Drug Information

Drug General Information
Drug ID
D0X0CB
Former ID
DIB010726
Drug Name
Lumacaftor
Synonyms
VRT-325; VRT-422; VRT-532; VX-809; CFTR correctors (cystic fibrosis), Vertex; Cystic fibrosis transmembrane regulator corrector (cystic fibrosis), Vertex
Drug Type
Small molecular drug
Indication Cystic fibrosis [ICD9: 277; ICD10:E84] Phase 2 [522612], [542504]
Company
Vertex Pharmaceuticals Inc
Formula
C24H18F2N2O5
InChI
InChI=1S/C16H14N2O/c1-11-7-8-16(19)13(9-11)15-10-14(17-18-15)12-5-3-2-4-6-12/h2-10,19H,1H3,(H,17,18)
InChIKey
AQWKVJFRGORALM-UHFFFAOYSA-N
PubChem Compound ID
16678941
Target and Pathway
Target(s) cystic fibrosis transmembrane conductance regulator Target Info Regulator [532863]
KEGG Pathway ABC transporters
cAMP signaling pathway
AMPK signaling pathway
Gastric acid secretion
Pancreatic secretion
Bile secretion
Vibrio cholerae infection
Reactome ABC-family proteins mediated transport
WikiPathways ABC-family proteins mediated transport
References
Ref 522612ClinicalTrials.gov (NCT00865904) Study of VX-809 in Cystic Fibrosis Subjects With the ???508-CFTR Gene Mutation. U.S. National Institutes of Health.
Ref 542504(http://www.guidetopharmacology.org/) Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Ligand id: 7481).
Ref 532863A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial. Lancet Respir Med. 2014 Jul;2(7):527-38.

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