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Revision as of 08:06, 3 November 2011 editBeetstra (talk | contribs)Edit filter managers, Administrators172,031 edits Script assisted update of identifiers for the Chem/Drugbox validation project (updated: 'StdInChI', 'StdInChIKey', 'CAS_number').← Previous edit Latest revision as of 22:52, 20 December 2024 edit undoInternetArchiveBot (talk | contribs)Bots, Pending changes reviewers5,383,277 edits Rescuing 1 sources and tagging 0 as dead.) #IABot (v2.0.9.5) (Whoop whoop pull up - 22142 
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{{Short description|Cystic fibrosis drug}}
{{Drugbox {{Drugbox
| Verifiedfields = changed
⚫ | IUPAC_name = 3-{6-{amino}-3-methylpyridin-2-yl}benzoic acid
| verifiedrevid = 458773847
⚫ | image = VX-807 skeletal.svg
⚫ | IUPAC_name = 3-<nowiki/>{6-<nowiki/>{amino}-3-methylpyridin-2-yl}benzoic acid
⚫ | image =Lumacaftor_skeletal.svg
| alt = | alt =
| caption = | caption =
Line 12: Line 15:
| pregnancy_US = <!-- A / B / C / D / X --> | pregnancy_US = <!-- A / B / C / D / X -->
| pregnancy_category= | pregnancy_category=
| licence_EU = yes
| legal_AU = <!-- S2, S3, S4, S5, S6, S7, S8, S9 or Unscheduled--> | legal_AU = <!-- S2, S3, S4, S5, S6, S7, S8, S9 or Unscheduled-->
| legal_CA = <!-- OTC, Rx-only, Schedule I, II, III, IV, V, VI, VII, VIII --> | legal_CA = <!-- OTC, Rx-only, Schedule I, II, III, IV, V, VI, VII, VIII -->
| legal_UK = <!-- GSL, P, POM, CD, CD Lic, CD POM, CD No Reg POM, CD (Benz) POM, CD (Anab) POM or CD Inv POM --> | legal_UK = <!-- GSL, P, POM, CD, CD Lic, CD POM, CD No Reg POM, CD (Benz) POM, CD (Anab) POM or CD Inv POM -->
| legal_US = <!-- OTC / Rx-only / Schedule I, II, III, IV, V --> | legal_US = rx-only
| legal_US_comment = available only as ]
| legal_status = Investigational | legal_status =
| routes_of_administration = | routes_of_administration = ]


<!--Pharmacokinetic data--> <!--Pharmacokinetic data-->
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<!--Identifiers--> <!--Identifiers-->
| IUPHAR_ligand = 7481
⚫ | CAS_number = <!-- blanked - oldvalue: 936727-05-8 -->
| CAS_number_Ref = {{cascite|changed|??}}
⚫ | CAS_number = 936727-05-8
| ATCvet = | ATCvet =
| ATC_prefix = None | ATC_prefix = R07
| ATC_suffix = | ATC_suffix = AX30
| ATC_supplemental = (combination with ])
| PubChem = 16678941 | PubChem = 16678941
| ChEBI_Ref = {{ebicite|correct|EBI}}
⚫ | DrugBank =
| ChEBI = 90951
| ChEMBL = 2103870
| DrugBank_Ref = {{drugbankcite|correct|drugbank}}
⚫ | DrugBank = DB09280
| ChemSpiderID = 17611836
| KEGG = D10134
| UNII = EGP8L81APK
| synonyms = VX-809


<!--Chemical data--> <!--Chemical data-->
| C=24|H=18|F=2|N=2|O=5 | C=24|H=18|F=2|N=2|O=5
| molecular_weight = 452.407 g/mol
| 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 | 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
| StdInChI_Ref = {{stdinchicite|changed|chemspider}}
| StdInChI = <!-- blanked - oldvalue: 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) --> | StdInChI = 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)
⚫ | StdInChIKey = <!-- blanked - oldvalue: UFSKUSARDNFIRC-UHFFFAOYSA-N -->
| StdInChIKey_Ref = {{stdinchicite|changed|chemspider}}
⚫ | StdInChIKey = UFSKUSARDNFIRC-UHFFFAOYSA-N
}} }}


