Lumacaftor

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 ;
PubChem CID	16678941;
IUPHAR/BPS	7481;
DrugBank	DB09280 ;
ChemSpider	17611836;
UNII	EGP8L81APK;
KEGG	D10134;
ChEBI	CHEBI:90951 ;
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 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) ; Key:UFSKUSARDNFIRC-UHFFFAOYSA-N ;
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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]