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, and FT-4202)}], By End-user (Hospitals, Specialty Clinics, and Others), and Forecast, 2022-2029

The sickle cell disease treatment market size was valued at USD 1.73 billion in 2021 and is projected to grow from USD 2.25 billion in 2022 to USD 8.75 billion by 2029, at a CAGR of 21.4% during the forecast period. Based on our analysis, the global sickle cell disease treatment market exhibited an average growth of 4.8% in 2020 as compared to 2019.    

Sickle Cell Disease (SCD) is an inherited disorder causing diseases such as hemoglobin sickle cell disease, hemoglobin SS disease, and others causing deformation of red blood cells. This leads to early death of cells causing blood shortage and also inhibits the blood flow causing blockage. The disease management is done through medication, blood transfusions, and bone-marrow transplants.

The global sickle cell disease treatment market growth is attributed to the increasing prevalence of SCD, rising awareness regarding the disease, and increasing focus of the key players on the launch of new effective drugs for sickle cell disease treatment.

• In February 2022, Agios Pharmaceuticals, Inc. announced the U.S. Food and Drug Administration (FDA) approval for its product PYRUKYND (mitapivat) for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency, a rare, debilitating, lifelong hemolytic anemia.

COVID-19 IMPACT

Lower Donation Bone Marrow Donations and Availability of Donors Hampered Market Growth During the COVID-19 Pandemic 

The outbreak of COVID-19 had no impact on the pharmacotherapy segment of the market due to the commercialization of branded pharmacotherapy drugs from 2019-2020.

• For instance, in June 2020, Emmaus Medical, Inc. received marketing approval from the Israeli Ministry of Health for the commercial distribution and sale of Endari in Israel.

Moreover, based on revenue, the pharmacotherapy segment in the global market witnessed a growth of 100.1% in 2020 as compared to the prior year.

However, blood transfusion and bone marrow transplant segments experienced a decline in growth in 2020 compared to the prior year due to the reallocation of healthcare resources for COVID-19 treatment. Furthermore, since the number of people opting for sickle cell disease treatment reduced, the market was affected negatively. Moreover, the postponement of non-urgent medical procedures also negatively affected the growth of both segments.

• For instance, as per the data of the COVID-19 Community Survey Report done by the National Organization for Rare Disorders (NORD) published in 2020, around 74.0% of 772 participants in the U.S. suffering from a rare disease had medical appointments canceled to reduce person to person contact and to control the spread of the virus.

Although the blood transfusion and bone marrow transplant segment experienced a negative COVID-19 impact, it was offset by the new launches in the pharmacotherapy segment. Therefore, the global market witnessed stable growth.

In 2021, the growth of the blood transfusion and bone marrow transplant segments bounced back to normal due to increasing vaccination among the population, implementation of strong COVID-19 guidelines, and the dismissal of travel restrictions.

LATEST TRENDS

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Increasing Gene Therapy Adoption Bolstered the Development of Effective SCD Treatments 

Gene therapy has emerged as the ultimate cure for various chronic diseases. In the case of hereditary diseases, gene therapy is a revolution that targets the root cause of a disease. With increasing research on the correction of genetic mutation for the treatment of the disease, market players have shifted their R&D focus toward gene therapy.

• For instance, in February 2018, David Williams, in collaboration with Boston Children's Hospital, started a clinical study to determine the feasibility and safety of administering a lentiviral gene transfer vector encoding a small hairpin (sh) RNA targeting the γ-globin gene repressor, BCL11A, in patients with severe SCD. The study is currently in the phase 1 trial and is expected to complete in April 2024.

Similarly, CRISPR Therapeutics and Vertex Pharmaceuticals have been conducting clinical trials on CRISPR-Cas9 gene-edited therapy CTX001. It is currently in phase 3 clinical and is estimated to complete in October 2024. Positive clinical studies on CTX001 suggest that the pipeline candidate can become a blockbuster drug when it is launched in the market. This factor is expected to significantly grow the global sickle cell disease treatment market in the coming years.

DRIVING FACTORS

Increasing Prevalence along with High Severity Associated with the Disease to Surge the Demand for Effective Treatment Procedures

Sickle cell disease affects millions of people throughout the world. The prevalence of SCD disease has been increasing at a faster pace.

• For instance, in May 2022, as per the data published and updated by CDC, the disease affected nearly 100,000 Americans.

The Vaso-occlusive Crisis (VOCs) is a common painful complication of sickle cell disease in adolescents and adults. It causes acute episodes of severe pain. Patients, including adults and children, need to seek emergency medical care.

