Vertex announced that the Food and Drug Administration (FDA) has granted Priority Review to the supplemental New Drug Application (sNDA) of Kalydeco (ivacaftor) for the use in patients with cystic fibrosis (CF) ages 2 and older who have one of 23 residual function mutations.
The sNDA submission was based on preclinical data for ivacaftor in the 23 residual function mutations, the established clinical profile of Kalydeco and on previously reported data from an exploratory Phase 2a study in 24 people with residual function mutations.
Kalydeco is currently approved for the treatment of CF in patients ages 2 and older who have one of 10 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or R117H. As with the 10 mutations Kalydeco is approved for, the 23 residual function mutations in the sNDA are known to have some CFTR protein at the cell surface and have shown in vitro increases in chloride transport in response to ivacaftor in cells expressing the CFTR form produced by each mutation, characteristics associated with clinical response to Kalydeco.
Kalydeco, a CFTR potentiator, is the first drug to treat the underlying cause of CF in people with specific mutations in the CFTR gene.
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