Lumacaftor

Cystic fibrosis drug
  • EU EMA: by INN
Routes of
administrationBy mouthATC code
  • R07AX30 (WHO) (combination with ivacaftor)
Legal statusLegal status
  • US: ℞-only available only as Lumacaftor/ivacaftor
Identifiers
  • 3-{6-{[1-(2,2-Difluoro-1,3-benzodioxol-5-yl)cyclopropanecarbonyl]amino}-3-methylpyridin-2-yl}benzoic acid
CAS Number
  • 936727-05-8 ☒N
PubChem CID
  • 16678941
IUPHAR/BPS
  • 7481
DrugBank
  • DB09280 checkY
ChemSpider
  • 17611836
UNII
  • EGP8L81APK
KEGG
  • D10134
ChEBI
  • CHEBI:90951 checkY
ChEMBL
  • ChEMBL2103870
CompTox Dashboard (EPA)
  • DTXSID30239523 Edit this at Wikidata
ECHA InfoCard100.241.800 Edit this at WikidataChemical and physical dataFormulaC24H18F2N2O5Molar mass452.414 g·mol−13D model (JSmol)
  • Interactive image
  • CC1=C(N=C(C=C1)NC(=O)C2(CC2)C3=CC4=C(C=C3)OC(O4)(F)F)C5=CC(=CC=C5)C(=O)O
  • InChI=1S/C24H18F2N2O5/c1-13-5-8-19(27-20(13)14-3-2-4-15(11-14)21(29)30)28-22(31)23(9-10-23)16-6-7-17-18(12-16)33-24(25,26)32-17h2-8,11-12H,9-10H2,1H3,(H,29,30)(H,27,28,31) ☒N
  • Key:UFSKUSARDNFIRC-UHFFFAOYSA-N ☒N
 ☒NcheckY (what is this?)  (verify)

Lumacaftor (VX-809) is a pharmaceutical drug that acts as a chaperone during protein folding and increases the number of CFTR proteins that are trafficked to the cell surface.[1] It is available in a single pill with ivacaftor; the combination, lumacaftor/ivacaftor (brand name Orkambi), is used to treat people with cystic fibrosis who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the defective protein that causes the disease.[2] It was developed by Vertex Pharmaceuticals and the combination was approved by the FDA in 2015.[3] As of 2015, lumacaftor had no medical use on its own.[1]

See also

  • Ataluren, targeting premature stop codons

References

  1. ^ a b Kuk K, Taylor-Cousar JL (December 2015). "Lumacaftor and ivacaftor in the management of patients with cystic fibrosis: current evidence and future prospects". Therapeutic Advances in Respiratory Disease. 9 (6): 313–26. doi:10.1177/1753465815601934. PMID 26416827.
  2. ^ Lumacaftor label. Last updated July 2015. Check index page here for label updates
  3. ^ "Orkambi (lumacaftor and ivacaftor)". CenterWatch. Retrieved 24 March 2016.
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