FDA reviewers: some R117H CF patients get Kalydeco benefit

FDA reviewers said "at least some CF patients with a R117H mutation" in the cystic fibrosis transmembrane conductance regulator ( CFTR) gene benefit from Kalydeco ivacaftor from Vertex Pharmaceuticals Inc. (NASDAQ:VRTX). However, the reviewers questioned whether there are sufficient data to support approval of Kalydeco for CF patients six years of age and older with the mutation -- the indication for which Vertex is now seeking approval.

The comments came in briefing documents released ahead of next Tuesday's meeting of the Pulmonary-Allergy Drugs Advisory Committee to discuss an sNDA seeking to add the R117H mutation to Kalydeco's label. ...