Positive phase III data from post-marketing study of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in people ages 12 and older with cystic fibrosis who have one copy of F508del mutation and one gating or residual function mutation

RCT (n=258) met primary endpoint of mean absolute within-group change in percent predicted forced expiratory volume in 1 second (ppFEV1) from baseline through 8 weeks of treatment, demonstrating statistically significant 3.7% (p<0.0001) improvement in ppFEV1 compared to baseline

SPS commentary:

Trikafta is already approved in US for use in people ages 12 years and older who have at least one copy of the F508del mutation, which includes the populations evaluated in this study. In June 2020, the European CHMP issued a positive opinion for the initial triple combination regimen application for people with CF ages 12 years and older with one F508del mutation and one minimal function mutation (F/MF) or two F508del mutations (F/F). Data announced from this study will be submitted to the European Medicines Agency to support a potential indication expansion of the EU label, once European Commission approval has been granted for the initial triple combination application.


Biospace Inc.