Lumacaftor

Lumacaftor
Clinical data
License data
ATC code
  • None
Legal status
Legal status
Identifiers
CAS Number
PubChem CID
IUPHAR/BPS
ChemSpider
KEGG
ChEBI
ChEMBL
Chemical and physical data
Formula C24H18F2N2O5
Molar mass 452.407 g/mol
3D model (JSmol)
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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.[1] 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 have the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR), the defective protein that causes the disease.[2] It was developed by Vertex Pharmaceuticals and the combination was approved by the FDA in 2015.[3] As of 2015 lumacaftor had no medical use on its own.[1]

See also

  • Ataluren, targeting premature stop codons

References

  1. 1 2 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. Review. PMID 26416827
  2. ↑ Lumacaftor label. Last updated July 2015. Check index page here for label updates
  3. ↑ "Orkambi (lumacaftor and ivacaftor)". CenterWatch. Retrieved 24 March 2016.
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