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IVA
IVA is a CFTR potentiator that increases the open probability of the cystic fibrosis transmembrane conductance regulator protein to enhance chloride ion transport.
IVA is a CFTR potentiator that increases the open probability of the cystic fibrosis transmembrane conductance regulator protein to enhance chloride ion transport. Used for Cystic fibrosis in patients aged 6 months and older with specific CFTR mutations (gating mutations including G551D), Cystic fibrosis in combination with lumacaftor in patients with F508del-CFTR mutations.
At a glance
| Generic name | IVA |
|---|---|
| Also known as | VX-770, ivacaftor, Ivacaftor, ivacaftor; VX-770, Kalydeco |
| Sponsor | Vertex Pharmaceuticals Incorporated |
| Drug class | CFTR potentiator |
| Target | CFTR (cystic fibrosis transmembrane conductance regulator) |
| Modality | Small molecule |
| Therapeutic area | Pulmonary/Respiratory |
| Phase | FDA-approved |
Mechanism of action
IVA (ivacaftor) binds directly to the CFTR protein and increases the likelihood that the channel will remain open, allowing more chloride ions to flow across cell membranes. This corrects the defective ion transport characteristic of cystic fibrosis, particularly in patients with gating mutations. By restoring CFTR function, ivacaftor improves mucociliary clearance and reduces airway inflammation.
Approved indications
- Cystic fibrosis in patients aged 6 months and older with specific CFTR mutations (gating mutations including G551D)
- Cystic fibrosis in combination with lumacaftor in patients with F508del-CFTR mutations
Common side effects
- Headache
- Upper respiratory tract infection
- Nausea
- Diarrhea
- Rash
- Abdominal pain
Key clinical trials
- Testing the Addition of a New Anti-Cancer Drug, Triapine, to the Usual Chemotherapy Treatment (Cisplatin) During Radiation Therapy for Advanced-stage Cervical and Vaginal Cancers (PHASE3)
- Brentuximab Vedotin and Combination Chemotherapy in Treating Children and Young Adults With Stage IIB, Stage IIIB, IVA, or IVB Hodgkin Lymphoma (PHASE3)
- A Phase 3 Study Evaluating Efficacy and Safety of Lanifibranor Followed by an Active Treatment Extension in Adult Patients With (NASH) and Fibrosis Stages F2 and F3 ( NATiV3 ) (PHASE3)
- Testing the Addition of the Immunotherapy Drug Pembrolizumab to the Usual Chemotherapy Treatment (Paclitaxel and Carboplatin) in Stage III-IV or Recurrent Endometrial Cancer (PHASE3)
- Evaluation of VX-828 in Healthy Participants and in Participants With Cystic Fibrosis (PHASE1)
- A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF) (PHASE1, PHASE2)
- Radiation Therapy With or Without Cisplatin in Treating Patients With Stage III-IVA Squamous Cell Carcinoma of the Head and Neck Who Have Undergone Surgery (PHASE2)
- CCCG-HD-2018 for Children and Adolescents With Newly Diagnosed Hodgkin Lymphoma (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 |