Sickle Cell Disease – Landscape & Forecast – Disease Landscape & Forecast
Sickle cell disease (SCD) is a rare genetic blood disorder characterized by polymerization of hemoglobin in red blood cells that distorts them into a sickle shape. This sickling leads to several complications, such as acute chest syndrome, anemia, and vaso-occlusive crisis (VOC), which is associated with pain. Despite the launch of novel therapies, hydroxyurea is the mainstay first-line treatment of SCD in combination with prophylactic penicillin, analgesics, and blood transfusions. The FDA’s approval of Emmaus Life Sciences’ Endari (L-glutamine), Global Blood Therapeutics’ Oxbryta (voxelotor), and Novartis’s Adakveo (crizanlizumab) has provided patients with additional disease management options. Allogenic hematopoietic stem cell transplantation has been the only cure, although Bluebird Bio’s emerging gene therapy, LentiGlobin, is expected to offer a possible cure for severe SCD. A high unmet need exists for therapies that can reduce or eliminate VOC in patients with the most severe HbSS and HbSβ0 and extend their life expectancy. Drug developers recognize this commercial opportunity and are focused on developing agents that target the VOC pain symptoms or the underlying genetic defect.
QUESTIONS ANSWERED
- How large is the diagnosed prevalent SCD population in the United States and EU5? How will the population change over the forecast period?
- What is the current treatment landscape, and how will it change in the next 10 years? How will recently approved therapies (voxelotor and crizanlizumab) and late-phase therapies (LentiGlobin, Forma Therapeutics’ FT 4202, Global Blood Therapeutics’ inclacumab, and Micelle BioPharma’s docosahexaenoic acid [Altemia]) affect SCD treatment?
- What sales / uptake could emerging therapies secure in the HbSS and HbSβ0 patient groups? How will new therapies be incorporated into medical practice?
PRODUCT DESCRIPTION
Niche & Rare Disease Landscape & Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.
RELEASE DATE
December 2021
GEOGRAPHIES
United States and EU5
PRIMARY RESEARCH
- Six country-specific interviews with thought-leading hematologists.
- Supported by survey data collected for this study.
EPIDEMIOLOGY
Diagnosed prevalent and drug-treatable cases of SCD by country, segmented by clinical subtype.
FORECAST
Drug-level sales and patient shares of key SCD therapies in 2030.
EMERGING THERAPIES
Phase III/PR/approved: 7 drugs; Phase II or I/II: >10 drugs.
Table of contents
- Sickle Cell Disease - Landscape & Forecast - Disease Landscape & Forecast
- COVID-19
- Key Findings
- Sickle Cell Disease - Key Findings
- Commercial Outlook
- Key Findings
- Positioning of Key Emerging Therapies for Sickle Cell Disease
- Regional Sales of Key Therapies to Treat Sickle Cell Disease: 2020 and 2030
- Sickle Cell Disease SWOT Analysis
- Drivers and Constraints
- What Factors Are Driving the Market for Sickle Cell Disease?
- What Factors Are Constraining the Market for Sickle Cell Disease?
- Selectin Inhibitors
- Hemoglobin Modifiers
- Gene Therapy
- Alternative Market Scenarios
- Alternative Scenarios for the Sickle Cell Disease Market Through 2030
- Key Findings
- Forecast
- Sales of Key Therapies for Sickle Cell Disease
- Etiology and Pathophysiology
- Disease Overview
- Normal Red Blood Cell vs. Sickle Cell
- Etiology
- Common Genotypic Disease Forms of Sickle Cell Disease
- Typical Laboratory Results for Common Genotypic Disease Forms in SCD
- Pathophysiology
- Clinical Features of Sickle Cell Disease
- Symptoms and Complications of Sickle Cell Disease
- Key Pathways and Drug Targets
- Drug Targets for Sickle Cell Disease
- Disease Overview
- Epidemiology Overview
- Introduction
- Key Findings
- Epidemiology Populations
- Disease Definition
- Methods
- Sources Used for Diagnosed Prevalence of SCD
- Diagnosed Prevalent Cases of Sickle Cell Disease in the Mature Markets: 2020-2030
- Drug-Treatable Cases of Sickle Cell Disease: 2020-2030
- Disease Definition
- Methods
- Sources Used for Diagnosed Prevalence of Sickle Cell Disease by Subtype
- Diagnosed Prevalent Cases of Sickle Cell Disease by Subtype: 2020-2030
- Introduction
- Current Treatment Overview
- Key Findings
- Diagnosis
- Treatment Providers and Referral Patterns
- Expert Insight on Diagnosis of Sickle Cell Disease
- Treatment Goals
- Key Current Therapies
- Overview
- Mechanism of Action of Key Current Drugs or Drug Classes Used for Sickle Cell Disease
- Current Treatments Used for Sickle Cell Disease
- Clinical Trial Outcomes for Voxelotor
- Advantages and Disadvantages of Voxelotor
- Key Results from Select Clinical Trials Investigating Voxelotor for the Treatment of Sickle Cell Disease
- Expert Insight: Voxelotor
- Clinical Trial Outcomes for Prophylaxis Penicillin
- Advantages and Disadvantages of Prophylactic Penicillin
