Therapy Areas: Hereditary Disorders
Two Phase 3 Studies of the Triple Combination of VX-659, Tezacaftor and Ivacaftor Met Primary Endpoint of Improvement in Lung Function (ppFEV1) in People with Cystic Fibrosis
28 November 2018 - - Treatment with the triple combination of the next-generation corrector VX-659, tezacaftor and ivacaftor resulted in statistically significant improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) in two Phase 3 studies in people with cystic fibrosis, US-based Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) said.

Data from a pre-specified interim analysis of the Phase 3 study in people with one F508delmutation and one minimal function mutation showed a mean absolute improvement in ppFEV1 of 14.0 percentage points from baseline at week 4 of treatment compared to placebo (p


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