Last reviewed 2026-04-20 · How we verify

Kalydeco (IVACAFTOR)

Kalydeco works by helping the defective protein in cystic fibrosis cells function more normally.

Kalydeco (Ivacaftor) is a cystic fibrosis transmembrane conductance regulator potentiator developed by Vertex Pharms Inc. It is a small molecule modality that targets the cystic fibrosis transmembrane conductance regulator to treat cystic fibrosis of the lung. Kalydeco was FDA approved in 2012 and remains a patented product. Key safety considerations include monitoring for liver enzyme elevations and potential interactions with other medications. Kalydeco is a critical treatment option for patients with specific cystic fibrosis mutations.

At a glance

Mechanism of action

Ivacaftor is potentiator of the CFTR protein. The CFTR protein is chloride channel present at the surface of epithelial cells in multiple organs. Ivacaftor facilitates increased chloride transport by potentiating the channel open probability (or gating) of CFTR protein located at the cell surface. The overall level of ivacaftor-mediated CFTR chloride transport is dependent on the amount of CFTR protein at the cell surface and how responsive particular mutant CFTR protein is to ivacaftor potentiation. CFTR Chloride Transport Assay in Fisher Rat Thyroid (FRT) cells expressing mutant CFTRThe chloride transport response of mutant CFTR protein to ivacaftor was determined in Ussing chamber electrophysiology studies using panel of FRT cell lines transfected with individual CFTR mutations. Ivacaftor increased chloride transport in FRT cells expressing CFTR mutations that result in CFTR protein being delivered to the cell surface. The in vitro CFTR chloride transport r

Approved indications

• Cystic fibrosis of the lung

Common side effects

• Headache
• Oropharyngeal pain
• Upper respiratory tract infection
• Nasal congestion
• Abdominal pain
• Nasopharyngitis
• Diarrhea
• Rash
• Nausea
• Dizziness
• Rhinitis
• Aspartate aminotransferase increased

Drug interactions

• P-glycoprotein Substrates
• carbamazepine
• enzalutamide
• fosphenytoin
• phenobarbital
• phenytoin
• primidone
• rifabutin
• rifampicin

Key clinical trials

• Ivacaftor in French Patients With Cystic Fibrosis and a G551D Mutation
• A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF) (PHASE1,PHASE2)
• Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®. (NA)
• Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older (PHASE3)
• Immunomodulatory Treatment of Interstitial Lung Disease Associated With Surfactant Related Gene Variants (PHASE2)
• Modulate-CF: Cystic Fibrosis Transmembrane Regulator (CFTR) Biomarker Study to Evaluate the Rescue of Mutant CFTR in Patients With Cystic Fibrosis Treated With CFTR-modulators
• Population Pharmacokinetics of Elexacaftor-tezacaftor-ivacaftor in a Paediatric Population
• Trikafta for Patients With Non-cystic Fibrosis Bronchiectasis (PHASE4)

Patents

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• Kalydeco CI brief — competitive landscape report
• Kalydeco updates RSS · CI watch RSS
• Vertex Pharms Inc portfolio CI