Lumacaftor

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
IUPHAR/BPS 7481
ChemSpider 17611836
ChEBI CHEBI:90951 YesY
Chemical data
Formula C24H18F2N2O5
Molar mass 452.407 g/mol
 NYesY (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.

Lumacaftor acts on ΔF508 CFTR during protein folding and suppresses misfolding.[2]

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 mean increase of 7.4% in lung function (FEV1) on a combination of lumacaftor and ivacaftor.[3] However these results did not show a significant improvement in lung function.[3][4]

See also

References

  1. ↑ Merk; Schubert-Zsilavecz. Pharmazeutische Zeitung (in German) 156 (37): 24–27. Missing or empty |title= (help)
  2. ↑ Ren, H. Y.; Grove, D. E.; De La Rosa, O.; Houck, S. A.; Sopha, P.; van Goor, F.; Hoffman, B. J.; Cyr, D. M. (7 August 2013). "VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1". Molecular Biology of the Cell 24 (19): 3016–3024. doi:10.1091/mbc.E13-05-0240.
  3. 1 2 Wilschanski, M. (2013). "Novel therapeutic approaches for cystic fibrosis". Discovery Medicine 15 (81): 127–133. PMID 23449115.
  4. ↑ Boyle, M. P.; Bell, S. C.; Konstan, M. W.; McColley, S. A.; Rowe, S. M.; Rietschel, E; Huang, X; Waltz, D; Patel, N. R.; Rodman, D; Vx09-809-102 Study, Group (2014). "A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: A phase 2 randomised controlled trial". The Lancet Respiratory Medicine 2 (7): 527–38. doi:10.1016/S2213-2600(14)70132-8. PMID 24973281.
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