ivacaftor (Kalydeco) Report issue

Small molecule Orphan Drug FDA Approved FDA First in Class Fast Track FDA Priority Review FDA

Active Ingredient History

  • Now
Ivacaftor (trade names KALYDECO® (ivacaftor) and ORKAMBI® (lumacaftor/ivacaftor)) is a cystic fibrosis transmembrane conductance regulator potentiator indicated for the treatment of cystic fibrosis in patients age 6 years and older who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R. One such defect G551D is characterized by a dysfunctional CFTR protein on the cell surface. Although the defective protein is trafficked to the correct area, the epithelial cell surface, while there it cannot transport chloride through the channel. Ivacaftor, a CFTR potentiator, improves the transport of chloride through the ion channel by binding to the channels directly to induce a non-conventional mode of gating which in turn increases the probability that the channel is open. Ivacaftor regulates fluid flow within cells and affects the components of sweat, digestive fluids, and mucus.   NCATS

  • SMILES: CC(C)(C)C1=CC(=C(O)C=C1NC(=O)C2=CNC3=C(C=CC=C3)C2=O)C(C)(C)C
  • Mol. Mass: 392.4907
  • ALogP: 5.08
  • ChEMBL Molecule:
More Chemistry
  • Mechanism of Action:
  • Multi-specific: Missing data
  • Black Box: No
  • Availability: Prescription Only
  • Delivery Methods: Oral
  • Pro Drug: No

Drug Pricing (per unit)



United States

$321.1538 - $428.8609
More Pricing Detail

Note: This drug pricing data is preliminary, incomplete, and may contain errors.

Combination drugs

( ivacaftor (Kalydeco), lumacaftor (Orkambi) - other names: ivacaftor and lumacaftor, lumacaftor/ ivacaftor, lumacaftor / ivacaftor, orkambi® oral tablet )
3-quinolinecarboxamide, n-(2,4-bis(1,1-dimethylethyl)-5-hydroxyphenyl)-1,4-dihydro-4-oxo- | d-ivacaftor | ivacaftor | ivacaftorum | kalydeco | lumacaftor / ivacaftor | n-(2,4-bis(1,1-dimethylethyl)-5-hydroxyphenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide | n-(2,4-di-tert-butyl-5-hydroxyphenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide | vx 770 | vx-770


Data collection and curation is an ongoing process for CDEK - if you notice any information here to be missing or incorrect, please let us know! When possible, please include a source URL (we verify all data prior to inclusion).

Report issue