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CFTR modulators
CFTR modulators work by modifying the cystic fibrosis transmembrane conductance regulator protein to restore normal chloride ion transport across epithelial cell membranes.
CFTR modulators work by modifying the cystic fibrosis transmembrane conductance regulator protein to restore normal chloride ion transport across epithelial cell membranes. Used for Cystic fibrosis, Cystic fibrosis with a F508del mutation.
At a glance
| Generic name | CFTR modulators |
|---|---|
| Also known as | Orkambi, Kalydeko..., EudraCT Number 2019-000833-37 |
| Sponsor | Hospices Civils de Lyon |
| Drug class | CFTR modulator |
| Target | CFTR |
| Modality | Small molecule |
| Therapeutic area | Respiratory |
| Phase | Phase 2 |
Mechanism of action
This is achieved through the correction of the underlying genetic defect in cystic fibrosis, allowing for improved lung function and reduced risk of pulmonary complications. CFTR modulators have been shown to increase chloride ion transport and improve sweat chloride levels in patients with cystic fibrosis.
Approved indications
- Cystic fibrosis
- Cystic fibrosis with a F508del mutation
Common side effects
- Upper respiratory tract infection
- Nausea
- Diarrhea
- Headache
- Fatigue
Key clinical trials
- Characterisation of a Population of Adults Suffering From Cystic Fibrosis in a Belgian Reference Center
- 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
- A Study to Test How Well BI 3720931 is Tolerated and Whether it Improves Lung Function in People With Cystic Fibrosis (Lenticlair™ 1) (PHASE1, PHASE2)
- Personalized Theratyping Trial (EARLY_PHASE1)
- Pharmacokinetics of CFTR Modulators in Pregnant Individuals and in Postpartum Breastfeeding Mothers
- Glucose Metabolism in Cystic Fibrosis Related Diabetes (CFRD)
- Testing Drug Efficacy in Cystic Fibrosis Through N-of-1 Trials (NA)
- Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators. (PHASE2)
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:
- CFTR modulators CI brief — competitive landscape report
- CFTR modulators updates RSS · CI watch RSS
- Hospices Civils de Lyon portfolio CI