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LUM/IVA
LUM/IVA is a combination of lumacaftor (a CFTR corrector) and ivacaftor (a CFTR potentiator) that work together to improve the processing and function of defective cystic fibrosis transmembrane conductance regulator (CFTR) protein.
LUM/IVA is a combination of lumacaftor (a CFTR corrector) and ivacaftor (a CFTR potentiator) that work together to improve the processing and function of defective cystic fibrosis transmembrane conductance regulator (CFTR) protein. Used for Cystic fibrosis in patients aged 6 years and older who are homozygous for the F508del-CFTR mutation or heterozygous for the F508del-CFTR mutation and one residual function mutation.
At a glance
| Generic name | LUM/IVA |
|---|---|
| Also known as | VX-809/VX-770, lumacaftor/ivacaftor, Lumacaftor/Ivacaftor, Orkambi, VX-809+VX-770 |
| Sponsor | Vertex Pharmaceuticals Incorporated |
| 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 corrects the misfolding of F508del-CFTR protein, allowing it to traffic properly to the cell surface. Ivacaftor then acts as a potentiator, increasing the open probability of the CFTR channel at the cell membrane, enhancing chloride ion transport. Together, these agents restore partial CFTR function in patients with cystic fibrosis carrying the F508del mutation.
Approved indications
- Cystic fibrosis in patients aged 6 years and older who are homozygous for the F508del-CFTR mutation or heterozygous for the F508del-CFTR mutation and one residual function mutation
Common side effects
- Nausea
- Diarrhea
- Fatigue
- Abdominal pain
- Headache
- Elevated liver transaminases
Key clinical trials
- 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)
- Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del (PHASE3)
- A Study to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del (PHASE2)
- Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor (PHASE3)
- Real Life Evaluation of the Multi-organ Effects of Lumacaftor/Ivacaftor on F508del Homozygous Cystic Fibrosis Patients.
- A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation (PHASE3)
- Lum-Iva-biota: Exploring the Respiratory Mycobiota and Microbiota Profile in French CF Patients Taking Lumacaftor-Ivacaftor
- A Study of the Effects of Lumacaftor/Ivacaftor (LUM/IVA) on Exercise Tolerance in Subjects With Cystic Fibrosis (CF), Homozygous for the F508del-CFTR Mutation (PHASE4)
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:
- LUM/IVA CI brief — competitive landscape report
- LUM/IVA updates RSS · CI watch RSS
- Vertex Pharmaceuticals Incorporated portfolio CI