McNamara JJ, et al. - In this open-label phase 3 study, authors evaluated the safety, pharmacokinetics, pharmacodynamics, and effectiveness of lumacaftor and ivacaftor in kids of age 2–5 years weighing at least 8 kg at enrolment. They observed cough (63%), vomiting (28%), pyrexia (28%), and rhinorrhoea (25%) as the most common adverse events. But serious adverse events like infective pulmonary exacerbation of cystic fibrosis, gastroenteritis viral, and constipation were reported in 4 kids. A decrease in mean sweat chloride concentrations by 31·7 mmol/L, improvement in biomarkers of pancreatic function (fecal elastase-1 concentrations increased and serum immunoreactive trypsinogen concentrations decreased), and incline in growth parameters were recorded at week 24. Overall, they concluded lumacaftor and ivacaftor safe and well tolerated for children with cystic fibrosis.