Vertex Selects VX-445 For CF Triple Therapy Due To Safety/Tolerability

Vertex Pharmaceuticals Inc. has decided to use its next-generation CFTR corrector VX-445 as the third component of its triple therapy regimen for cystic fibrosis, choosing that compound over VX-659 mainly on considerations of safety and tolerability. The Boston-area firm now plans to file its triple regimen for US approval in the third quarter and for European approval in the fourth quarter.

Already the dominant player in cystic fibrosis due to therapies that address the disease’s underlying cause by increasing and/or improving the function of the cystic fibrosis transmembrane conductance (CFTR) regulator gene, Vertex said on 30 May that it will file a regimen combining the next-generation CFTR corrector VX-445 (elexacaftor) with the components of its double therapy Symdeko, the CFTR corrector tezacaftor and CFTR potentiator ivacaftor