CF causes repeat and persistent lung infections that result in frequent exacerbations, sudden severe flare-ups of symptoms, and hospitalizations. Patients endure multiple daily medications, taking an average of seven per day, frequent hospitalizations and, ultimately, selected patients have lung transplants.
The quality of life for CF patients is compromised as a result of spending significant time on self-care every day and frequent medical visits and hospitalizations. The US and European regulatory authorities consider CF to be a rare, or orphan, disease and provide incentives to encourage development of effective new treatments.
Median life expectancy for CF patients is 37 years
CF affects about 70,000 people worldwide and approximately 1,000 new cases are diagnosed each year
According to the Cystic Fibrosis Foundation, more than 30,000 people in the US and more than 70,000 people worldwide are living with CF and approximately 1,000 new cases of CF are diagnosed each year.
In clinical trials, ensifentrine has been shown to activate the Cystic Fibrosis Transmembrane Conductance Regulator, which is beneficial in reducing mucous viscosity and improving mucociliary clearance. This suggests ensifentrine has potential as a therapy for CF.