Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinus, sweat gland, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least 1 F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.1
- Over 10,500 people in the UK are living with CF2 – the second highest number of CF patients in the world.3
- Currently, there is no cure for CF and for people born in the UK with CF today, they are statistically likely to die before they are 48.4
- The daily impact of treatment is significant. It can take four or more hours involving nebulisers, physiotherapy and up to 70 tablets a day.5
- CF accounts for 9,500 hospital admissions and over 100,000 hospital bed days a year. A third of these are used by children under 15.6
At Vertex, our cystic fibrosis programme is united around a singular passion: to bring new medicines to all people living with this rare, life-shortening disease.
Our programme began as a small research project in 1998. Today, Vertex has the most investigational CF compounds, and is focused on developing medicines for all people with CF.
- 1CF Trust. UK CF Registry at-a-glance report 2018 cysticfibrosis.org.uk/the-work-we-do/uk-cf-registry/reporting-and-resources/at-a-glance-report-2018. Last accessed: November 2020.
- 2CF Trust. FAQs. Available at: cysticfibrosis.org.uk/what-is-cystic-fibrosis/faqs. Last accessed: November 2020.
- 3Lopes-Pacheco M. CFTR Modulators: Shedding Light on Precision Medicine for Cystic Fibrosis. Front Pharmacol. 2016;7:275.
- 4CF Trust. UK CF Registry at-a-glance report 2018 cysticfibrosis.org.uk/the-work-we-do/uk-cf-registry/reporting-and-resources/at-a-glance-report-2018. Last accessed: November 2020.
- 5CF Trust. Transition and adherence. Available at: cysticfibrosis.org.uk/the-work-we-do/research/research-areas/transition-and-adherence. Last accessed: November 2020.
- 6BLF CF Statistics. Available at: statistics.blf.org.uk/cystic-fibrosis. Last accessed: November 2020