CF is a life-limiting disease affecting around 70,000 individuals worldwide.
The market for CF therapies is estimated at $6 billion. The disease is characterized by the build-up of thick mucus in a number of organs including the linings of the lungs; creating an ideal environment for bacterial infection and colonization. Progressive lung disease is the most critical manifestation in CF with mortality in >90% of all CF patients caused by respiratory failure. CF patients generally undergo intensive, long-term antibiotic therapy, resulting in unavoidable resistance to many anti-infective drugs. The emergence of CF-specific multi-drug resistant pathogens is a source of major concern.
Exacerbations, intermittent episodes of acute worsening symptoms, often as a result of bacterial infection, are a key feature of CF. They are the principle cause of morbidity and mortality as a result of the decline in lung function. A typical adult patient would have multiple exacerbations annually.