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Ivacaftor Exposed
Ivacaftor Exposed is a CFTR potentiator Small molecule drug developed by Vertex Pharmaceuticals Incorporated. It is currently in Phase 2 development for Cystic fibrosis.
Potentiates CFTR chloride channel function
Ivacaftor is a small molecule that acts as a positive modulator of the cystic fibrosis transmembrane conductance regulator. It is used to treat cystic fibrosis, as indicated by clinical trials registered on ClinicalTrials.gov.
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Baseline phase 2 → approval rate
+15.3pp
Industry-wide phase 2 drugs reach approval ~15.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Big-pharma sponsor
+3.0pp
Vertex Pharmaceuticals Incorporated is a top-20 pharma sponsor — historical approval rates run ~3pp above average due to scale, regulatory experience, and trial-design quality.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2031–2034 | — |
| EMA | EU | 2032–2035 | +0.7 yr |
| MHRA | GB | 2032–2035 | +0.7 yr |
| Health Canada | CA | 2032–2036 | +0.9 yr |
| TGA | AU | 2032–2036 | +1.2 yr |
| PMDA | JP | 2032–2036 | +1.5 yr |
| NMPA | CN | 2033–2037 | +2.3 yr |
| MFDS | KR | 2032–2036 | +1.4 yr |
| CDSCO | IN | 2032–2037 | +1.8 yr |
| ANVISA | BR | 2033–2037 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | Ivacaftor Exposed |
|---|---|
| Sponsor | Vertex Pharmaceuticals Incorporated |
| Drug class | CFTR potentiator |
| Target | CFTR |
| Modality | Small molecule |
| Therapeutic area | Respiratory |
| Phase | Phase 2 |
Mechanism of action
Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that works by increasing the amount of chloride ions that are secreted through the CFTR protein, which helps to thin mucus and improve lung function.
Approved indications
- Cystic fibrosis
Common side effects
- Nausea
- Vomiting
- Diarrhea
- Headache
- Fatigue
Key clinical trials
- Gut Imaging for Function & Transit in CF - GIFT-CF 3
- CFTR Modulators in Pregnancy and Postpartum
- Impact of Triple Combination CFTR Therapy on Sinus Disease.
- A Study to Confirm the Long-term Safety and Effectiveness of Kalydeco in Patients With Cystic Fibrosis Who Have an R117H-CFTR Mutation, Including Pediatric Patients
- An Ocular Safety Study of Ivacaftor-Treated Pediatric Patients 11 Years of Age or Younger With Cystic Fibrosis
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 Exposed CI brief — competitive landscape report
- Ivacaftor Exposed updates RSS · CI watch RSS
- Vertex Pharmaceuticals Incorporated portfolio CI
Frequently asked questions about Ivacaftor Exposed
What is Ivacaftor Exposed?
How does Ivacaftor Exposed work?
What is Ivacaftor Exposed used for?
Who makes Ivacaftor Exposed?
What drug class is Ivacaftor Exposed in?
What development phase is Ivacaftor Exposed in?
What are the side effects of Ivacaftor Exposed?
What does Ivacaftor Exposed target?
Related
- Drug class: All CFTR potentiator drugs
- Target: All drugs targeting CFTR
- Manufacturer: Vertex Pharmaceuticals Incorporated — full pipeline
- Therapeutic area: All drugs in Respiratory
- Indication: Drugs for Cystic fibrosis
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing