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Lumacaftor plus Ivacaftor
Lumacaftor and ivacaftor work together to correct the misfolding and trafficking of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, restoring its function at the cell surface.
Lumacaftor and ivacaftor work together to correct the misfolding and trafficking of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, restoring its function at the cell surface. Used for Cystic fibrosis in patients homozygous for the F508del-CFTR mutation.
At a glance
| Generic name | Lumacaftor plus Ivacaftor |
|---|---|
| Also known as | Orkambi; VX-770; VX-809, VX-809+VX-770, LUM+IVA |
| Sponsor | Hannover Medical School |
| Drug class | CFTR modulator combination (corrector + potentiator) |
| Target | CFTR (cystic fibrosis transmembrane conductance regulator) |
| Modality | Small molecule |
| Therapeutic area | Pulmonary / Cystic Fibrosis |
| Phase | Phase 3 |
Mechanism of action
Lumacaftor is a CFTR corrector that helps the mutant F508del-CFTR protein fold properly and traffic to the cell membrane. Ivacaftor is a CFTR potentiator that increases the open probability of the CFTR channel once it reaches the cell surface, allowing chloride ions to flow through. Together, they address both the trafficking defect and the gating defect of F508del-CFTR, the most common cystic fibrosis mutation.
Approved indications
- Cystic fibrosis in patients homozygous for the F508del-CFTR mutation
Common side effects
- Nausea
- Diarrhea
- Abdominal pain
- Headache
- Elevated transaminases
Key clinical trials
- 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*)
- Cystic Fibrosis Blood Neutrophils (NA)
- Long-term Safety of Lumacaftor/Ivacaftor in Participants With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation (PHASE3)
- Combined Effect of CFTR Protein Modulator Drugs and Exercise in Cystic Fibrosis (NA)
- Effect of Lumacaftor/Ivacaftor in Children With Cystic Fibrosis Homozygote for F508del on Small Airway Function
- Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del (PHASE3)
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:
- Lumacaftor plus Ivacaftor CI brief — competitive landscape report
- Lumacaftor plus Ivacaftor updates RSS · CI watch RSS
- Hannover Medical School portfolio CI