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Product NO.: 936727-05-8
Aliases Lumacaftor;VRT 826809 
CAS NO. 936727-05-8
Formula C24H18F2N2O5 
Weight 452.41 

Description: Lumacaftor, also known as VX-809, is a potent CFTR corrector, many be useful for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation. Cystic fibrosis (CF) is a genetic disorder that causes multiorgan morbidity and premature death, most commonly from pulmonary dysfunction.

Name: Lumacaftor 
CAS#: 936727-05-8 
Chemical Formula: C24H18F2N2O5 
Exact Mass: 452.11838 
Molecular Weight: 452.41 
Elemental Analysis: C, 63.72; H, 4.01; F, 8.40; N, 6.19; O, 17.68

Synonym: VX-809; VX809; VX 809; Lumacaftor

IUPAC/Chemical Name: 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl)cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid

SMILES Code: O=C(O)C1=CC=CC(C2=NC(NC(C3(C4=CC=C(OC(F)(F)O5)C5=C4)CC3)=O)=CC=C2C)=C1

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3: Bosch B, De Boeck K. Searching for a cure for cystic fibrosis. A 25-year quest in a nutshell. Eur J Pediatr. 2015 Nov 14. [Epub ahead of print] PubMed PMID: 26567541.

4: Dong ZW, Chen J, Ruan YC, Zhou T, Chen Y, Chen Y, Tsang LL, Chan HC, Peng YZ. CFTR-regulated MAPK/NF-κB signaling in pulmonary inflammation in thermal inhalation injury. Sci Rep. 2015 Oct 30;5:15946. doi: 10.1038/srep15946. PubMed PMID: 26515683; PubMed Central PMCID: PMC4626762.

5: Rehman A, Baloch NU, Janahi IA. Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR. N Engl J Med. 2015 Oct 29;373(18):1783. doi: 10.1056/NEJMc1510466#SA1. PubMed PMID: 26510035.

6: Wainwright CE, Elborn JS, Ramsey BW. Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR. N Engl J Med. 2015 Oct 29;373(18):1783-4. doi: 10.1056/NEJMc1510466. PubMed PMID: 26510034.

7: Elborn JS, Ramsey B, Wainwright C, Boyle M. Response to: 'Lumacaftor/ivacaftor for patients homozygous for Phe508del-CFTR: should we curb our enthusiasm?' by Jones and Barry. Thorax. 2015 Oct 27. pii: thoraxjnl-2015-207611. doi: 10.1136/thoraxjnl-2015-207611. [Epub ahead of print] PubMed PMID: 26506855.

8: Matthes E, Goepp J, Carlile GW, Luo Y, Dejgaard K, Billet A, Robert R, Thomas DY, Hanrahan JW. Low free drug concentration prevents inhibition of F508del CFTR functional expression by the potentiator VX-770 (ivacaftor). Br J Pharmacol. 2015 Oct 22. doi: 10.1111/bph.13365. [Epub ahead of print] PubMed PMID: 26492939.

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10: 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. doi: 10.1177/1753465815601934. Epub 2015 Sep 28. Review. PubMed PMID: 26416827.

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13: Bali V, Lazrak A, Guroji P, Fu L, Matalon S, Bebok Z. A synonymous codon change alters the drug sensitivity of ΔF508 cystic fibrosis transmembrane conductance regulator. FASEB J. 2015 Sep 3. pii: fj.15-273714. [Epub ahead of print] PubMed PMID: 26336913.

14: Goralski JL, Davis SD. Improving complex medical care while awaiting next-generation CFTR potentiators and correctors: The current pipeline of therapeutics. Pediatr Pulmonol. 2015 Oct;50 Suppl 40:S66-73. doi: 10.1002/ppul.23253. PubMed PMID: 26335956.

15: Phuan PW, Veit G, Tan JA, Finkbeiner WE, Lukacs GL, Verkman AS. Potentiators of Defective ΔF508-CFTR Gating that Do Not Interfere with Corrector Action. Mol Pharmacol. 2015 Oct;88(4):791-9. doi: 10.1124/mol.115.099689. Epub 2015 Aug 5. PubMed PMID: 26245207; PubMed Central PMCID: PMC4576684.

16: Sinha C, Zhang W, Moon CS, Actis M, Yarlagadda S, Arora K, Woodroofe K, Clancy JP, Lin S, Ziady AG, Frizzell R, Fujii N, Naren AP. Capturing the Direct Binding of CFTR Correctors to CFTR by Using Click Chemistry. Chembiochem. 2015 Sep 21;16(14):2017-22. doi: 10.1002/cbic.201500123. Epub 2015 Aug 11. PubMed PMID: 26227551; PubMed Central PMCID: PMC4672727.

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18: Tomati V, Sondo E, Armirotti A, Caci E, Pesce E, Marini M, Gianotti A, Jeon YJ, Cilli M, Pistorio A, Mastracci L, Ravazzolo R, Scholte B, Ronai Z, Galietta LJ, Pedemonte N. Genetic Inhibition Of The Ubiquitin Ligase Rnf5 Attenuates Phenotypes Associated To F508del Cystic Fibrosis Mutation. Sci Rep. 2015 Jul 17;5:12138. doi: 10.1038/srep12138. PubMed PMID: 26183966; PubMed Central PMCID: PMC4505316.

19: Nau JY. [Cystic Fibrosis: toward a genetic treatment; Clostridium difficile versus Clostridium difficile]. Rev Med Suisse. 2015 May 27;11(476):1210-1. French. PubMed PMID: 26182641.

20: Farinha CM, Sousa M, Canato S, Schmidt A, Uliyakina I, Amaral MD. Increased efficacy of VX-809 in different cellular systems results from an early stabilization effect of F508del-CFTR. Pharmacol Res Perspect. 2015 Aug;3(4):e00152. doi: 10.1002/prp2.152. Epub 2015 Jun 11. PubMed PMID: 26171232; PubMed Central PMCID: PMC4492728.