Lumacaftor

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Lumacaftor
Systematic (IUPAC) name
3-{6-{[1-(2,2-difluoro-1,3-benzodioxol-5-yl)cyclopropanecarbonyl]amino}-3-methylpyridin-2-yl}benzoic acid
Clinical data
Legal status Investigational
Identifiers
CAS number 936727-05-8 N
ATC code None
PubChem CID 16678941
Chemical data
Formula C24H18F2N2O5 
Mol. mass 452.407 g/mol
 N (what is this?)  (verify)

Lumacaftor (USAN, codenamed VX-809) is an experimental drug for the treatment of cystic fibrosis, being developed by Vertex Pharmaceuticals. The drug is designed to be effective in patients that have the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR), the defective protein that causes the disease. F508del, meaning that the amino acid phenylalanine in position 508 is missing, is found in about 60% of cystic fibrosis patients in Europe,[1] and in about 90% of persons with some mutation in the CFTR gene.

Results from a Phase II clinical trial indicate that patients with the most common form of genetic mutation causing cystic fibrosis—homozygous F508del—had a 7.4% increase in lung function (FEV1) on a combination of lumacaftor and ivacaftor.[2]

See also

References

  1. ↑ Merk; Schubert-Zsilavecz. Pharmazeutische Zeitung (in German) 156 (37): 24–27. 
  2. ↑ Wilschanski, M. (2013). "Novel therapeutic approaches for cystic fibrosis". Discovery medicine 15 (81): 127–133. PMID 23449115. 


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