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FDA approves Vertex's Kalydeco for tenth CF mutation

December 30, 2014 2:32 AM UTC

FDA approved an sNDA for Kalydeco ivacaftor from Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) to treat cystic fibrosis in patients aged six and older with an R117H mutation in the CF transmembrane conductance regulator ( CFTR) gene. The small molecule potentiator of CFTR was previously approved in the U.S. to treat CF in patients six and older with one of nine other mutations including G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D.

Approval for the R117H mutation was based on data from the 69-patient Phase III KONDUCT trial, in which Kalydeco led to a statistically significant improvement in the percent predicted FEV1 from baseline to week 24 vs. placebo in a subgroup of patients who were 18 years and older, but missed the endpoint in patients ages 6-11 years (p=0.03). ...