Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) released results from its once-daily vanzacaftor/tezacaftor/deutivacaftor (vanza triple) program for cystic fibrosis (CF), a progressive, multi-organ disease caused by dysfunction of the CFTR protein.
The Phase 3 program included two trials: SKYLINE 102 and SKYLINE 103, evaluating vanzacaftor (20 mg)/tezacaftor (100 mg)/deutivacaftor (250 mg) in patients aged 12 and above, compared to Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor).
A third Phase 3 single-arm, 24?week, open-label study, RIDGELINE 105, evaluated vanza triple in children ages 6 to 11.
In SKYLINE 102 and SKYLINE 103, the primary endpoint of absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1, measurement of lung function) through week 24 was met and showed that treatment with vanza triple was non-inferior to treatment with Trikafta.