Denufosol
Structural formula | ||||||||||||||||
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General | ||||||||||||||||
Non-proprietary name | Denufosol | |||||||||||||||
other names |
2′-deoxycytidine (5 ′) tetraphospho (5 ′) uridine ( IUPAC ) |
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Molecular formula | C 18 H 27 N 5 O 21 P 4 | |||||||||||||||
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Drug information | ||||||||||||||||
ATC code | ||||||||||||||||
Drug class |
P2Y2 receptor agonist |
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properties | ||||||||||||||||
Molar mass | 773.32 g · mol -1 | |||||||||||||||
safety instructions | ||||||||||||||||
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As far as possible and customary, SI units are used. Unless otherwise noted, the data given apply to standard conditions . |
The active ingredient Denufosol (-Tetrasodium) (INS37217), which has not yet been approved, is a P2Y2 receptor agonist for inhalation, which activates an alternative chloride channel in the epithelial cells of the lungs, so that the salt which is disturbed in the disease cystic fibrosis by the defective CFTR channel and water transport is at least partially corrected. This normalizes the dehydrated fluid film, increases the frequency of ciliary beats, and improves mucociliary clearance (the removal of mucus through the cilia ). Denufosol is said to have a positive effect on the course, especially in the early stages of lung involvement. Several phase 3 studies were carried out, some of which produced good results, some of which did not produce any statistically significant results. The development was funded by the American Cystic Fibrosis Foundation . Studies of the same substance for the treatment of retinal diseases were abandoned in 2006.
literature
- Kellermann et al .: In: Adv Drug Deliv Rev . 54./11., 2002, pp. 1463-1474.
Web links
Individual evidence
- ↑ This substance has either not yet been classified with regard to its hazardousness or a reliable and citable source has not yet been found.
- ↑ Inspire Pharmaceuticals, Inc., USA ( Memento of the original from October 23, 2016 in the Internet Archive ) Info: The archive link was inserted automatically and has not yet been checked. Please check the original and archive link according to the instructions and then remove this notice.
- ↑ D. Kellerman, A. Rossi Mospan, J. Engels, A. Schaberg, J. Gorden, L. Smiley: Denufosol: a review of studies with inhaled P2Y (2) agonists that led to phase 3. In: Pulmonary pharmacology & therapeutics. Volume 21, Number 4, August 2008, pp. 600-607, doi : 10.1016 / j.pupt.2007.12.003 , PMID 18276176 (review).
- ↑ TIGER-1 study in clinicaltrials.gov
- ↑ TIGER-2 study in clinicaltrials.gov
- ↑ FJ Accurso, RB Moss, RW Wilmott, RD Anbar, AE Schaberg, TA Durham, BW Ramsey: denufosol tetrasodium in patients with cystic fibrosis and normal to mildly impaired lung function. In: American journal of respiratory and critical care medicine. Volume 183, Number 5, March 2011, pp. 627-634, doi : 10.1164 / rccm.201008-1267OC , PMID 21169471 .
- ↑ Inspire Announces Results of Second Phase 3 Trial with Denufosol for Cystic Fibrosis. TIGER-2 Trial Did Not Meet Primary or Key Secondary Endpoints. In: Business Wire. January 3, 2011, accessed August 10, 2018 .