Drug intelligence / Profile preview

elexacaftor + tezacaftor + VX-561

Development stage
Unknown
Lead developer
Vertex
Modality
Small Molecules
Administration
Oral
01

Overview

**elexacaftor + tezacaftor + VX-561** is an investigational fixed-dose **triple combination therapy** developed for the treatment of cystic fibrosis. Elexacaftor and tezacaftor are CFTR (cystic fibrosis transmembrane conductance regulator) correctors that facilitate increased quantity of functioning CFTR protein at the cell surface, while VX-561 (deutivacaftor) is a deuterated form of ivacaftor that acts as a CFTR potentiator, enhancing the protein’s ability to function as a chloride channel. This once-daily combination is being evaluated in late-stage clinical trials for efficacy and safety in patients with cystic fibrosis, particularly aiming to improve upon current therapies (like Trikafta) in terms of efficacy and dosing convenience[1][2][6].

Other names
deutivacaftor + elexacaftor + tezacaftor
02

Targets

CFTR (Cystic fibrosis transmembrane conductance regulator)

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