Triple therapy safe, effective in cystic fibrosis with single Phe508del allele - Healio
Triple therapy safe, effective in cystic fibrosis with single Phe508del allele - Healio Triple therapy safe, effective in cystic fibrosis with single Phe508del allele - Healio Posted: 31 Oct 2019 01:21 PM PDT Combination therapy with elexacaftor/tezacaftor/ivacaftor improved lung function and the rate of pulmonary exacerbations with minimal adverse effects in patients aged 12 years and older with cystic fibrosis with Phe508del-minimal function genotypes who did not previously respond to cystic fibrosis transmembrane conductance regulator modulator therapy, according to a study published in The New England Journal of Medicine . In the phase 3, double-blind, randomized, placebo-controlled trial, the percentage of predicted FEV 1 was 13.8 points higher among patients treated with elexacaftor/tezacaftor/ivacaftor (Trikafta, Vertex Pharmaceuticals), as compared with placebo, at 4 weeks ( P < .001) and 14.3 points higher at 24 weeks ( P ...