Cystic fibrosis (CF) is a genetic disease affecting chloride transport in a variety of tissues, most notably the lungs and pancreas, and is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CF is inherited in an autosomal recessive manner, and more than 1,900 different mutations in the CFTR gene have been identified. CF patients generally suffer from pancreatic damage, which affects metabolism and nutrient absorption and is associated with failure to thrive in infants. Chronic respiratory problems, such as persistent lung infection stemming from accumulation of thick, viscous mucus in the lungs, can result in respiratory failure–the leading cause of death in CF. Historically, therapeutic options for CF were limited to physical therapy to dislodge mucus build-up, inhaled antibiotics, and pancreatic replacement therapy. However, in 2012, Vertex’s Kalydeco, a small-molecule CFTR potentiator, reached the market. Interviewed experts are very excited about Kalydeco and other agents following Kalydeco that address the underlying cause of CF.

This report provides an overview of the CF market including a comprehensive analysis of patient populations, current therapies and medical practice, and opportunities for emerging therapies. The findings described in this report are derived from detailed interviews with expert U.S. and European specialists, secondary research, and best-in-class epidemiological analysis. This report provides deep insights into this complex and evolving clinical space and includes a detailed analysis of specific opportunities for current and emerging therapies, including experts’ views on which unmet needs have not yet been addressed by developers of new therapies.

Questions Answered in This Report:

  • CF is a rare genetic disease affecting only a small percentage of the population. What is the size of the U.S. and EU5 (France, Germany, Italy, Spain, and the United Kingdom) diagnosed prevalent population for CF? How will these populations change over the ten-year forecast period? What are the most common CFTR mutations in CF patients in the U.S. and EU5?

  • Kalydeco is the only current therapy that addresses the underlying cause of the disease. What are the new therapies addressing the underlying cause of the disease that will launch by 2023? How will these disease-modifying therapies be used by 2023? What percentage of CF populations will be eligible for these treatment options?

  • Ivacaftor/lumacaftor combination therapy is the first therapy addressing the underlying cause of the F508del CFTR mutation. What are the interviewed CF experts’ views on the Phase III TRAFFIC and TRANSPORT clinical studies that evaluated ivacaftor/lumacaftor combination therapy? What role do they anticipate for Vertex’s second-generation corrector VX-661?

  • We forecast six new therapies indicated for use in CF will reach the U.S. and five major European markets by 2023. Which treatments will provide additional symptom-relief options? What percentage of CF populations will be eligible for these treatment options? What will be the therapeutic landscape in 2023?

  • Unmet needs in CF are many and span a range of challenging issues. What are the most promising avenues of research and development? Which unmet needs do thought leaders expect to remain unaddressed by mid- to late-stage emerging therapies?


Market covered: United States, France, Germany, Italy, Spain, and the United Kingdom.

Primary research: Eight country-specific interviews with thought-leading CF specialists.

Epidemiology: Diagnosed prevalent cases of CF and top five most frequent CFTR mutations by market.

Emerging Therapies: Phase II: 9; Phase III/PR: 5; coverage of select preclinical and Phase I products.

Author(s): Jing Wu, M.S., M.B.A.
Seamus Levine-Wilkinson, Ph.D.
Catherine Vasilakis-Scaramozza, Ph.D., M.P.H.

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