Tezacaftor
SYMDEKO with ivacaftor
What it isA CFTR corrector that improves folding and trafficking of mutant CFTR, always given with ivacaftor (SYMDEKO) or within the triple combination.
Why this oneIt corrected F508del processing with fewer drug interactions and less rash than lumacaftor, becoming the corrector backbone the triple therapy built on.
What limits itIt does nothing alone — pairing with a potentiator is obligatory — and CYP3A inhibitors demand dose adjustment.
FDA label
Read the full label on DailyMed ↗
| FDA dosing | Population | Start | Target | Max |
|---|---|---|---|---|
| Cystic fibrosis (F508del homozygous or CFTR mutation responsive to tezacaftor/ivacaftor) | Aged 6 years and older | 6 to <12 y & <30 kg: tezacaftor 50 mg/ivacaftor 75 mg AM + ivacaftor 75 mg PM; ≥12 y or 6–<12 y & ≥30 kg: tezacaftor 100 mg/ivacaftor 150 mg AM + ivacaftor 150 mg PM | Morning and evening doses ~12 h apart | — |
Renal Undefined
Hepatic Mild (Child-Pugh A): no adjustment; Moderate (B): once-daily morning tablet only, no evening ivacaftor; Severe (C): use with caution at reduced dose (once daily or less frequently)
Missed dose: take if ≤6 h late; otherwise skip and resume schedule
Instructions
- Swallow tablets whole
- Take with fat-containing food
- Morning and evening doses ~12 h apart
- Avoid grapefruit
- Adjust dose with moderate/strong CYP3A inhibitors; avoid strong CYP3A inducers
Cautions
- Transaminase (ALT/AST) elevations — monitor before and during treatment
- Hypersensitivity reactions, including anaphylaxis
- Intracranial hypertension
- Neuropsychiatric events including suicidal ideation and behavior
- Reduced effectiveness with CYP3A inducers
- Non-congenital cataracts in pediatric patients — baseline and follow-up eye exams
Adverse reactions
- Headache
- Nausea
- Sinus congestion
- Dizziness
Pregnancy Limited human data; no teratogenicity or adverse developmental effects in animal studies
Lactation No human data; both tezacaftor and ivacaftor excreted in rat milk
NO BOXED WARNINGS
Classes and tags
Clinical profile
Mechanism of action: Tezacaftor ballicule
