RARE Daily

FDA Approves Vertex’s Kalydeco to Treat Eligible Infants with Cystic Fibrosis Ages 1 Month and Older

May 4, 2023

Rare Daily Staff

The U.S. Food and Drug Administration approved Vertex Pharmaceuticals’ Kalydeco for use in children with cystic fibrosis ages 1 month to less than four months old who have at least one mutation in their CFTR gene that is responsive to Kalydeco based on clinical and/or in vitro assay data.

Kalydeco is already approved in the U.S. and EU for the treatment of CF in patients ages four months and older.

“Treating the underlying cause of cystic fibrosis as early as possible is important, and this approval, the first for a CFTR modulator in this age group, means families will now have a medicine for eligible infants,” said Carmen Bozic, executive vice president, Global Medicines Development and Medical Affairs, and chief medical officer, Vertex.

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 cystic fibrosis transmembrane conductance regulator (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.

The approval was supported by a cohort in the phase 3, 24-week, open-label study to evaluate the safety, pharmacokinetics, and pharmacodynamics of Kalydeco (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. In people with certain types of mutations in the CFTR gene, the CFTR protein at the cell surface does not function properly. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.

Photo: Carmen Bozic, executive vice president, Global Medicines Development and Medical Affairs, and chief medical officer, Vertex

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