lumacaftor

{{Short description|Cystic fibrosis drug}}

{{Drugbox

| Verifiedfields = changed

| verifiedrevid = 458773847

| IUPAC_name = 3-{6-{[1-(2,2-Difluoro-1,3-benzodioxol-5-yl)cyclopropanecarbonyl]amino}-3-methylpyridin-2-yl}benzoic acid

| image =Lumacaftor_skeletal.svg

| alt =

| caption =

| tradename =

| Drugs.com =

| MedlinePlus =

| pregnancy_AU =

| pregnancy_US =

| pregnancy_category=

| licence_EU = yes

| legal_AU =

| legal_CA =

| legal_UK =

| legal_US = rx-only

| legal_US_comment = available only as Lumacaftor/ivacaftor

| legal_status =

| routes_of_administration = By mouth

| bioavailability =

| protein_bound =

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| IUPHAR_ligand = 7481

| CAS_number_Ref = {{cascite|changed|??}}

| CAS_number = 936727-05-8

| ATCvet =

| ATC_prefix = R07

| ATC_suffix = AX30

| ATC_supplemental = (combination with ivacaftor)

| PubChem = 16678941

| ChEBI_Ref = {{ebicite|correct|EBI}}

| ChEBI = 90951

| ChEMBL = 2103870

| DrugBank_Ref = {{drugbankcite|correct|drugbank}}

| DrugBank = DB09280

| ChemSpiderID = 17611836

| KEGG = D10134

| UNII = EGP8L81APK

| synonyms = VX-809

| C=24|H=18|F=2|N=2|O=5

| 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 = 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_Ref = {{stdinchicite|changed|chemspider}}

| StdInChIKey = UFSKUSARDNFIRC-UHFFFAOYSA-N

}}

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.[https://www.accessdata.fda.gov/drugsatfda_docs/label/2015/206038Orig1s000lbl.pdf Lumacaftor label]. Last updated July 2015. Check index page [http://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm?fuseaction=Search.Set_Current_Drug&ApplNo=206038&DrugName=ORKAMBI&ActiveIngred=IVACAFTOR%3B%20LUMACAFTOR&SponsorApplicant=VERTEX%20PHARMS%20INC&ProductMktStatus=1&goto=Search.Label_ApprovalHistory here]{{dead link|date=May 2025|bot=medic}}{{cbignore|bot=medic}} for label updates It was developed by Vertex Pharmaceuticals and the combination was approved by the FDA in 2015.{{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}} As of 2015, lumacaftor had no medical use on its own.{{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 }}