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 painful vaso-occlusive crisis (VOC). Despite the launch of novel therapies, hydroxyurea is the mainstay first-line treatment for 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) gives patients additional treatment options. Allogenic hematopoietic stem cell transplantation is the only available curative therapy, although Bluebird Bio’s emerging gene therapy lovo-cel and Vertex / CRISPR Therapeutics’ gene-edited cell therapy exa-cel have the potential to cure severe SCD. Therapies that can reduce or eliminate VOCs in the most severely ill HbSS and HbSβ0 patients and extend life expectancy are strongly needed. Recognizing the commercial opportunity, drug developers are focused on developing agents that target VOC pain and/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 the launches of recently approved therapies (voxelotor and crizanlizumab) and late-phase therapies (lovo-cel, exa-cel, Forma Therapeutics’ etavopivat, Global Blood Therapeutics’ inclacumab, and Agios’s mitapivat) 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?
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
- Diagnosed prevalent cases of sickle cell disease segmented by clinical subtype.
FORECAST
Drug-level sales and patient share of key sickle cell disease therapies through 2031.
EMERGING THERAPIES
Phase III / PR / approved: 8 drugs; Phase II and Phase I/II: > 10 drugs.
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.
SOLUTION ENHANCEMENT
Niche & Rare Disease Landscape & Forecast introduces a new Drug Pipeline chapter with real-time, global pipeline intelligence content directly from Cortellis. This chapter is updated daily and features interactive figures that can be easily downloaded for detailed analysis or presentations.
Table of contents
- Sickle Cell Disease - Landscape & Forecast - Disease Landscape & Forecast
- Key findings
- Sickle cell disease - key findings
- Commercial outlook
- Key findings
- Positioning of key therapies for sickle cell disease
- Regional sales of key therapies to treat sickle cell disease: 2021 and 2031
- 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
- Sickle hemoglobin polymerization inhibitors
- Gene therapies
- Alternative market scenario
- Alternative scenario for the sickle cell disease market through 2031
- Key findings
- Forecast
- Sales of key therapies for sickle cell disease
- Etiology and pathophysiology
- Disease overview
- Healthy 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
- Introduction
- Key findings
- Epidemiology populations
- Disease definition
- Methods
- Sources used for diagnosed prevalence of sickle cell disease
- Diagnosed prevalent cases of sickle cell disease: 2021-2031
- Drug-treatable cases of sickle cell disease: 2021-2031
- Disease definition
- Methods
- Sources used for diagnosed prevalence of sickle cell disease by subtype
- Diagnosed prevalent cases of sickle cell disease by subtype: 2021-2031
- Introduction
- Current treatment
- Key findings
- Diagnosis
- Treatment providers and referral patterns
- Expert insight on diagnosing 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 unmet needs in the treatment of sickle cell disease
- Drug Pipeline
- Pipeline
- Regulatory Milestones
- Indication Comparison
- Emerging therapies
- 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
- Mitapivat profile
- Key ongoing clinical trials of mitapivat for the treatment of sickle cell disease
- Analysis of the clinical development program for mitapivat
- Expert insight on mitapivat
- Expectations for launch and sales opportunity of mitapivat in sickle cell disease
- Lovo-cel profile
- Key ongoing clinical trials of lovo-cel in the treatment of sickle cell disease
- Analysis of the clinical development program for lovo-cel
- Expert insight: Lovo-cel
- Expectations for launch and sales opportunity of lovo-cel in sickle cell disease
- Exa-cel profile
- Key ongoing clinical trials of exa-cel in the treatment of sickle cell disease
- Analysis of the clinical development program for exa-cel
- Expert insight: Exa-cel
- Expectations for launch and sales opportunity of exa-cel 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
- Key findings
- Access and reimbursement overview
- Region-specific reimbursement practices
- Key market access considerations in sickle cell disease: United States
- General reimbursement environment: United States
- Key market access considerations in sickle cell disease: EU5
- General reimbursement environment: EU5
- Region-specific reimbursement practices
- Appendix
- Abbreviations
- Sickle cell disease bibliography
- Key findings
Preeti Attri, M.Sc., Ph.D., is an associate analyst on the Infectious, Niche, and Rare Diseases team. Prior to joining Clarivate, she was a business analyst at Smart Analyst (India) Private Limited. She has experience in market assessment, competitive intelligence, primary market research, market forecasting, analogue and indication assessment, disease portfolio optimization, and database research. She received her Ph.D. in cancer epigenetics from the National Institute of Immunology in New Delhi.
Latashree Goswami, M.P.H., is an associate epidemiologist at Clarivate. Her areas of interest include oncology and autoimmune disorders. She holds an M.P.H. from the Indian Institute of Public Health, where she specialized in health economics, financing, and policy, and a B.D.S. from India’s ITS Dental College.