First CFTR Modulator Approved for 6-Month Old Infants With Cystic Fibrosis

Study Safety Data Support Early Treatment of Underlying Cause of CF

The FDA has approved ivacaftor (KALYDECO®, Vertex Pharmaceuticals Incorporated) for the treatment of children aged 6 months to less than 12 months with cystic fibrosis (CF) who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to ivacaftor based on clinical and/or in vitro assay data.

The approval is based on data from ARRIVAL, a phase 3 open-label safety cohort of 11 children aged 6 months to less than 12 months who have CF with one of 10 mutations in the CFTR gene.

Most adverse events (AEs) in ARRIVAL were mild or moderate in severity, and no patient discontinued therapy as a result of AEs. The most common AEs were cough, nasal congestion, and rhinorrhea.

Cystic fibrosis affects approximately 30,000 people in the U.S. The disease is caused by a defective or missing CFTR protein as a result of mutations in the CFTR gene. Around 10 million people—approximately one in every 31 individuals—are carriers of the defective CF gene, but do not have the disease. 

The CFTR protein’s defective function, or its absence, results in the poor flow of salt and water into and out of the cell in various organs. This causes a buildup of thick, sticky mucus that can lead to chronic lung infections and progressive lung damage in many patients, and eventually lead to death. The median age of death in patients with CF is in the mid-to-late 20s.

Ivacaftor, a CFTR potentiator, is an oral medicine that keeps CFTR proteins at the cell surface open longer. This improves the movement of salt and water across the cell membrane, aiding hydration and clearing mucus from the airways.

The most common side effects of ivacaftor are headache; upper respiratory tract infection, including sore throat, nasal or sinus congestion, and runny nose; abdominal pain; diarrhea; rash; nausea; and dizziness.

Source: Vertex Pharmaceuticals Incorporated, April 30, 2019