Long-term data on CFTR modulators in children with cystic fibrosis

Cystic fibrosis (CF) is one of the most common congenital metabolic diseases in Europe. The treatment options for CF patients have expanded significantly in recent years. CFTR modulators improve the function of the defective CFTR protein and are therefore the first drugs with at least partial causal effect in CF. In an open label Phase IIIb extension study, the use of the combination therapy of ivacaftor, elexacaftor and tezacaftor was investigated in 6-11-year-old children over a period of 96 weeks.