FDA panel backs Kalydeco for R117H CF patients

FDA's Pulmonary-Allergy Drugs Advisory Committee voted 13-2 that data support approval of Kalydeco ivacaftor from Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) to treat cystic fibrosis patients six and older with an R117H mutation in the CF transmembrane conductance regulator ( CFTR) gene. The panel also voted 9-6 that efficacy data provide substantial evidence of a clinically meaningful benefit, and 14-1 that safety data for the overall Kalydeco CF program are sufficient for approval. The PDUFA date is Dec. 30.

Kalydeco missed the primary endpoint of improving the mean absolute change in percent predicted FEV1 from baseline to week 24 but significantly reduced sweat chloride levels, a secondary endpoint, vs. placebo in the 69-patient Phase III KONDUCT trial in CF patients six and older with the R117H mutation. ...