Cystic Fibrosis – Landscape & Forecast – Disease Landscape & Forecast
Cystic fibrosis (CF) is a genetic disease caused by any one of the more than 2,000 mutations identified in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CFTR mutations lead to aberrant chloride transport in a variety of tissues, most notably in the lungs and pancreas; as a result, patients can develop pancreatic damage, which affects their metabolism and nutrient absorption. These patients can also develop chronic respiratory problems, such as persistent lung infection, stemming from the accumulation of thick, viscous mucus in the lungs. Respiratory failure is the leading cause of death in CF.
Historically, therapeutic options for CF had been limited to symptomatic therapies. However, disease-modifying therapies (DMTs) have become a reality for a subset of CF patients with the market entry of Vertex’s Kalydeco (ivacaftor), Orkambi, and Symdeko, as well as the recent approval of Vertex’s Trikafta, a three-drug combination therapy. Pipeline agents are poised to compete in this lucrative market by seeking to improve the performance of marketed DMTs or expand DMTs’ reach in the CF population.
QUESTIONS ANSWERED
· How will the size of the CF population change through 2029? What percentage of the CF population receives drug treatment?
· How is CF currently managed in the United States and EU5? What are the most pressing unmet clinical needs according to CF experts?
· Which emerging therapies do CF experts consider most promising? If approved, how would emerging therapies influence the management of CF patients? What commercial impact will they have on the CF market?
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
Table of contents
- Cystic Fibrosis - Landscape & Forecast - Disease Landscape & Forecast
- Key Findings
- Cystic Fibrosis Key Findings - November 2021
- Commercial Outlook
- Key Findings
- Regional Sales of Key Therapies for Cystic Fibrosis: 2020 and 2030
- Cystic Fibrosis SWOT Analysis
- Drivers and Constraints
- What Factors Are Driving Sales in Cystic Fibrosis?
- What Factors Are Constraining Sales in Cystic Fibrosis?
- Drug-Specific Trends
- Kalydeco
- Orkambi
- Symdeko / Symkevi
- Trikafta / Kaftrio
- Key Findings
- Forecast
- Sales of Disease-Modifying Therapies in Cystic Fibrosis
- Etiology and Pathophysiology
- Key Findings
- Etiology
- Genotype-Phenotype Correlation
- Classifications of CFTR Mutations
- Genotype-Phenotype Correlation in Cystic Fibrosis
- Pathophysiology
- Respiratory Pathophysiology
- Gastrointestinal, Hepatobiliary, and Pancreatic Pathophysiology
- Anatomy of the Lungs
- Pancreatic Insufficiency in Cystic Fibrosis
- Clinical Presentation and Symptomology of Cystic Fibrosis
- Symptoms and Complications of Cystic Fibrosis
- Common Symptoms and Complications Associated with Cystic Fibrosis
- Clinical Subtypes
- Key Pathways and Drug Targets
- CFTR Modulators for Cystic Fibrosis: Potentiators and Correctors
- Mechanism of Action of Potentiators and Correctors
- ENaC Modulators
- Key Inflammatory Pathways
- Key Drug Targets for Antibiotics Against Bacterial Infections
- Key Findings
- Epidemiology
- Key Findings
- Epidemiology Populations
- Disease Definition
- Methods
- Sources Used for Diagnosed Prevalent Cases of Cystic Fibrosis
- Number of Diagnosed Prevalent Cases of Cystic Fibrosis: 2020-2030
- Definition
- Methods
- Sources Used for Diagnosed Prevalent Cases of Cystic Fibrosis Stratified by CFTR Mutation
- Diagnosed Prevalent Cases of Cystic Fibrosis Stratified by CFTR Mutation
- Definition
- Methods
- Sources Used for Diagnosed Prevalent Cases of Cystic Fibrosis Stratified by MRSA Infection
- Diagnosed Prevalent Cases of Cystic Fibrosis Stratified by MRSA Infection
- Number of Diagnosed, Drug-Treated Prevalent Cases in the United States and Europe: 2020-2030
- Key Findings
- Current Treatment
- Key Findings
- Diagnosis
- Sweat Chloride Test for Diagnosis of Cystic Fibrosis
- Treatment Providers and Referral Patterns
- Expert Insight on Diagnosis, Treatment Providers, and Referral Patterns
- Treatment Goals
- Key Endpoints Used in Clinical Trials for Cystic Fibrosis
- Expert Insight on Clinical Endpoints
- Key Current Therapies
- Mechanism of Action of Key Current Drugs / Drug Classes Used for Cystic Fibrosis
- Current Treatments Used for Cystic Fibrosis
- Approved Indications and Label Expansions for Kalydeco
- Clinical Trial Outcomes for Kalydeco
- Advantages and Disadvantages of Kalydeco
- Key Results from Select Clinical Trials Investigating Kalydeco for the Treatment of Cystic Fibrosis
- Key Ongoing Clinical Trials of Kalydeco in the Treatment of Cystic Fibrosis
- Expert Insight on Kalydeco for Cystic Fibrosis
- Approved Indications and Label Expansions for Orkambi
- Clinical Trial Outcomes for Orkambi in Cystic Fibrosis
- Advantages and Disadvantages of Orkambi
- Key Results from Select Clinical Trials Investigating Orkambi for the Treatment of Cystic Fibrosis
- Ongoing Clinical Development for Orkambi
- Key Ongoing Clinical Trials of Orkambi in the Treatment of Cystic Fibrosis
- Expert Insight on Orkambi for Cystic Fibrosis
- Clinical Trial Outcomes for Symdeko / Symkevi
- Advantages and Disadvantages of Symdeko / Symkevi
- Key Results from Select Clinical Trials Investigating Symdeko / Symkevi for the Treatment of Cystic Fibrosis
- Key Ongoing Clinical Trials of Symdeko / Symkevi in the Treatment of Cystic Fibrosis
- Expert Insight on Symdeko / Symkevi for Cystic Fibrosis
- Clinical Trial Outcomes for Trikafta / Kaftrio
- Advantages and Disadvantages of Trikafta / Kaftrio
- Key Results from Select Clinical Trials Investigating Trikafta / Kaftrio for the Treatment of Cystic Fibrosis
- Key Ongoing Clinical Trials of Trikafta / Kaftrio in the Treatment of Cystic Fibrosis
- Expert Insight on Trikafta / Kaftrio for Cystic Fibrosis
- Antibiotics Used in the Treatment of Cystic Fibrosis
- Expert Insights on Antibiotics for Cystic Fibrosis
- Key Results from Select Clinical Trials Investigating Inhaled Tobramycin and Inhaled Aztreonam for the Treatment of Cystic Fibrosis
- Advantages and Disadvantages of Inhaled Tobramycin
- Key Results from Select Clinical Trials Investigating Inhaled Colistimethate Sodium for the Treatment of Cystic Fibrosis
- Advantages and Disadvantages of Inhaled Colistimethate Sodium
- Key Results from Select Clinical Trials Investigating Azithromycin for the Treatment of Cystic Fibrosis
- Advantages and Disadvantages of Azithromycin
- Key Results from Select Clinical Trials Investigating Inhaled Amikacin for the Treatment of Cystic Fibrosis
- Advantages and Disadvantages of Inhaled Amikacin
- Clinical Trial Outcomes for Key Mucolytic Therapies
- Advantages and Disadvantages of Mucolytics
- Key Results from Select Clinical Trials Investigating Mucolytics for the Treatment of Cystic Fibrosis
- Expert Insights on Mucolytics for Cystic Fibrosis
- Clinical Trial Outcomes for Key Pancreatic Enzyme Replacement Therapies
- Advantages and Disadvantages of Pancreatic Enzyme Replacement Therapies
- Medical Practice
- Factors Influencing Drug Selection in Cystic Fibrosis
- Cystic Fibrosis Treatment and Management
- Generalized Treatment Decision Tree for Cystic Fibrosis
- Key Findings
- Unmet Need Overview
- Current and Future Attainment of Unmet Needs in Cystic Fibrosis
- Top Unmet Needs in Cystic Fibrosis: Current and Future Attainment
- Expert Insight: Unmet Need in Cystic Fibrosis
- Emerging Therapies
- Key Findings
- Pipeline Trends in Cystic Fibrosis
- Key Emerging Therapies
- Key Therapies in Development for Cystic Fibrosis
- Estimated Launch Dates of Key Emerging Therapies for the Treatment of Cystic Fibrosis
- Key Results from Select Clinical Trials Investigating Yumanity's CFTR Therapies for the Treatment of Cystic Fibrosis
- Planned Clinical Trials of Proteostasis's CFTR Therapies in the Treatment of Cystic Fibrosis
- Analysis of the Clinical Development Program for Proteostasis's CFTR Therapies
- Key Results from Select Clinical Trials Investigating AbbVie / Galapagos's CFTR Therapies for the Treatment of Cystic Fibrosis
- Key Ongoing Clinical Trials of AbbVie / Galapagos's CFTR Therapies in the Treatment of Cystic Fibrosis
- Expert Insight: Disease-Modifying Combination Therapies
- Key Ongoing Clinical Trials of OligoG in the Treatment of Cystic Fibrosis
- Analysis of the Clinical Development Program for OligoG
- Expectations for Launch and Sales Opportunity of OligoG in Cystic Fibrosis
- Early-Phase Pipeline Analysis
- Select Disease-Modifying Therapies in Early-Phase Development for Cystic Fibrosis
- Select Mucolytics in Early-Phase Development for Cystic Fibrosis
- Select Antibiotics in Early-Phase Development for Cystic Fibrosis
- Select Anti-Inflammatory Agents in Early-Phase Development for Cystic Fibrosis
- Key Discontinuations and Failures in Cystic Fibrosis
- Patient Registries
- Patient Registries for Cystic Fibrosis
- Orphan Drug Designation
- Key Findings
- Access and Reimbursement Overview
- Region-Specific Reimbursement Practices
- Key Market Access Considerations in Cystic Fibrosis: United States
- General Reimbursement Environment: United States
- Key Market Access Considerations in Cystic Fibrosis: EU5
- General Reimbursement Environment: EU5
- Region-Specific Reimbursement Practices
- Appendix
- Bibliography
- Key Findings
Stephanie Niquita Phankon, M.B.B.S., M.P.H., is a senior epidemiologist at DRG, part of Clarivate. She trained as a community physician and has supervised and coordinated various governmental and nongovernmental public health projects. She holds an M.P.H. specializing in epidemiology from the Tata Institute of Social Sciences in Mumbai and a medical degree from Hubei University of Chinese Medicine in the People’s Republic of China.