- Expert Insight on Prophylactic Penicillin
- Clinical Trial Outcomes for L-Glutamine
- Advantages and Disadvantages of L-Glutamine
- Key Results from Select Clinical Trials Investigating L-Glutamine for the Treatment of Sickle Cell Disease
- Expert Insight: L-Glutamine
- Clinical Trial Outcomes for Hydroxyurea
- Advantages and Disadvantages of Hydroxyurea
- Key Results from Select Clinical Trials Investigating Hydroxyurea for the Treatment of Sickle Cell Disease
- Expert Insight on Hydroxyurea
- Expert Insight on Analgesics
- Clinical Trial Outcomes for Crizanlizumab
- Advantages and Disadvantages of Crizanlizumab
- Key Results from Select Clinical Trials Investigating Crizanlizumab for the Treatment of Sickle Cell Disease
- Expert Insight: Crizanlizumab
- Advantages and Disadvantages of Blood Transfusions
- Expert Insight on Blood Transfusions
- Expert Insight on Hematopoietic Stem Cell Transplantation
- Medical Practice
- Overview
- Generalized Treatment Decision Tree for Sickle Cell Disease
- Key Findings
- Unmet Need Overview
- Current and Future Attainment of Unmet Needs in Sickle Cell Disease
- Top Unmet Needs in Sickle Cell Disease: Current and Future Attainment
- Expert Insight on Sickle Cell Disease Unmet Needs
- Drug Pipeline
- Pipeline
- Regulatory Milestones
- Indication Comparison
- Emerging Therapies Overview
- Key Findings
- ASH and FDA Joint 2019 Recommendations to Guide Clinical Development of Sickle Cell Disease Therapies
- Key Endpoints for Patient-Related Outcomes in Sickle Cell Disease
- Key Endpoints for Pain (non-PRO) in Sickle Cell Disease
- Key Endpoints for Brain Outcomes in Sickle Cell Disease
- Key Endpoints for Cardiovascular and Renal Complications Associated with Sickle Cell Disease
- Key Biomarkers for Therapies with Curative Intent in Sickle Cell Disease
- Key Emerging Therapies
- Notable Developments Among Key Emerging Therapies for Sickle Cell Disease
- Key Therapies in Development for Sickle Cell Disease
- Estimated Launch Dates of Key Emerging Therapies for the Treatment of Sickle Cell Disease
- Inclacumab Profile
- Key Ongoing Clinical Trials of Inclacumab for the Treatment of Sickle Cell Disease
- Analysis of the Clinical Development Program for Inclacumab
- Expert Insight on Inclacumab
- Expectations for Launch and Sales Opportunity of Inclacumab in Sickle Cell Disease
- Etavopivat Profile
- Key Ongoing Clinical Trials of Etavopivat for the Treatment of Sickle Cell Disease
- Analysis of the Clinical Development Program for Etavopivat
- Expert Insight on Etavopivat
- Expectations for Launch and Sales Opportunity of Etavopivat in Sickle Cell Disease
- Altemia Profile
- Key Ongoing Clinical Trials of Altemia in the Treatment of Sickle Cell Disease
- Analysis of the Clinical Development Program for Altemia
- Expert Insight on Altemia
- Expectations for Launch and Sales Opportunity of Altemia in Sickle Cell Disease
- Zynteglo Profile
- Key Ongoing Clinical Trials of Zynteglo in the Treatment of Sickle Cell Disease
- Analysis of the Clinical Development Program for Zynteglo
- Expert Insight: Zynteglo
- Expectations for Launch and Sales Opportunity of Zynteglo in Sickle Cell Disease
- Early-Phase Pipeline Analysis
- Select Compounds in Early-Phase Development for Sickle Cell Disease
- Key Discontinuations in Sickle Cell Disease
- Patient Registries
- Patient Registries for Sickle Cell Disease
- Prominent Sickle Cell Disease Patient Organizations
- Orphan Drug Designation
- Key Findings
- Access and Reimbursement Overview
- Region-Specific Reimbursement Practices
- General Reimbursement Environment: United States
- General Reimbursement Environment: EU5
- Region-Specific Reimbursement Practices
- Appendix
- Brands, Marketers, and Generic Availability of Key Therapies for Sickle Cell Disease by Market
- Sickle Cell Disease Bibliography
- COVID-19
Preeti Attri, Ph.D., is an associate analyst on the Infectious, Niche, and Rare Diseases team at Clarivate. Previously, she was a business analyst in the biopharmaceutical industry. She has experience in market assessment, competitive intelligence, primary market research, pharma forecasting, analogue and indication assessment, disease portfolio optimization, and database research. She obtained her doctorate degree in cancer epigenetics from the National Institute of Immunology in New Delhi.
Sunali D. Goonesekera, S.M., is a senior epidemiologist at Clarivate. Previously, Ms. Goonesekera conducted epidemiological research on racial/ethnic disparities in metabolic diseases at the New England Research Institute and lead-authored two manuscripts. She has contributed to multiple publications in peer-reviewed journals in epidemiology and the biological sciences. She holds a master’s degree in epidemiology from the Harvard School of Public Health and a B.A. in biology (Honors) from Dartmouth College.