Vertex Pharmaceuticals announces FDA acceptance of triple combination therapy

Vertex Pharmaceuticals announced that the FDA has accepted its new drug application, or NDA, for investigational once-daily vanzacaftor/tezacaftor/deutivacaftor triple combination therapy for people living with cystic fibrosis, or CF, ages six years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator, or CFTR, gene responsive to the vanza triple. Vertex used a priority review voucher for this submission reducing the review time from 10 months to six months, resulting in a Prescription Drug User Fee Act, or PDUFA, target action date of January 2, 2025. Vertex also received validation of its marketing authorization application, or MAA, submission by the European Medicines Agency, or EMA, in the EU for patients ages six years and older. The company has also submitted in Canada, Australia, Switzerland and the U.K.