FDA approved expanded use of TRIKAFTA for cystic fibrosis

U.S. Food and Drug Administration (FDA) approved expanded use of TRIKAFTA^® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis (CF) ages 6 through 11 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive to TRIKAFTA based on in vitro data. TRIKAFTA was previously approved by the FDA for use in people with cystic fibrosis 12 years and older with at least one copy of the F508del mutation or one copy of a mutation that is responsive in vitro. An additional dosage strength of TRIKAFTA tablets is now available (elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg and ivacaftor 75 mg) in connection with this approval.

According to a 24-week Phase 3 open-label, multicenter study which enrolled 66 children ages 6 through 11 years old with cystic fibrosis (CF) who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation to evaluate the safety, pharmacokinetics and efficacy of TRIKAFTA. The regimen was generally well-tolerated, and safety data were similar with those observed in previous studies of patients ages 12 years and older.

Cystic fibrosis is a progressive, genetic disease that causes persistent lung infections and limits the ability to breathe over time. More than 30,000 people are living with cystic fibrosis (more than 70,000 worldwide). Approximately 1,000 new cases of CF are diagnosed each year. More than 75 percent of people with CF are diagnosed by age 2.