elexacaftor

{{Short description|Cystic fibrosis medication}}

{{Infobox drug

| drug_name =

| INN =

| type =

| image = Elexacaftor.svg

| width =

| alt =

| caption =

| pronounce =

| tradename = Trikafta and Kaftrio (with ivacaftor and tezacaftor)

| Drugs.com = {{drugs.com|monograph|elexacaftor-tezacaftor-and-ivacaftor}}

| MedlinePlus = a619061

| licence_CA =

| licence_EU = yes

| DailyMedID = Elexacaftor

| licence_US =

| pregnancy_AU =

| pregnancy_AU_comment =

| pregnancy_US = N

| pregnancy_US_comment =

| pregnancy_category=

| dependency_liability =

| addiction_liability =

| routes_of_administration = By mouth

| class =

| ATCvet =

| ATC_prefix = None

| ATC_suffix =

| ATC_supplemental =

| legal_AU = S4

| legal_AU_comment =

| legal_BR =

| legal_BR_comment =

| legal_CA =

| legal_CA_comment =

| legal_DE =

| legal_DE_comment =

| legal_NZ =

| legal_NZ_comment =

| legal_UK =

| legal_UK_comment =

| legal_US = Rx-only

| legal_US_comment =

| legal_EU = Rx-only

| legal_EU_comment =

| legal_UN =

| legal_UN_comment =

| legal_status =

| bioavailability =

| protein_bound =

| metabolism =

| metabolites =

| onset =

| elimination_half-life =

| duration_of_action =

| excretion =

| CAS_number = 2216712-66-0

| CAS_number_Ref = {{cascite|correct|CAS}}

| UNII_Ref = {{fdacite|correct|FDA}}

| UNII = RRN67GMB0V

| CAS_supplemental =

| PubChem = 134587348

| IUPHAR_ligand =

| DrugBank = DB15444

| ChemSpiderID = 75531299

| KEGG = D11507

| ChEBI =

| ChEMBL = 4298128

| NIAID_ChemDB =

| PDB_ligand =

| synonyms = VX-445

| IUPAC_name = N-(1,3-dimethylpyrazol-4-yl)sulfonyl-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide

| C=26|H=34|F=3|N=7|O=4|S=1

| SMILES = C[C@H]1CC(N(C1)C2=C(C=CC(=N2)N3C=CC(=N3)OCC(C)(C)C(F)(F)F)C(=O)NS(=O)(=O)C4=CN(N=C4C)C)(C)C

| StdInChI=1S/C26H34F3N7O4S/c1-16-12-25(5,6)35(13-16)22-18(23(37)33-41(38,39)19-14-34(7)31-17(19)2)8-9-20(30-22)36-11-10-21(32-36)40-15-24(3,4)26(27,28)29/h8-11,14,16H,12-13,15H2,1-7H3,(H,33,37)/t16-/m0/s1

| StdInChIKey = MVRHVFSOIWFBTE-INIZCTEOSA-N

| density =

| density_notes =

| melting_point =

| melting_high =

| melting_notes =

| boiling_point =

| boiling_notes =

| solubility =

| sol_units =

| specific_rotation =

}}

Elexacaftor is a medication that acts as cystic fibrosis transmembrane conductance regulator (CFTR) corrector.

It is available in a single pill with ivacaftor and tezacaftor; the fixed-dose combination, elexacaftor/tezacaftor/ivacaftor (brand name Trikafta), is used to treat people with cystic fibrosis who are homozygous for the f508del mutation.{{cite web | title=Trikafta (elexacaftor, ivacaftor and tezacaftor) Patient Information | website=Drugs.com | date=October 23, 2019 | url=https://www.drugs.com/trikafta.html | archive-url=https://web.archive.org/web/20191030035925/https://www.drugs.com/trikafta.html | archive-date=October 30, 2019 | url-status=live | access-date=November 13, 2019}}{{cite journal | vauthors = Ridley K, Condren M | title = Elexacaftor-Tezacaftor-Ivacaftor: The First Triple-Combination Cystic Fibrosis Transmembrane Conductance Regulator Modulating Therapy | journal = The Journal of Pediatric Pharmacology and Therapeutics | volume = 25 | issue = 3 | pages = 192–197 | date = 2020 | pmid = 32265602 | pmc = 7134581 | doi = 10.5863/1551-6776-25.3.192 }} This combination was approved for medical use in the United States in 2019.{{cite press release | title=FDA approves new breakthrough therapy for cystic fibrosis | website=U.S. Food and Drug Administration (FDA) | date=October 21, 2019 | url=https://www.fda.gov/news-events/press-announcements/fda-approves-new-breakthrough-therapy-cystic-fibrosis | archive-url=https://web.archive.org/web/20191113165814/https://www.fda.gov/news-events/press-announcements/fda-approves-new-breakthrough-therapy-cystic-fibrosis | archive-date=November 13, 2019 | url-status=dead | access-date=November 13, 2019}} {{PD-notice}}{{cite web | title=Drug Trials Snapshots: Trikafta | website=U.S. Food and Drug Administration (FDA) | date=October 31, 2019 | url=https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots-trikafta | archive-url=https://web.archive.org/web/20191120203919/https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots-trikafta | archive-date=November 20, 2019 | url-status=dead | access-date=November 20, 2019}} {{PD-notice}}

The fixed-dose combination elexacaftor/tezacaftor/ivacaftor (Kaftrio) was approved for medical use in the European Union in August 2020, for the treatment of cystic fibrosis.{{cite web | title=Kaftrio EPAR | website=European Medicines Agency (EMA) | date=23 June 2020 | url=https://www.ema.europa.eu/en/medicines/human/EPAR/kaftrio | access-date=24 September 2020}}{{cite press release | title=New medicine for cystic fibrosis patients | website=European Medicines Agency (EMA) | date=26 June 2020 | url=https://www.ema.europa.eu/en/news/new-medicine-cystic-fibrosis-patients | access-date=26 June 2020}}