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Ivacaftor+lumacaftor
Lumacaftor corrects the trafficking of defective CFTR protein to the cell surface, while ivacaftor enhances the channel activity of CFTR, together restoring chloride transport in cystic fibrosis.
Lumacaftor corrects the trafficking of defective CFTR protein to the cell surface, while ivacaftor enhances the channel activity of CFTR, together restoring chloride transport in cystic fibrosis. Used for Cystic fibrosis in patients homozygous for the F508del-CFTR mutation or heterozygous for F508del and a minimal function mutation.
At a glance
| Generic name | Ivacaftor+lumacaftor |
|---|---|
| Also known as | Orkambi |
| Sponsor | Assistance Publique - Hôpitaux de Paris |
| Drug class | CFTR modulator combination (corrector + potentiator) |
| Target | CFTR (cystic fibrosis transmembrane conductance regulator) |
| Modality | Small molecule |
| Therapeutic area | Respiratory / Genetic disease |
| Phase | FDA-approved |
Mechanism of action
Lumacaftor is a CFTR corrector that helps misfolded CFTR protein (particularly the F508del mutation) fold correctly and traffic to the apical membrane. Ivacaftor is a CFTR potentiator that increases the open probability and duration of the CFTR channel at the cell surface. Together, they address both the trafficking defect and the gating defect of mutant CFTR, improving ion and fluid transport in the airways.
Approved indications
- Cystic fibrosis in patients homozygous for the F508del-CFTR mutation or heterozygous for F508del and a minimal function mutation
Common side effects
- Nausea
- Diarrhea
- Abdominal pain
- Headache
- Rash
- Elevated liver transaminases
Key clinical trials
- Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®. (NA)
- 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
- Personalized Theratyping Trial (EARLY_PHASE1)
- Real-life Follow-up of Cystic Fibrosis Patients Treated With Ivacaftor+Lumacaftor (Orkambi*)
- Impact of the Introduction of ORKAMBI on Anxiety, Depression, Quality of Life and Adherence of Adolescents and Young Adults
- Cystic Fibrosis Blood Neutrophils (NA)
- Mutation-specific Therapy for the Long QT Syndrome (PHASE2)
- Monitoring Response to Orkambi in Cystic Fibrosis Lung Disease by Inhaled Xenon MRI
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Ivacaftor+lumacaftor CI brief — competitive landscape report
- Ivacaftor+lumacaftor updates RSS · CI watch RSS
- Assistance Publique - Hôpitaux de Paris portfolio CI