Vertex Pharmaceuticals Inc.’s latest new drug application in cystic fibrosis could introduce therapy for the underlying cause of the disease to patients with mutations that result in minimal cystic fibrosis transmembrane conductance regulator (CFTR) gene function – “the largest remaining group of people with CF that still do not have an approved Vertex medicine,” the company noted.
Vertex announced the NDA submission for the novel next-generation CFTR corrector elexacaftor (VX-445) in combination with the CFTR corrector tezacaftor and the CFTR potentiator ivacaftor on 22 July, with a request for priority review