lumacaftor 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. 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. it was developed by vertex pharmaceuticals and the combination was approved by the fda in 2015. as of 2015, lumacaftor had no medical use on its own. lumacaftorclinical dataother namesvx-809license data eu ema: by inn routes ofadministrationby mouthatc coder07ax30 (who) (combination with ivacaftor)legal statuslegal status us: ℞-only available only as lumacaftor/ivacaftor identifiers iupac name 3--3-methylpyridin-2-yl}benzoic acid cas number936727-05-8 npubchem cid16678941iuphar/bps7481drugbankdb09280 ychemspider17611836uniiegp8l81apkkeggd10134chebichebi:90951 ychemblchembl2103870comptox dashboard (epa)dtxsid30239523 echa infocard100.241.800 chemical and physical dataformulac24h18f2n2o5molar mass452.414 g·mol−13d 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) nkey:ufskusardnfirc-uhfffaoysa-n n ny (what is this?) (verify)