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 the lungs and pancreas; as a result, CF patients generally suffer from pancreatic damage, which affects their metabolism and nutrient absorption. Chronic respiratory problems, such as persistent lung infection stemming from the accumulation of thick, viscous mucus in the lungs, can result in respiratory failure and is the leading cause of death in CF.

Historically, therapeutic options for CF were limited to symptomatic therapies. Recently, disease-modifying therapies (DMTs) have become a reality for a subset of CF patients with the market entry of Vertex’s franchise, including Kalydeco (ivacaftor), a small-molecule CFTR potentiator; Orkambi, a combination of Kalydeco and the CFTR corrector lumacaftor; and Symdeko, a combination of Kalydeco and a next-generation CFTR corrector (tezacaftor). Pipeline agents from Vertex and other companies (e.g., Galapagos/AbbVie) are poised to compete in this lucrative market by seeking to improve upon the performance of marketed DMTs or expand DMTs’ reach in the CF population.

Table of contents

  • Disease Landscape & Forecast
    • Commercial Outlook
      • Key Findings
      • 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
        • AeroVanc
      • Alternative Market Scenarios
    • Forecast
    • Etiology and Pathophysiology
      • Key Findings
      • Etiology
        • Genotype-Phenotype Correlation
      • Pathophysiology
        • Respiratory Pathophysiology
        • Gastrointestinal, Hepatobiliary, and Pancreatic Pathophysiology
      • Clinical Presentation and Symptomology of Cystic Fibrosis
        • Symptoms and Complications of Cystic Fibrosis
      • Key Pathways and Drug Targets
        • CFTR Modulators for Cystic Fibrosis: Potentiators and Correctors
        • ENaC modulators
        • Key Inflammatory Pathways
    • Epidemiology Overview
      • Introduction
        • Key Findings
      • Epidemiology Populations
        • Diagnosed Prevalent Cases of Cystic Fibrosis, 2018-2028
        • Diagnosed Prevalent Cases of Cystic Fibrosis Stratified by CFTR Mutation, 2018-2028
        • Diagnosed Prevalent Cases of Cystic Fibrosis Stratified by MRSA Infection, 2018-2028
        • Diagnosed Prevalent Cases of Cystic Fibrosis, Age Distribution, 2018
        • Drug-Treated Cases of Cystic Fibrosis
    • Current Treatment
      • Key Findings
      • Diagnosis
        • Treatment Providers and Referral Patterns
      • Treatment Goals
      • Key Current Therapies
        • Disease-Modifying Therapies
        • Mucolytics
        • Key Antibiotics Prescribed for Cystic Fibrosis
        • Pancreatic Enzyme Replacement Therapies for Cystic Fibrosis
      • Medical Practice
        • Region-Specific Treatment
    • Unmet Need Overview
      • Current and Future Attainment of Unmet Needs in Cystic Fibrosis
    • Emerging Therapies
      • Key Findings
      • Key Emerging Therapies
        • Disease-Modifying Therapies
        • Mucolytics
        • Antimicrobial Agents for Cystic Fibrosis
      • Early-Phase Pipeline Analysis
      • Key Discontinuations and Failures in Cystic Fibrosis
      • Patient Registries
      • Orphan Drug Designation
        • Orphan Drug Provisions: United States
        • Orphan Drug Provisions: Europe
    • Access and Reimbursement Overview
      • Region-Specific Reimbursement Practices
        • United States
        • EU5
      • Looking for More?
    • Methodology
      • Bottom-Up Forecasting Overview
        • Patient Populations
        • Drug-Specific Assumptions
      • Bottom-Up Forecast Assumptions
        • General Sources of Data
        • General Statements About Pricing
        • Dosing, Days of Therapy, and Compliance
        • Generic Erosion
        • Out-Year Forecasting
        • Emerging Therapy Prices
      • Primary Market Research
        • Experts Interviewed
    • Appendix
      • Bibliography

Author(s): Pallavi Rajput, Ph.D; Stephanie Niquita

Pallavi Rajput joined Decision Resources Group as an associate analyst in 2019. Her focus is on the disease landscape and forecast of infectious diseases, niche and rare diseases. She holds a in molecular biology and virology from the International Centre for Genetic Engineering and Biotechnology, New Delhi, India. Prior to joining Decision Resources Group, Pallavi was a postdoctoral fellow at the University of California- Davis, where her research focused on the role of DNA repair in cancer development.

Stephanie Niquita works as an associate epidemiologist at Decision Resources Group. Stephanie holds a masters in public health specializing in epidemiology from TISS, Mumbai and a medical degree from Hubei University of Chinese Medicine, People’s Republic of China. She has been trained as a community physician and has also supervised and coordinated various governmental and non-governmental public health projects.

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