Lumacaftor

Lumacaftor

Clinical data
ATC code	None
Legal status
Legal status	US: Available only as Lumacaftor/ivacaftor which is Rx-only
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
ChemSpider	17611836
ChEBI	CHEBI:90951
Chemical and physical data
Formula	C24H18F2N2O5
Molar mass	452.407 g/mol
3D model (Jmol)	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 have the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR), 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. Kuk K, Taylor-Cousar JL. Lumacaftor and ivacaftor in the management of patients with cystic fibrosis: current evidence and future prospects. Ther Adv Respir Dis. 2015 Dec;9(6):313-26. Review. 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.