Lumacaftor

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]

Lumacaftor
Clinical data
Other namesVX-809
License data
Routes of
administration		By mouth
ATC code
Legal status
Legal status
Identifiers
IUPAC name
  • 3-{6-{[1-(2,2-Difluoro-1,3-benzodioxol-5-yl)cyclopropanecarbonyl]amino}-3-methylpyridin-2-yl}benzoic acid
CAS Number
PubChem CID
IUPHAR/BPS
DrugBank
ChemSpider
UNII
KEGG
ChEBI
ChEMBL
CompTox Dashboard (EPA)
ECHA InfoCard100.241.800
Chemical and physical data
FormulaC24H18F2N2O5
Molar mass452.414 g·mol−1
3D model (JSmol)
SMILES
  • 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
  • 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
 NY (what is this?)  (verify)

See also
  • Ataluren, targeting premature stop codons

References
  1. 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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