. sickle cell disease treatment market share in 2022. The growth of these companies in the market is attributed to the market players’ increasing emphasis on partnerships to expand the accessibility of their products.

• In November 2021, Emmaus Life Sciences, Inc. partnered with UpScript IP Holdings, LLC. (UpScript), to offer telehealth services to sickle cell disease patients, to expand the accessibility of Endari.

Other players in the market include Forma Therapeutics, Inc., Alexion Pharmaceuticals, and Magenta Therapeutics, Inc. These players have increased their focus on developing advanced pharmacotherapy products to strengthen their position in the U.S. market.

LIST OF KEY COMPANIES PROFILED:

• Bristol-Myers Squibb Company (U.S.)


• Addmedica (France)


• Novartis AG (Switzerland)


• Global Blood Therapeutics, Inc. (Pfizer Inc.) (U.S.)


• Emmaus Medical, Inc. (U.S.)


• Bluebird Bio Inc. (U.S.)


• Agios Pharmaceuticals, Inc. (U.S.)

KEY INDUSTRY DEVELOPMENTS:

• August 2022 – Bluebird Bio Inc. received U.S. Food and Drug Administration (FDA) approval for ZYNTEGLO for the treatment of beta‑thalassemia in adult and pediatric patients.


• August 2019 – Emmaus Medical, Inc. partnered with Oncology Supply for the distribution of Endari.


• March 2019 – Emmaus Medical, Inc. signed an agreement with Express Scripts, a pharmacy benefits manager (PBM). This agreement ensured that patients in need had access to Emmaus's Endari.

REPORT COVERAGE

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The U.S. sickle cell disease treatment market analysis report provides a detailed analysis of the market. The report includes market segmentation, key points such as pipeline analysis, and the product portfolio of the key players operating in the U.S. market. Additionally, it includes the adoption of pharmacotherapy drugs in the U.S., new product launches, key industry developments such as mergers, collaborations, acquisitions, and the impact of COVID-19 pandemic on the market. Besides this, the report also offers insights into the market trends and highlights key industry dynamics. In addition to the aforementioned factors, it encompasses several factors that have contributed to the growth of the market over recent years.

Report Scope & Segmentation