ivacaftor (Kalydeco) Report issue

Small molecule Orphan Drug FDA Approved FDA First in Class Fast Track FDA Priority Review FDA
AACT ChEMBL DrugBank Drug Central Drugs@FDA KEGG MeSH PubChem repoDB

Active Ingredient History

NOW
  • 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

Chemistry

  • SMILES: CC(C)(C)C1=CC(=C(O)C=C1NC(=O)C2=CNC3=C(C=CC=C3)C2=O)C(C)(C)C
  • InChIKey: PURKAOJPTOLRMP-UHFFFAOYSA-N
  • Mol. Mass: 392.4907
  • ALogP: 5.08
  • ChEMBL Molecule:
    ivacaftor  
More Chemistry

Pharmacology

  • Mechanism of Action:
  • Multi-specific: Missing data
  • Black Box: No
  • Availability: Prescription Only
  • Delivery Methods: Oral
  • Pro Drug: No

Drug Pricing (per unit)

Australia

$295.9292

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 )

Alternative names

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

Organizations (47)

Research & Development (46)

Organization Org Type FDA approvals Clinical Trials involvement Org ID Force Sort

Marketing (2)

Organization Org Type FDA approvals Clinical Trials involvement Org ID Force Sort

Indications (11)

Approved Indications (1)


 

Cystic Fibrosis

Experimental Indications - Clinical Trial Phases 1-4 (11)


 

Bronchitis, Chronic (Phase 4)

Ciliary Motility Disorders (Phase 2)

Cystic Fibrosis ()

Drug Interactions (Phase 4)

Healthy Volunteers (Phase 1)

Liver Diseases (Phase 1)

Long QT Syndrome (Phase 2)

Lung Diseases (Phase 3)

Medication Adherence (Phase 4)

Pulmonary Disease, Chronic Obstructive (Phase 2)

Sinusitis (Phase 1)

Overview

  • Highest Phase: Phase 4
  • # of Trials: 122
  • # of Trial Organizations: 46

Trials by Phase

Trial Phase Start Date Organizations Indications

Feedback

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