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 names	VX-809
License data	EU EMA: by INN
Routes of administration	By mouth
ATC code	R07AX30 (WHO) (combination with ivacaftor)
Legal status
Legal status	US: ℞-only available only as Lumacaftor/ivacaftor
Identifiers
IUPAC name 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 Y
ChemSpider	17611836
UNII	EGP8L81APK
KEGG	D10134
ChEBI	CHEBI:90951 Y
ChEMBL	ChEMBL2103870
CompTox Dashboard (EPA)	DTXSID30239523
ECHA InfoCard	100.241.800
Chemical and physical data
Formula	C24H18F2N2O5
Molar mass	452.414 g·mol−1
3D model (JSmol)	Interactive image
SMILES Cc1ccc(NC(=O)C2(c3ccc4c(c3)OC(F)(F)O4)CC2)nc1-c1cccc(C(=O)O)c1
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