On May 3, 2023, Vertex announced the Food and Drug Administration (FDA) had approved KALYDECO (ivacaftor) for use in children with cystic fibrosis (CF) ages 1 month to less than four months old who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to KALYDECO based on clinical and/or in vitro assay data. KALYDECO was already approved in the U.S. and EU for the treatment of CF in patients ages four months and older.

The approval was supported by a cohort in the Phase 3, 24-week, open-label study to evaluate the safety, pharmacokinetics and pharmacodynamics of ivacaftor in subjects with CF who are less than 24 months of age and have an ivacaftor-responsive CFTR mutation. This cohort demonstrated a safety profile similar to that observed in older children and adults. KALYDECO^® was first approved in 2012 in the U.S. and is now available in more than 30 countries.

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 88,000 people globally. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene.

Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein 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, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the early 30s.