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denufosol tetrasodium
- Development stage
- Phase 3
- Lead developer
- Inspire Medical Systems
- Modality
- Small Molecules
- Administration
- Inhalation
01
Overview
Denufosol tetrasodium is a selective P2Y2 receptor agonist designed to enhance mucosal hydration and mucociliary clearance in cystic fibrosis patients. It works by activating an alternative ion channel that compensates for the defective CFTR (cystic fibrosis transmembrane conductance regulator) channel. The drug has several pharmacological actions: increasing chloride secretion on epithelial cell surfaces, inhibiting epithelial sodium absorption, enhancing ciliary beat frequency, and stimulating mucin secretion. This mechanism of action is independent of CFTR genotype, making it potentially useful for all cystic fibrosis patients. Denufosol was administered via inhalation using a jet nebulizer three times daily in clinical trials.
- Other names
- 2'-Deoxycytidine(5')tetraphospho(5')uridine tetrasodium saltP(1)-(uridine 5')-P(4)-(2'-deoxycytidine 5')tetraphosphate, tetrasodium saltUridine 5'-(pentahydrogen tetraphosphate), P'''-5'-ester with 2'-deoxycytidine, tetrasodium salt
02
Targets
P2RY2 (P2Y Purinoceptor 2)
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