Sickle cell disease (SCD) is a rare genetic blood disorder characterized by polymerization of hemoglobin in red blood cells (RBCs) that distorts them into a sickle shape. This sickling leads to several complications, such as acute chest syndrome, anemia, and vaso occlusive crisis (VOC) associated with pain. Most patients are managed with a combination of hydroxyurea, prophylactic penicillin, analgesics, and blood transfusions. The recent FDA approval of Global Blood Therapeutics’ Oxbryta (voxelotor) and Novartis’s Adakveo (crizanlizumab) and their expected commercial launches starting in 2020 will provide patients with additional disease management options. Allogenic HSCT with an HLA-matched (most often sibling) donor is the only available curative therapy; however, pipeline gene therapies, such as Bluebird Bio’s Zynteglo (LentiGlobin), are expected to offer additional, potentially curative options to patients. The most severe HbSS and HbSβ0 patients suffer from painful episodes of VOC, which substantially impacts quality of life. A high unmet exists for therapies that can reduce or eliminate VOC and extend life expectancy. Drug developers recognize the commercial opportunity in SCD 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 ten years? How will the launches of voxelotor, crizanlizumab, LentiGlobin, and docosahexaenoic acid impact SCD treatment?
  • What pipeline molecules are promising? What sales / uptake could they secure in the HbSS and HbSβ0 patient groups? How will new therapies impact medical practice?

PRODUCT DESCRIPTION

Niche & Rare Disease Landscape & Forecast: Comprehensive market intelligence providing 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

April 2020

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 sickle cell disease by country, segmented by clinical subtypes.

FORECAST

Drug-level sales and patient shares of key sickle cell disease therapies in 2029.

EMERGING THERAPIES

Phase III/PR/approved: 5 drugs; Phase II: 4 drugs. Coverage of select preclinical and Phase I products.

Table of contents

  • Sickle Cell Disease - Landscape & Forecast - Disease Landscape & Forecast
    • COVID-19
      • Key Findings
        • SCD - 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: 2019 and 2029
          • 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 2029
            • Forecast
              • Sales of Key Therapies in Sickle Cell Disease
            • Etiology and Pathophysiology
              • Disease Overview
                • Normal Red Blood Cell vs. Sickle Cell
              • Etiology
                • Common Genotypic Disease Forms for 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
              • 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: 2019-2029
                  • Drug-Treatable Cases of Sickle Cell Disease, 2019-2029
                  • Disease Definition
                  • Methods
                  • Sources Used for Diagnosed Prevalence of SCD Subtypes
                  • Diagnosed Prevalent Cases of Sickle Cell Anemia (Hb SS Disease) or Hb β0 Thalassemia in the Mature Markets: 2019-2029
              • Current Treatment Overview
                • Key Findings
                  • Diagnosis
                    • Treatment Providers and Referral Patterns
                    • Expert Insight on Diagnosis of Sickle Cell Disease
                  • Treatment Goals
                    • ASH and FDA Joint 2019 Recommendations to Guide Clinical Development of Sickle Cell Disease Therapies
                    • Key Endpoints for Patient-Related Outcomes (PRO) 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 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 Prophylaxis Penicillin
                    • Advantages and Disadvantages of Prophylactic Penicillin
                    • Expert Insight on Prophylactic Penicillin
                    • 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
                    • 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
                    • Expert Insight on Analgesics
                    • Advantages and Disadvantages of Blood Transfusion
                    • Expert Insight on Blood Transfusion
                    • Expert Insight on Hematopoietic Stem Cell Transplantation
                  • Medical Practice
                    • Overview
                    • Country Sickle Cell Disease Treatment Guidelines
                    • Generalized Treatment Decision Tree for Sickle Cell Disease
                • 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
                • Emerging Therapies Overview
                  • Key Findings
                    • Pipeline Trends for 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
                    • Crizanlizumab Profile
                    • Key Ongoing Clinical Trials of Crizanlizumab for the Treatment of Sickle Cell Disease
                    • Analysis of the Clinical Development Program for Crizanlizumab
                    • Expert Insight on Crizanlizumab
                    • Expectations for Launch and Sales Opportunity of Crizanlizumab in Sickle Cell Disease
                    • Rivipansel Profile
                    • Analysis of the Clinical Development Program for Rivipansel
                    • Expectations for Launch and Sales Opportunity of Rivipansel in Sickle Cell Disease
                    • Voxelotor Profile
                    • Key Ongoing Clinical Trials of Voxelotor for the Treatment of Sickle Cell Disease
                    • Analysis of the Clinical Development Program for Voxelotor
                    • Expert Insight on Voxelotor
                    • Expectations for Launch and Sales Opportunity of Voxelotor in Sickle Cell Disease
                    • Altemia Profile
                    • Key Ongoing Clinical Trials of Apixaban 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
                    • Ticagrelor Profile
                    • Key Ongoing Clinical Trials of Ticagrelor in the Treatment of Sickle Cell Disease
                    • Analysis of the Clinical Development Program for Ticagrelor
                    • Expert Insight on Ticagrelor
                    • Expectations for Launch and Sales Opportunity of Ticagrelor 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
                  • Patient Registries
                    • Patient Registries for Sickle Cell Disease
                    • Prominent Sickle Cell Disease Patient Organizations
                  • Orphan Drug Designation
                  • Access and Reimbursement Overview
                    • Region-Specific Reimbursement Practices
                      • General Reimbursement Environment: United States
                      • General Reimbursement Environment: EU5
                    • Looking for More?
                  • Methodology
                    • Bottom-Up Forecasting Overview
                      • Patient Populations
                      • Drug-Specific Assumptions
                    • Bottom-Up Forecast Assumptions
                      • General Sources of Data
                      • Patients Eligible for Adakveo, Oxbryta and Zynteglo
                      • Adakveo (crizanlizumab) Patient Flow
                      • Oxbryta (voxelotor) Patient Flow
                      • Zynteglo (LentiGlobin) Patient Flow
                      • Drug-Treatable Rate in Sickle Cell Disease
                      • Drug-Treatable Rate Assumptions in Sickle Cell Disease
                      • Patient-Share Assumptions in Sickle Cell Disease
                      • General Statements About Pricing
                      • Sources for Drug Prices
                      • 2019 Exchange Rates
                      • Price per Treated Day: 2029
                      • Dosing, Days of Therapy, and Compliance
                      • Out-Year Forecasting
                      • Emerging Therapy Prices
                    • Primary Market Research
                      • Experts Interviewed
                  • Appendix
                    • Key Abbreviations Related to Sickle Cell Disease
                    • Brands, Marketers, and Generic Availability of Key Therapies for Sickle Cell Disease by Market
                    • Sickle Cell Disease Bibliography

                Author(s): Archita Kukreja; Sunali D. Goonesekera, SM

                Archita is a member of Decision Resources Group’s Infectious, Niche, and Rare Diseases (INRD) team. In this role she works on a range of antibacterial and antiviral indications as well as numerous niche and rare diseases.

                Archita holds a Masters in Science degree in biotechnology from Jamia Millia Islamia, New Delhi and has completed her MBA from Amity University, Noida.

                Sunali Goonesekera is an Associate Epidemiologist at Decision Resources Group.

                Sunali holds a Master’s degree in Epidemiology from the Harvard School of Public Health and a B.A. in Biology (Honors) from Dartmouth College. Prior to joining Decision Resources Group, Sunali conducted epidemiological research and lead authored two manuscripts on racial/ethnic disparities in metabolic diseases at the New England Research Institutes. She has contributed to multiple publications in peer-reviewed journals in epidemiology and in the biological sciences.