US Sickle Cell Disease - Market Insight, Epidemiology And Market Forecast - 2032

Key Highlights

• Sickle cell disease (SCD) is a genetic blood disorder characterized by abnormal hemoglobin, causing red blood cells to become sickle-shaped. This abnormal shape leads to complications, including chronic pain, organ damage, and increased infection susceptibility.
• The primary cause of SCD is a mutation in the HBB gene, which affects the production of hemoglobin. Individuals with SCD inherit two copies of the mutated gene, one from each parent, resulting in abnormal hemoglobin production. The abnormal red blood cells tend to become rigid and sticky, obstructing blood flow and causing episodes of intense pain called sickle cell crises.
• Hydroxyurea, NSAIDs, blood transfusions, chelating agents, nutritional supplements, and broad-spectrum antibiotics treat SCD. Moreover, a few therapies that the US FDA approves for the treatment of sickle cell disease include ENDARI (L-glutamine oral powder), ADAKVEO (crizanlizumab-tmca), and OXBRYTA (voxelotor).
• A few therapies that are being investigated for sickle cell disease include Exa-cel (Vertex Pharmaceuticals/CRISPR Therapeutics), Inclacumab (Global Blood Therapeutics/Pfizer), Etavopivat (Forma Therapeutics/Novo Nordisk) and others.
• The total prevalent cases of SCD trait in the US comprised ~4,284,000 in 2022 and are projected to increase by 2032.
• The market of SCD in the US is estimated to be around ~USD 700 million in 2022.
• SCD is a complex genetic blood disorder characterized by abnormal hemoglobin, leading to complications and reduced life expectancy. However, advancements in treatment approaches and ongoing research efforts offer hope for improved management and potential cures. Enhancing healthcare services, raising awareness, and supporting individuals with SCD are essential steps toward addressing the challenges associated with this disease.

Report Summary

• The report offers extensive knowledge regarding the epidemiology segments and predictions, presenting a deep understanding of the potential future growth in diagnosis rates, disease progression, and treatment guidelines. It provides comprehensive insights into these aspects, enabling a thorough assessment of the subject matter.
• Additionally, an all-inclusive account of the current management techniques and emerging therapies and the elaborative profiles of late-stage (Phase III and Phase II) and prominent therapies that would impact the current treatment landscape and result in an overall market shift has been provided in the report.
• The report also encompasses a comprehensive analysis of the SCD market, providing an in-depth examination of its historical and projected market size (2019-2032). It also includes the market share of therapies, detailed assumptions, and the underlying rationale for our methodology.
• The report includes qualitative insights that provide an edge while developing business strategies, by understanding trends, through SWOT analysis and expert insights/KOL views, including experts from various hospitals and prominent universities, patient journeys, and treatment preferences that help shape and drive the United States SCD market.

Various key players currently leading the treatment landscape of SCD include Novartis Pharmaceuticals, Global Blood Therapeutics/Pfizer, and Emmaus Life Sciences. Moreover, various key players are investigating their therapeutic candidates in the late phases of clinical development. Vertex Pharmaceuticals/CRISPR Therapeutics, Global Blood Therapeutics/Pfizer, Forma Therapeutics/Novo Nordisk, Bluebird Bio, etc. The details of the country-wise and therapy-wise market size have been provided below

• In 2022, the United States accounted for ~USD 700 million, expected to increase by the end of 2032.
• As per the analysis, OXBRYTA (voxelotor) generated the highest revenue of ~USD 310 million in 2022.

Sickle Cell Disease (SCD) Drug Chapters

The section dedicated to drugs in the SCD report provides an in-depth evaluation of marketed therapies and the late-stage pipeline drugs (Phase III and Phase II) related to SCD.

The drug chapters section provides valuable information on various aspects related to clinical trials of SCD, including specific details, such as the pharmacological mechanisms of the drugs involved, designations, approval status, patent information, and a comprehensive analysis of the pros and cons associated with each drug. Furthermore, it presents the most recent news updates and press releases on drugs targeting SCD.

Marketed Therapies

ENDARI: Emmaus Life Sciences

ENDARI (L-glutamine) is an oral-administered pharmaceutical grade L-glutamine (PGLG), an amino acid formulation to relieve pain, swelling, and other complications of sickle cell anemia in adults and children 5 years and older.

ADAKVEO (crizanlizumab): Novartis Pharmaceuticals

ADAKVEO (crizanlizumab) is a humanized anti-P selectin monoclonal antibody that binds a molecule called P-selectin on the surface of endothelial cells and platelets in the blood vessels, causing the blockade of P-selectin; it drives the vaso-occlusive process. The therapeutic blockade of P-selectin can prevent painful vaso-occlusion in small blood vessels and maintain blood flow.

