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VX-371 + ivacaftor
- Development stage
- Unknown
- Lead developer
- Vertex
- Modality
- Small Molecules
- Administration
- Inhalation (VX-371), Oral (ivacaftor)
01
Overview
VX-371 + ivacaftor is an investigational combination therapy under development for the treatment of **primary ciliary dyskinesia** (PCD). VX-371 (code name P-1037) is formulated as an inhalation solution and acts as a small molecule agent to restore ciliary function, potentially improving mucociliary clearance. **Ivacaftor** (VX-770) is a small molecule **CFTR potentiator** that increases the probability of the defective cystic fibrosis transmembrane conductance regulator (CFTR) protein channel opening, improving chloride ion transport across epithelial cells. The combination aims to enhance overall mucociliary function more effectively than either agent alone. Clinical studies have evaluated the safety and efficacy of inhaled VX-371 in combination with oral ivacaftor in subjects aged 12 years and older with diagnosed PCD[2].
- Other names
- Ivacaftor
02
Targets
CFTR (Cystic fibrosis transmembrane conductance regulator)ENaC
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