Last reviewed 2026-04-20 · How we verify

Kalydeco

At a glance

Approved indications

• Cystic fibrosis of the lung

Common side effects

• Headache
• Upper respiratory tract infection
• Nasal congestion
• Nausea
• Rash
• Rhinitis
• Dizziness
• Arthralgia
• Bacteria in sputum
• Oropharyngeal pain
• Abdominal pain
• Nasopharyngitis

Serious adverse events

• Abdominal pain
• Increased hepatic enzymes
• Hypoglycemia
• Elevated liver transaminases

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)

Primary sources

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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
• Concert Pharmaceuticals portfolio CI