OXBRYTA (voxelotor): Global Blood Therapeutics/Pfizer

OXBRYTA is an oral, once-a-day tablet developed by Global Blood Therapeutics. It is designed to block sickle hemoglobin polymerization, the root cause of sickle cell disease. This results in poor blood flow that affects the oxygenation of tissues, ultimately leading to painful vaso-occlusive crises. By increasing hemoglobin's affinity to oxygen, OXBRYTA halts this process.

Emerging Therapies

Exa-cel: Vertex Pharmaceuticals/CRISPR Therapeutics

Exa-cel (CTX001) is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patient's hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. Currently, it is being evaluated in Phase III for treating SCD patients.

Inclacumab: Global Blood Therapeutics/Pfizer

Inclacumab is a novel, fully human monoclonal antibody that selectively targets P-selectin. This protein mediates cell adhesion and is clinically validated to reduce pain due to VOCs in people with SCD. Preclinical results suggest that inclacumab can be a best-in-class option for reducing VOCs in people with SCD. The therapy is estimated to launch by 2025 and has a slow-medium uptake.

Etavopivat: Forma Therapeutics/Novo Nordisk

Etavopivat is an orally available, small-molecule allosteric activator of the selective red blood cell (RBC) isoform of pyruvate kinase (PK-R), with the potential to improve symptoms in sickle cell disease (SCD) patients. Currently being evaluated in Phase II/III clinical development, the therapy is estimated to launch by 2026 and attain its peak in 7 years.

Sickle Cell Anemia (SCD) Market Outlook

The treatment goals for SCD aim to relieve pain, prevent infections, and specifically manage complications. The goals are for symptom control and management of disease complications. The treatment starts with the recognition of pain in SCD patients. Recurrent episodes of acute, severe pain are the hallmark of SCD.

Initial management aims at providing rapid pain control. Many drug regimens have effectively treated acute pain in SCD. Pain management should follow the three-step “analgesic ladder” recommended by the World Health Organization for treating cancer-related pain. The choice of analgesic and dosage should be based on the severity of pain in the individual patient.

The treatment pattern currently consists of different approaches classified into pharmacologic and nonpharmacological therapies. The pharmacological therapies, including hydroxyurea, ENDARI (L-glutamine), ADAKVEO (crizanlizumab), and OXBRYTA (voxelotor), are approved. Pain management agents are segregated into opioids, nonsteroidal anti-inflammatory drugs (NSAIDs), acetaminophen, corticosteroids, etc. Acute vaso-occlusive crisis is generally managed using opioids and nonsteroidal anti-inflammatory drugs (NSAIDs). Further, nonpharmacological therapies include cognitive behavioral therapy, biofeedback, relaxation techniques, acupuncture, hypnosis, etc.

The current SCD treatment patterns allow emerging therapies to fill the voids, such as providing a complete cure, tolerable treatment side effects per se, and improved dosing and dosage forms. The patient burden will increase in the coming times, and approval of the emerging drug will expand the market. Therefore, the overall market size will increase, which could be a better investment therapy area.

Sickle Cell Disease (SCD) Understanding and Treatment

Sickle Cell Disease (SCD) Overview

Sickle Cell Disease (SCD) is a group of lifelong inherited conditions that affect hemoglobin. It is characterized as a chronic hemolytic disorder marked by the tendency of hemoglobin molecules within red blood cells to polymerize and deform the red cell into sickle (or crescent) shape (Hb S), resulting in characteristic vaso-occlusive events and accelerated hemolysis.

Sickle cell disease is inherited in an autosomal fashion, whether in the homozygous or double heterogeneous state. Sickle cell disease is called sickle cell anemia (SCA) when there is an inheritance in the homozygous state. Other known SCD genotypes include hemoglobin SC disease, sickle beta plus thalassemia, sickle beta zero thalassemia (which has similar severity with sickle cell anemia), hemoglobin SD Punjab disease, hemoglobin SO Arab disease, and others. Hemoglobin S (Hb S) differs from normal hemoglobin (Hb A) because of the substitution of valine for glutamic acid in the sixth position in the ß-globin gene.

Sickle cell anemia can lead to many complications, including stroke, acute chest syndrome, pulmonary hypertension, organ damage, splenic sequestration, leg ulcers, gallstones, priapism, deep vein thrombosis, and others.

Sickle Cell Disease (SCD) diagnosis

SCD diagnosis starts with a blood test that is analyzed for defective genes or hemoglobin cells. Various screening programs also help in the early diagnosis of the disease during the prenatal or infancy period.

Sickle Cell Disease (SCD) treatment

The primary objective of early intervention is to relieve pain. Numerous medication plans have successfully addressed sudden pain in sickle cell disease (SCD). For effective pain relief, it is advisable to adhere to the three-stage “analgesic ladder” endorsed by the World Health Organization, which is commonly used for managing cancer-related pain. The selection of the appropriate painkiller and its dosage needs to be determined by the intensity of pain experienced by each patient.

