Vertex’s Kalydeco (ivacaftor) Receives FDA’s Expanded Label Approval for Cystic Fibrosis (CF) Aged 12 to < 24 Months

 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

Click here to read full press release/ article | Ref: Vertex | Image: Vertex