THURSDAY, Feb. 7, 2019 (HealthDay News) — Lumacaftor/ivacaftor treatment is generally safe and well tolerated for children aged 2 to 5 years with cystic fibrosis (CF) homozygous for the F508del-CFTR mutation, according to a study published online Jan. 24 in The Lancet Respiratory Medicine.

John J. McNamara, M.D., from Children’s Minnesota in Minneapolis, and colleagues enrolled children aged 2 to 5 years weighing ≥8 kg at enrollment who had a confirmed diagnosis of CF homozygous for the F508del-CFTR mutation in an open-label phase 3 study. Patients received lumacaftor 100 mg/ivacaftor 125 mg (body weight <14 kg) or lumacaftor 150 mg/ivacaftor 188 mg (body weight ≥14 kg) orally for 15 days in part A (12 children) and for 24 weeks in part B (60 children).

The researchers found that safety and pharmacokinetics were consistent with the known safety profile. In part B, 98.3 percent of children had one or more treatment-emergent adverse events (AEs); most were of mild-to-moderate severity. The most common AEs were cough, vomiting, pyrexia, and rhinorrhea. Four children experienced serious AEs: infective pulmonary exacerbation of CF, viral gastroenteritis, and constipation. Because of elevated transaminases, 5 percent of the children discontinued treatment. At week 24, there was a 31.7 mmol/L decrease in mean sweat chloride concentrations, improvement in biomarkers of pancreatic function, and increases in growth parameters.

“These data highlight the potential of early intervention with lumacaftor/ivacaftor to improve clinically relevant outcomes,” the authors write.

Several authors are employees of and disclosed financial ties to Vertex Pharmaceuticals, which manufactures lumacaftor/ivacaftor and funded the study.

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