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VX-809
VX-809 is a cystic fibrosis transmembrane conductance regulator (CFTR) corrector.
VX-809 is a cystic fibrosis transmembrane conductance regulator (CFTR) corrector. Used for Cystic fibrosis.
At a glance
| Generic name | VX-809 |
|---|---|
| Also known as | lumacaftor |
| Sponsor | Vertex Pharmaceuticals Incorporated |
| Drug class | CFTR corrector |
| Target | CFTR |
| Modality | Small molecule |
| Therapeutic area | Cystic Fibrosis |
| Phase | Phase 3 |
Mechanism of action
VX-809 corrects the function of the CFTR protein by binding to the protein and facilitating its proper folding and trafficking to the cell surface. This increases the amount of functional CFTR protein on the cell surface, which helps to restore normal chloride transport across epithelial cells.
Approved indications
- Cystic fibrosis
Common side effects
- Nausea
- Vomiting
- Diarrhea
- Headache
- Fatigue
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*)
- Cystic Fibrosis Blood Neutrophils (NA)
- Mutation-specific Therapy for the Long QT Syndrome (PHASE2)
- 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)
- Surrogate Markers of Response to New Therapies in Cystic Fibrosis Patients (NA)
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 |