Long-term benefits of early initiation of ivacaftor (IVA) in people with cystic fibrosis (CF): interim results from an ongoing registry-based study

Abstract

<b>Background:</b> Clinical studies show IVA is safe and effective in people with CF, including young children. <b>Aims and objectives:</b> To evaluate long-term safety and efficacy of IVA in children aged 2–5 y at treatment initiation from US and UK CF registries. <b>Methods:</b> IVA cohorts from US CF Foundation Patient Registry and UK CF Registry included children aged 2–5 y with CFTR gating mutations who initiated IVA Mar 18, 2015–Dec 31, 2016(US) or Jan 1, 2017–Dec 31, 2017(UK). Longitudinal trends in key outcomes were compared to a concurrent cohort of modulator-untreated children homozygous for <i>F508del-CFTR</i> and a historical cohort of modulator-naïve children with CFTR gating mutations. <b>Results:</b> IVA cohorts included 150(US) and 52(UK) children; as of Dec 31, 2020, &gt;90% remained on treatment (mean exposure 5.2[US] and 3.7[UK] y). US IVA cohort had reductions in pulmonary exacerbations and increases in weight (Figure); increases in other nutritional parameters and decreases in&nbsp;hospitalizations, pancreatic enzyme supplement use, and <i>P. aeruginosa</i> prevalence versus the untreated concurrent comparator and historical modulator-naïve cohorts were also seen. Results from UK and US cohorts were consistent. <b>Conclusions:</b> Interim results of this registry-based study support the long-term benefits of early IVA initiation on CF disease progression. <b>Sponsor:</b> Vertex Pharmaceuticals Incorporated.