'''Lumacaftor''' ('''VX-809''') is a pharmaceutical drug that acts as a ] during ] and increases the number of CFTR proteins that are trafficked to the cell surface.<ref name=2015revKuk/> It is available in a single pill with ]; the combination, ] (brand name ''Orkambi''), is used to treat people with ] who are homozygous for the ] mutation in the ] (CFTR) gene, the defective protein that causes the disease.<ref name=Label>. Last updated July 2015. Check index page for label updates</ref> It was developed by ] and the combination was approved by the FDA in 2015.<ref name=CW>{{cite web|title=Orkambi (lumacaftor and ivacaftor)|url=http://www.centerwatch.com/drug-information/fda-approved-drugs/drug/100082/orkambi-lumacaftor-and-ivacaftor|publisher=CenterWatch|accessdate=24 March 2016|archive-date=19 March 2016|archive-url=https://web.archive.org/web/20160319043353/http://www.centerwatch.com/drug-information/fda-approved-drugs/drug/100082/orkambi-lumacaftor-and-ivacaftor|url-status=dead}}</ref> As of 2015, lumacaftor had no medical use on its own.<ref name=2015revKuk>{{cite journal | vauthors = Kuk K, Taylor-Cousar JL | title = Lumacaftor and ivacaftor in the management of patients with cystic fibrosis: current evidence and future prospects | journal = Therapeutic Advances in Respiratory Disease | volume = 9 | issue = 6 | pages = 313–26 | date = December 2015 | pmid = 26416827 | doi = 10.1177/1753465815601934 | doi-access = free }}</ref>
'''VX-807''' is an experimental drug for the treatment of ]. The drug is designed to be effective in patients that have the F508del mutation in the ] (CFTR), the defective protein that causes the disease. F508del, meaning that the amino acid ] in position 508 is missing, is found in about 90% of cystic fibrosis patients.<ref>{{cite journal|journal=Pharmazeutische Zeitung|language=German|authors=Merk; Schubert-Zsilavecz|volume=156|issue=37|pages=24–27}}</ref>

==See also==
* ], targeting premature stop codons


==References== ==References==
{{Reflist}} {{Reflist}}


{{Other respiratory system products}}
{{Uncategorized stub|date=November 2011}}


]
]
]
]
]
]


{{pharma-stub}} {{respiratory-system-drug-stub}}

Latest revision as of 22:52, 20 December 2024

Cystic fibrosis drug Pharmaceutical compound
Lumacaftor
Clinical data
Other namesVX-809
License data
Routes of
administration		By mouth
ATC code
Legal status
Legal status
Identifiers
IUPAC name
  • 3-{6-{amino}-3-methylpyridin-2-yl}benzoic acid
CAS Number
PubChem CID
IUPHAR/BPS
DrugBank
ChemSpider
UNII
KEGG
ChEBI
ChEMBL
CompTox Dashboard (EPA)
ECHA InfoCard100.241.800 Edit this at Wikidata
Chemical and physical data
FormulaC24H18F2N2O5
Molar mass452.414 g·mol
3D model (JSmol)
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
  (what is this?)  (verify)

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.

See also

  • Ataluren, targeting premature stop codons

References

  1. ^ Kuk K, Taylor-Cousar JL (December 2015). "Lumacaftor and ivacaftor in the management of patients with cystic fibrosis: current evidence and future prospects". Therapeutic Advances in Respiratory Disease. 9 (6): 313–26. doi:10.1177/1753465815601934. PMID 26416827.
  2. Lumacaftor label. Last updated July 2015. Check index page here for label updates
  3. "Orkambi (lumacaftor and ivacaftor)". CenterWatch. Archived from the original on 19 March 2016. Retrieved 24 March 2016.
Other respiratory system products (R07)
Lung surfactants
Respiratory stimulants
5-HT4 receptor agonists
Other agents for treating respiratory depression
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