Paediatric data for elexacaftor/tezacaftor/ivacaftor in the treatment of cystic fibrosis (CF)

A global Phase III study in children aged 6-11 with CF and either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation found safety data were consistent with those observed in previous studies.

SPS commentary:

In the UK, this treatment (brand name Kaftrio) is licensed for use in a combination regimen with ivacaftor 150 mg tablets for the treatment of cystic fibrosis in patients aged 12 years and older who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or heterozygous for F508del in the CFTR gene with a minimal function (MF) mutation.

Based on the findings of this study, the company will submit a supplemental New Drug Application to the UD FDA, with global regulatory submissions to follow.

Biospace Inc.