195-21 | A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)

Overview

“A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-121 Combination Therapy in Subjects with Cystic Fibrosis Who are Heterozygous for F508del and a Minimal Function Mutation (F/MF)”

This study will evaluate the efficacy and safety of VX-121/TEZ/D-IVA in CF subjects who are heterozygous for F508del and a minimal function mutation. While ELX/TEZ/IVA is approved in certain regions for  subjects with F/MF genotypes, there remains a need for more highly effective CFTR modulators that can restore CFTR function (e.g., CFTR-mediated chloride transport) toward levels seen in carriers (who do not develop CF) in as many patients with CF as possible. The potential for benefit in these patients is supported by in vitro data and clinical data in F/MF subjects; in addition, VX-121/TEZ/D-IVA is generally safe and well tolerated.