• As per a research study published by NCBI in 2021, patients who have the disease can experience up to 18 VOCs in a year.

Major companies operating in the market have increased their focus on the launch of drugs to reduce the severity of these diseases and treat painful complications associated with them.

• For instance, Adakveo, approved by the FDA in November 2019 generated a revenue of USD 164.0 million in 2021, experiencing a growth of USD 56.2% from the prior year. 

The increasing prevalence of SCD and the efficacy of the products for the disease treatment in reducing the chances of VOCs are increasing its adoption among the patient population.

Emergence of New Treatment Modalities to Contribute to Market Growth

Earlier, SCD management only involved blood transfusion and bone marrow transplantation. The increasing prevalence of the disease and rising awareness regarding the disease increased the demand for other effective treatment types.

Major players increased their focus on the launch of new products for the treatment of this disease and to meet demands.

• For instance, in 2017, FDA approved Emmaus Life Sciences' drug Endari for sickle cell disease patients aged five years and older. Until then, there was a limited focus on innovation for developing a treatment for the disease, as Endari was the first branded drug approved in decades.

After the launch of Endari few more companies emerged in the market with new products launches, including Oxbryta (Global Blood Therapeutics), Adakveo (Novartis), Zynteglo (bluebird bio Inc.), and PYRUKYND (Agios Pharmaceuticals, Inc.).

Moreover, FDA and EMA are also playing a significant role by providing speedy approval to these products. FDA provides accelerated approval to drugs for serious conditions to fill an unmet medical need based on a result that is reasonably likely to predict a clinical benefit to patients.

• In November 2019, Global Blood Therapeutics (Pfizer Inc.) announced the FDA approval of Oxbryta 3 months ahead of a statutory deadline for agency action. 

The expected launch of new drugs in the forecast period and the rising prevalence of the disease are likely to fuel the market growth.

RESTRAINING FACTORS

Limited Availability of Treatment Options for SCD in the Emerging Countries to Limit Market Growth

The pharmacotherapy of sickle cell disease treatment consists of hydroxyurea and a few branded drugs. Hydroxyurea is considered the first line of the disease treatment and is recommended by many healthcare professionals. However, the lack of availability of treatment options in countries and regions across the globe stands out as a restraining factor.

• According to the American Society of Hematology, in the Sub-Saharan Africa region, around 300,000 babies are born with SCD yearly. Despite the inclusion of hydroxyurea in the WHO Model List of Essential Medicines for Children, it remains unavailable in the region. Also, the drug is considered to be too expensive in Africa.

Moreover, blood transfusions for SCD management are also dependent on donor availability. Even if the donors are available, there are chances of inappropriate screening, leading to increased blood transfusion-transmitted infections.

Public and private funding in African countries is insufficient to improve the healthcare facilities and care required by the patients suffering from the disease.

Furthermore, combined with the lack of awareness about the disease among the population in emerging economies, it can limit the global market growth during the forecast period.

SEGMENTATION

By Treatment Modality Analysis 

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Blood Transfusion Segment to Lead Backed by its Efficiency in Treatment Modality for Stroke Management

Based on treatment modality, the market is segmented into bone marrow transplant, blood transfusion, and pharmacotherapy. The pharmacotherapy segment is further bifurcated into hydroxyurea and branded products. The blood transfusion segment generated the highest revenue in 2021 and is expected to grow at a stagnant CAGR during the forecast period. Blood transfusion is the most efficient treatment method to manage strokes, which is one of the severe symptoms of SCD. The increasing number of episodes of strokes among SCD patients has increased the demand for blood transfusion treatment. This factor is responsible for the segment’s dominance.

Furthermore, the pharmacotherapy segment is expected to grow at the fastest CAGR during the forecast period.  The high growth rate of the segment is due to the introduction of new branded pharmacotherapy drugs and increasing government initiatives on early launches of these drugs. Moreover, limited treatment options currently available in the market are also responsible for the segment’s growth in the forecast period. The government supports research activities for sickle cell disease treatment through funding and designations such as orphan drugs, fast track, and priority review, among others. These factors are expected to accelerate the introduction of new branded pharmacotherapy drugs into the market and provide significant momentum to the pharmacotherapy segment.

• For instance, in September 2018, National Heart, Lung, and Blood Institute (NHLBI) launched the Cure Sickle Cell Initiative. The Cure Sickle Cell Initiative actively engages in the SCD community and explores the scientific questions needed to safely and effectively advance potential therapies.

By End-user Analysis

Hospitals Segment to Dominate Attributable to Rising Prevalence of Patients Visiting Hospitals

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

The hospitals segment accounted for a significant global sickle cell disease treatment market share in 2021. The large share of the segment is attributed to the increasing prevalence of SCD disease and the rising number of hospital admissions for sickle cell disease treatment.

