EPS3.05 GLPG2222 in subjects with cystic fibrosis homozygous for F508del: results from a phase II study (FLAMINGO)

ivacaftor, sweat chloride concentrations were reduced by 18 mmol/L.Further, NPD and ICM showed partial rescue of CFTR function in nasal and rectal epithelia to levels of 10% and 18% of normal, respectively.All patients improved in at least one CFTR biomarker, but no correlations were found between CFTR biomarker responses and clinical outcomes.We could show that lumacaftor-ivacaftor results in partial rescue of Phe508del CFTR function to levels comparable to the lower range of CFTR activity found in patients with residual function mutations.Functional improvement was detected even in the absence of short-term improvement of FEV1% predicted and BMI.