Oxygen therapy is often used routinely in managing vaso-occlusive crises, despite a lack of evidence supporting the effectiveness of these measures in all patients. Fluids should be administered in a quantity sufficient to correct existing deficits and replace ongoing losses to maintain a normal state. Moreover, antibiotics should be considered if there is evidence of infection.

Hydroxyurea is often used in patients with severe complications who can reliably follow the regimen. Hydroxyurea reduces the frequency of painful crises and the need for blood transfusions in patients with recurrent painful crises. Lastly, the only chance for a cure for Sickle Cell Disease is bone marrow or stem cell transplantation. The bone marrow nurtures stem cells, which are early cells that mature into red and white blood cells and platelets.

Sickle Cell Disease (SCD) Epidemiology

The Sickle Cell Disease (SCD) epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by Total Prevalent Cases of SCD Trait, Total Prevalent Cases of SCD, Diagnosed Cases of SCD, Age-specific Prevalence of SCD, and Type-specific Prevalence of SCD in the United States from 2019 to 2032.

• In 2022, the total prevalent cases of SCD trait in the US were ~4,284,000, projected to increase during the forecast period (2023-2032).
• As per the analysis, the total prevalent cases of SCD in the US were ~105,000 in 2022, projected to increase during the forecast period (2023-2032).
• In 2022, the diagnosed cases of SCD in the United States were estimated to be ~100,000 cases.
• The highest prevalent cases of SCD were estimated in the age group of 18-44 years, with ~33,000 cases, in 2022.
• Sickle cell anemia (hemoglobin S/S or hemoglobin S/ß0-thalassemia) type contributed to the highest number of SCD cases, ~58,000 cases. These cases are estimated to increase by 2032.

KOL Views

To stay abreast of the latest trends in the market, we conduct primary research by seeking the opinions of Key Opinion Leaders (KOLs) and Subject Matter Experts (SMEs) who work in the relevant field. This helps us fill any gaps in data and validate our secondary research.

The publisher has reached out to industry experts to gather insights on various aspects of SCD, including the evolving treatment landscape, patients' reliance on conventional therapies, their acceptance of therapy switching, drug uptake, and challenges related to accessibility. The experts we contacted included medical/scientific writers, professors, and researchers from prestigious universities in the United States.

The team of analysts connected with more than 10 KOLs in the United. We contacted institutions such as the Brooklyn Hospital Center, the Department of Pediatrics, Duke University, and others. By obtaining the opinions of these experts, we gained a better understanding of the current and emerging treatment patterns in the SCD market, which will assist our clients in analyzing the overall epidemiology and market scenario.

Qualitative Analysis

The publisher performs Qualitative and Market Intelligence analysis using various approaches, such as SWOT analysis and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, designation, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

In efficacy, the trial's primary and secondary outcome measures are evaluated; for instance, in trials for SCD, one of the most important primary endpoints was the proportion of subjects who have not experienced any severe vaso-occlusive crisis, frequency, and severity of collected adverse events (AEs). Based on these, the overall efficacy is evaluated.

Further, the therapies' safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the route of administration, order of entry and designation, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.

Market Access and Reimbursement

Because newly authorized drugs are often expensive, some patients escape receiving proper treatment or use off-label, less expensive prescriptions. Reimbursement plays a critical role in how innovative treatments can enter the market. The cost of the medicine, compared to the benefit it provides to patients who are being treated, sometimes determines whether or not it will be reimbursed. Regulatory status, target population size, the setting of treatment, unmet needs, the number of incremental benefit claims, and prices can all affect market access and reimbursement possibilities.

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Sickle Cell Disease (SCD) Report Insights

• Patient Population
• Therapeutic Approaches
• Sickle Cell Disease (SCD) Market Size and Trends
• Existing Market Opportunity

Sickle Cell Disease (SCD) Report Key Strengths

• Ten Year Forecast
• The United States Coverage
• Sickle Cell Disease (SCD) Epidemiology Segmentation
• Key Cross Competition

Sickle Cell Disease (SCD) Report Assessment

• Current Treatment Practices
• Unmet Needs
• Market Attractiveness
• Qualitative Analysis (SWOT, Conjoint Analysis)

Key Questions Answered

• Would there be any changes in the treatment approach that can cater to the needs of old patients, too, as over 50% of sickle cell disease patients belong to the older age group?
• Will there be any improvements in management recommendations for the pain in sickle cell disease?
• Would research and development advances pave the way for future tests and therapies for sickle cell disease?
• Would the diagnostic testing space experience a significant impact and lead to a positive shift in the treatment landscape of sickle cell disease?
• What kind of uptake will the new therapies witness in the coming years in sickle cell disease patients?