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navocaftor

Development stage: Phase 2
Lead developer: Sionna Therapeutics
Modality: Small Molecules
Administration: Oral

Overview

Navocaftor is an investigational small molecule drug being developed for the treatment of cystic fibrosis. It acts as a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator. The drug is administered orally and has been studied in combination with other CFTR modulators such as galicaftor and ABBV compounds in clinical trials targeting adult patients with cystic fibrosis. Development has involved multiple companies including Galapagos NV and AbbVie; more recently, Sionna Therapeutics acquired rights to develop navocaftor from AbbVie.

Brand names: navocaftor
Other names: 2159103-66-7Navocaftor [INN]Navocaftor [USAN](5-(3-amino-5-((4-(trifluoromethoxy)phenyl)sulfonyl)pyridin-2-yl)-1,3,4-oxadiazol-2-yl)methanol

Targets

CFTR (Cystic fibrosis transmembrane conductance regulator)

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