FDA accepts supplemental New Drug Application for Kalydeco (ivacaftor) in patients aged 2 and older with one of 23 cystic fibrosis CTFR mutations- Vertex Pharmaceuticals

The FDA has accepted for review a supplemental New Drug Application (sNDA) for the use of Kalydeco (ivacaftor), from Vertex Pharmaceuticals, in people with cystic fibrosis (CF) ages 2 and older who have one of 23 residual function mutations. The FDA granted Vertex’s request for Priority Review of this sNDA, and set a target review date of 6 February 2016.

The sNDA was based on preclinical data for ivacaftor in the 23 residual function mutations, the established clinical profile of ivacaftor and on previously reported data from an exploratory Phase IIa study in 24 people with residual function mutations. In 19 of the 24 patients enrolled in this study, 8 of the 23 mutations proposed in the sNDA were represented.

Comment: ivacaftor is currently approved to treat people with CF ages 2 and older who have one of 10 mutations in the CFTR gene (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R or R117H). The 23 residual function mutations included in the sNDA are: 2789+5G->A, 3849+10kbC->T, 3272-26A->G, 711+3A->G, E56K, P67L, R74W, D110E, D110H, R117C, L206W, R347H, R352Q, A455E, D579G, E831X, S945L, S977F, F1052V, R1070W, F1074L, D1152H, and D1270N.