Shots:

• 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.

Ref: Vertex Pharmaceuticals | Image: Vertex