The safety and efficacy of elexacaftor/tezacaftor/ ivacaftor (ELX/TEZ/IVA) for the treatment of cystic fibrosis with at least one F508del-CFTR allele in children aged 6-11 are consistent with the results reported for this combination in adolescents and adults, according to a study published in the American Journal of Respiratory and Critical Care Medicine. Researchers examined the safety, pharmacokinetics, and efficacy of ELX/TEZ/IVA in children aged 6-11 with F508del-minimal function or F508del-F508 del genotypes in a 24-week, phase III, openlabel study. A total of 66 children received ELX/TEZ/IVA (50% of adult daily dose for those weighing <30 kg; full adult daily dose for those weighing ≥30 kg). The safety and pharmacokinetic profiles were generally consistent with those seen in older patients. Cough, headache, and pyrexia were the most commonly reported adverse events (AEs); AEs were mostly mild or moderate in severity. ELX/TEZ/IVA treatment improved the percentage of predicted FEV1, Cystic Fibrosis Questionnaire-Revised respiratory domain score, lung clearance index 2.5, and sweat chloride concentration through week 24; compared with the pretreatment baseline, there was an increase in BMI-for-age z-score during the 24-week treatment period.
