NDAORALTABLETPriority Review
Approved
Jan 2012
Lifecycle
Peak
Competitive Pressure
8/100
Clinical Trials
21
Data completeness
6/8 — Strong
Priority Review
KALYDECO (ivacaftor) by Vertex Pharmaceuticals is chloride channel activation potentiators [moa]. Approved for cystic fibrosis transmembrane conductance regulator potentiator [epc]. First approved in 2012.
Drug data last refreshed 16h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
KALYDECO (ivacaftor) is an oral small-molecule CFTR potentiator approved in 2012 for cystic fibrosis patients with specific gating mutations. It activates the chloride channel conductance regulator, improving chloride transport and mucus clearance in CF patients. The drug represents a precision medicine approach targeting individual CFTR mutations.
Peak3.6 years to LOE
KALYDECO remains in peak commercial phase with established market penetration; team focus is on lifecycle management and defending position against newer triple-combination therapies.
Mechanism of Action
Chloride Channel Activation Potentiators
Pharmacologic Class:
Cystic Fibrosis Transmembrane Conductance Regulator Potentiator
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (21)
Effect of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) on the PK of Rosuvastatin in Healthy Participants
Started Jan 2026
18 enrolled
Cystic Fibrosis
Ivacaftor Impact in an Adolescent Without a Cystic Fibrosis-Associated CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) Mutation for Chronic Pancreatitis: Single-Patient Study Tracking Flare Frequency and Health Data Compared to Historical Records.
Started May 2025
1 enrolled
Chronic Pancreatitis
A Study of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) in Healthy Adult Panelists
Started Mar 2024
10 enrolled
Cystic Fibrosis
A Study To Evaluate the Relative Bioavailability, Food Effect, and Dose Proportionality of a Granule Formulation of Vanzacaftor/Tezacaftor/Deutivacaftor(VNZ/TEZ/D-IVA)
Started Mar 2024
34 enrolled
Cystic Fibrosis
Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older
Started Aug 2023
174 enrolled
Cystic Fibrosis
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.