Shots:

• The approval is based on the results of ongoing P-III ARRIVAL study assessing Kalydeco in patients who have either of 10 mutations in the CFTR gene (G551D- G178R- S549N- S549R- G551S- G1244E- S1251N- S1255P- G1349D or R117H)
• Results: mean sweat chloride level @ 24 wks. 33.8 mmol/L; mean absolute change -73.5 mmol/L; presented at European Cystic Fibrosis Society (ECFS) Conference in Jun- 2018
• Kalydeco is a CFTR potentiator- already approved in EU for CF aged 2 to 5 yrs. with either of 9 CFTR gene and >18 yrs. who have an R117H mutation in the CFTR gene

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