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 2,000 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. Vertex’s combination of Kalydeco and the CFTR corrector lumacaftor reached the market in 2015, significantly expanding the pool of patients eligible for treatment. Interviewed experts are very excited about Kalydeco, Orkambi, and emerging agents that address the underlying cause of CF.

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 the size of 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 and Orkambi are the only current therapies that address the underlying cause of the disease. What are new therapies addressing the underlying cause of the disease that will launch by 2024? How will these disease-modifying therapies be used by 2024?  What percentage of the CF population will be eligible for these treatment options?

  • Ivacaftor/lumacaftor (Orkambi) combination therapy is the first therapy addressing the underlying cause of the F508del CFTR mutation. What are 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 multiple new symptomatic therapies indicated for use in CF will reach the U.S. and five major European markets by 2024. 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 2024?

  • 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 will 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: 11; Phase III; 4; coverage of select preclinical and Phase I products.

Author(s): Michael Breen, Ph.D.
Catherine Vasilakis-Scaramozza, Ph.D., M.P.H.

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