• According to the WHO, around 20-25 million people are living with the disease across the globe. Moreover, the number of patients is projected to increase by 30% by 2050, thereby facilitating the hospitals segment’s growth.

The specialty clinics segment is estimated to grow at a significant CAGR during the forecast period. This segment’s growth is attributable to the increasing number of specialty clinics providing care and sickle cell disease treatment.

REGIONAL INSIGHTS

The U.S. market generated a revenue of USD 1.05 billion in 2021. The dominance of the market in the country is attributed to better access to SCD treatment, potential pipeline candidates, strong government support, and rising collaborations to improve the treatment of the disease.

• In December 2019, Global Blood Therapeutics, Inc. announced a research collaboration with Syros Pharmaceuticals Inc. to develop and commercialize novel therapies for SCD and beta-thalassemia.

The market in Europe accounted for a significant market share in 2021 and is projected to witness considerable growth during the forecast period. The market growth in Europe is attributed to the favorable reimbursement policies, the increasing prevalence of the disease, and increasing market players’ emphasis on the expansion of their offerings in the region.

• According to the data from the European Medicines Agency (EMA), in 2019, approximately 1 in 10,000 people lived with the disease in the European Economic Area (EEA).

The rest of the world is expected to expand at a significant CAGR in the projected years. The high market growth in the region is attributed to the highest prevalence of SCD in Africa, the Middle East, South America, and the Mediterranean regions and increasing disposable incomes. Moreover, the increasing awareness about SCD and the strong pipeline of branded drugs are also expected to fuel the market growth in the region.

KEY INDUSTRY PLAYERS

Companies with Strong Sickle Cell Disease Treatment Sales to Dominate the Competition 

Global Blood Therapeutics Inc. (Pfizer Inc.), Novartis AG, and Emmaus Medical, Inc. are the prominent players in the market and captured a considerable global market share in 2021.

Global Blood Therapeutics Inc. (Pfizer Inc.) dominated the market in 2021 and accounted for a significant market share due to the company’s strong sales from Oxbryta for SCD treatment.

• For instance, Oxbryta generated a revenue of USD 195.0 million in 2021, experiencing an increase of 57.5% in its sales from the prior year.

Similarly, Novartis AG held a considerable share of the market in 2021 due to increasing focus on partnerships with several government organizations. This partnership may allow the company to address the unmet needs of the patient population. Moreover, the company’s emphasis on the expansion of the availability of its product globally is also responsible for the company’s significant position in the market.

• In June 2020, Novartis AG announced the expansion of the Africa Sickle Cell Disease program to East Africa with the signature of two new memoranda of understanding with the Ministries of Health of Uganda and Tanzania. The program aims to improve and extend the lives of people with SCD in sub-Saharan Africa.

Furthermore, blood transfusion also generated significant revenue accounting for 51.4% of the market value in 2021 due to increasing SCD complications, such as stroke, which can be prevented through blood transfusion therapy.

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. released details of its U.S. commercial infrastructure to support rapid access to ZYNTEGLO, including an innovative, outcomes-based contract offering and a comprehensive patient support program.


• November 2021 – Emmaus Life Sciences, Inc. announced its partnership with UpScript IP Holdings, LLC. (UpScript), to offer telehealth solutions to the patients, expanding access to Endari.


• September 2020 – Global Blood Therapeutics, Inc. inked an exclusive agreement with Biopharma-MEA to distribute Oxbryta in Bahrain, Kuwait, Oman, Qatar, Saudi Arabia, and the United Arab Emirates.


• December 2019 - Emmaus Medical, Inc. received the initial purchase order from Taiba Healthcare’s early access program for Endari in Oman. Taiba is a leading healthcare marketing, distribution, and retail pharmacy group focused on GCC.


• November 2019–Addmedica appointed Master Specialty Pharma as the exclusive distributor of Siklos in the U.K.

REPORT COVERAGE 

An Infographic Representation of Sickle Cell Disease Treatment Market

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The research report provides a detailed competitive landscape. It includes the prevalence of sickle cell disease and key industry developments such as partnerships, mergers, & acquisitions. Additionally, it focuses on key points such as new product launches in the market. Furthermore, the report covers regional analysis of different segments and company profiles of key sickle cell disease treatment players, including business overview, financial data, and SWOT analysis for each company. Moreover, the report includes market trends and the impact of COVID-19 on the market. The report consists of quantitative and qualitative insights contributing to the market's growth.

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