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Three-Drug Regimen Treats Cystic Fibrosis in Younger Children

Last Updated: April 14, 2021.

WEDNESDAY, April 14, 2021 (HealthDay News) -- 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 to 11 years are consistent with the results reported for this combination in adolescents and adults, according to a study published online March 18 in the American Journal of Respiratory and Critical Care Medicine.

Edith T. Zemanick, M.D., from the University of Colorado Anschutz Medical Campus and Children's Hospital Colorado in Aurora, and colleagues examined the safety, pharmacokinetics, and efficacy of ELX/TEZ/IVA in children aged 6 to 11 years with F508del-minimal function or F508del-F508del genotypes in a 24-week, phase 3, open-label study. A total of 66 children received ELX/TEZ/IVA (50 percent of adult daily dose for those weighing <30 kg; full adult daily dose for those weighing ≥30 kg).

The researchers found that 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 forced expiratory volume in one second, Cystic Fibrosis Questionnaire-Revised respiratory domain score, lung clearance index2.5, and sweat chloride concentration through week 24; compared with the pretreatment baseline, there was an increase in body mass index-for-age z-score during the 24-week treatment period.

"The most exciting aspect of our findings is that this population of children had normal lung function at the start of the study and still had a significant improvement," a coauthor said in a statement.

Several authors disclosed financial ties to Vertex Pharmaceuticals, which manufactures ELX/TEZ/IVA and funded the study.

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