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Vertex receives complete response letter from U.S. FDA for Use of KALYDECO® (ivacaftor)

 

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Vertex Pharmaceuticals announced that it received a Complete Response Letter from the U.S. Food and Drug Administration (FDA) for its supplemental New Drug Application (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 (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.

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.

CF is caused by defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) proteins resulting from mutations in the CFTR gene. The defective or missing proteins result in poor flow of salt (chloride) and water into and out of the cell in a number of organs, including the lungs. Chloride transport is a marker of the function of the CFTR protein at the cell surface.

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