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' Dueling Phase III Programs Pay Off In NDA Submission Speed
Next-generation CFTR corrector elexacaftor aims to be first cystic fibrosis product with minimal function mutations claim after winning out over VX-659 thanks to tolerability advantage in dueling Phase III programs testing combination with the components of Vertex’ Symdeko.
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