NDAORALGRANULEPriority Review
Approved
Mar 2015
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
21
Data completeness
7/8 — Comprehensive
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 2015.
Drug data last refreshed 7h ago · AI intelligence enriched 6d ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
KALYDECO (ivacaftor) is an oral small-molecule CFTR potentiator approved in 2015 for cystic fibrosis patients with specific gating mutations. It works by activating chloride channel function in the CFTR protein, improving airway clearance and lung function. The drug is indicated for CF patients with G551D and other gating mutations in the CFTR gene.
$25MPart D
PeakStable
7.2 years to LOEMature, well-established franchise with stable revenue supporting a stable commercial team focused on patient retention and indication expansion.
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.