Vertex has announced positive results from the Phase 3 trial of Kalydeco (ivacaftor) in patients with cystic fibrosis (CF) aged 1 to 2 years who have 1 of 10 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Ivacaftor is currently approved for patients ≥2 years and is available as tablets and oral granules. The ongoing 2-part, open-label ARRIVAL study included 19 patients (with 18 completing) aged 1 to 2 years, who had 1 of the CFTR gene: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H mutations. 

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The primary endpoint of safety was met, as ivacaftor was generally well tolerated through 24 weeks of treatment and the majority of adverse events were mild or moderate in severity. These results were consistent with those observed in Phase 3 trials involving children aged 2 to 11 years.

Sweat chloride, a diagnostic characteristic of CF, was also considerably reduced with ivacaftor treatment. Median baseline sweat chloride levels were 104.1mmol/L. At 24 weeks of treatment there was a mean absolute decrease of -73.5mmol/L.

Additionally, levels of elastase in the stool significantly increased at 24 weeks of treatment. Low levels of elastase in the stool is suggestive of pancreatic insufficiency, a clinical manifestation of CF. The mean absolute fecal elastase at baseline was 182.2µg/g and there was an absolute improvement of 164.7µg/g at 24 weeks of the study.

“These results are an important step in our goal of treating children as early as possible to intervene in this progressive disease,” said Jeffrey Chodakewitz, MD, EVP and CMO at Vertex. 

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