- The approval is based on P-III ARRIVAL study results assessing Kalydeco in 25 children with CF who have either of 10 mutations in CFTR gene (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H)
- P-III ARRIVAL results: mean baseline sweat chloride level (SCL) (104.1 mmol/L); @24wks. SCL (33.8 mmol/L); patient with paired sweat chloride @24 wks. mean absolute change (-73.5 mmol/L), presented at European Cystic Fibrosis Society (ECFS) Conference in Jun,2018
- Kalydeco (ivacaftor, 150mg) is a CFTR potentiator, approved to treat adults and pediatric patients with CF mutation age 6yrs and older and is approved by the US and EU for CFTR mutation in patients aged 12 to <24 mos.
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