Vertex Pharma Gets Complete Response Letter From FDA For Kalydeco - Quick Facts

(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) said it has received a Complete Response Letter from the U.S. Food and Drug Administration for its supplemental New Drug Application or sNDA for the use of Kalydeco (ivacaftor) in people with cystic fibrosis (CF), ages 2 and older who have one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator or CFTR gene.

The FDA determined that it cannot approve the application in its present form. Vertex plans to meet with the FDA to determine an appropriate path forward.

Vertex Executive Vice President and Chief Medical Officer, Jeffrey Chodakewitz said, "We are disappointed by this decision and look forward to discussing with the FDA the next steps to bring KALYDECO to people with CF who have these residual function mutations."

The sNDA is based on preclinical data for ivacaftor in residual function mutations, the established clinical profile of KALYDECO and on previously reported data from an exploratory Phase 2a study. In 19 of the 24 patients enrolled in this study, eight of the 23 mutations proposed in the sNDA were represented.

There are more than 1,500 people ages two and older with CF in the U.S. who have one of the 23 residual function mutations included in the sNDA.