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Ivacaftor and Symdeko
Ivacaftor is a CFTR potentiator that increases the opening probability of the cystic fibrosis transmembrane conductance regulator (CFTR) channel, while Symdeko combines ivacaftor with lumacaftor, a CFTR corrector that helps misfolded CFTR protein reach the cell surface.
Ivacaftor is a CFTR potentiator that increases the opening probability of the cystic fibrosis transmembrane conductance regulator (CFTR) channel, while Symdeko combines ivacaftor with lumacaftor, a CFTR corrector that helps misfolded CFTR protein reach the cell surface. Used for Cystic fibrosis in patients with specific CFTR mutations (F508del and other gating mutations for ivacaftor; F508del-mediated CF for Symdeko).
At a glance
| Generic name | Ivacaftor and Symdeko |
|---|---|
| Sponsor | University of Alabama at Birmingham |
| Drug class | CFTR modulator (potentiator and corrector combination) |
| Target | CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) |
| Modality | Small molecule |
| Therapeutic area | Pulmonary/Respiratory |
| Phase | FDA-approved |
Mechanism of action
Ivacaftor works by binding to and stabilizing the open state of CFTR channels, allowing increased chloride ion transport across cell membranes. Lumacaftor acts as a corrector by assisting in proper folding and trafficking of CFTR protein to the cell membrane. Together, these agents restore CFTR function in cystic fibrosis patients, improving mucociliary clearance and reducing lung inflammation.
Approved indications
- Cystic fibrosis in patients with specific CFTR mutations (F508del and other gating mutations for ivacaftor; F508del-mediated CF for Symdeko)
Common side effects
- Elevated transaminases (ALT/AST)
- Headache
- Nausea
- Diarrhea
- Rash
Key clinical trials
- Novel Therapeutic Approaches for Treatment of CF Patients With W1282X Premature Termination Codon Mutations (PHASE4)
- Evaluation of the ReX-C System, in Patients Receiving CFTR Modulators for the Treatment of Cystic Fibrosis (CF). (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 |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Ivacaftor and Symdeko CI brief — competitive landscape report
- Ivacaftor and Symdeko updates RSS · CI watch RSS
- University of Alabama at Birmingham portfolio CI