Drug intelligence / Profile preview

galicaftor + navocaftor

Development stage
Unknown
Lead developer
Sionna Therapeutics
Modality
Small Molecules
Administration
Oral
01

Overview

Galicaftor + navocaftor is a novel combination therapy under development for the treatment of cystic fibrosis. Both agents are designed as **CFTR modulators**: galicaftor is a next-generation CFTR corrector, and navocaftor is a CFTR potentiator. This combination aims to improve the quantity and function of the CFTR protein at the cell surface for various cystic fibrosis mutations, potentially providing benefit to individuals not adequately addressed by existing modulators. It is being developed by Vertex Pharmaceuticals for patients with cystic fibrosis, particularly those with specific CFTR mutations who do not respond optimally to approved therapies. Clinical trials are ongoing to determine its safety and efficacy profile.

02

Targets

CFTR (Cystic fibrosis transmembrane conductance regulator)

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