FDA rejects Vertex’s sNDA for Kalydeco to treat cystic fibrosis in people aged two years and older – Pharmaceutical Technology

he US Food and Drug Administration (FDA) has rejected Vertex Pharmaceuticals’ supplemental new drug application (sNDA) for Kalydeco (ivacaftor) designed to treat people with cystic fibrosis (CF) aged two and older, with one of 23 residual function mutations in the CF transmembrane conductance regulator (CFTR) gene.

The company received a complete response letter from the FDA, which stated that the agency cannot approve the sNDA in its current form.

In order to determine an appropriate path forward for the approval of the drug, the company plans to meet with the FDA.

Source: FDA rejects Vertex’s sNDA for Kalydeco to treat cystic fibrosis in people aged two years and older – Pharmaceutical Technology