Vertex Pharmaceuticals Incorporated Initiates Phase 3 Registration Program for VX-770, an Oral CFTR Potentiator Targeting the Defective Protein Responsible for Cystic Fibrosis

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the initiation of a Phase 3 registration program for VX-770, an investigational Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) potentiator that targets the defective CFTR protein that causes cystic fibrosis (CF). The VX-770 registration program will consist of three clinical trials, including a primary 48-week Phase 3 trial that is currently open to enrollment of patients aged 12 years and older who carry the G551D mutation on at least one allele. Two additional trials will evaluate VX-770 in patients aged 6 to 11 years with the G551D mutation on at least one allele and in patients homozygous for the F508del mutation, respectively.
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