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🏅 FDA Orphan Designation

Trikafta

elexacaftor/tezacaftor/ivacaftor

Manufacturer: Vertex Pharmaceuticals

Indicated for:
Cystic fibrosisOrphan
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Black Box Warning (Boxed Warning)

A Boxed Warning is the FDA's strongest safety warning — it indicates serious or life-threatening risks identified during clinical trials or post-market surveillance. It does not mean the medication is unsafe for all patients; your prescriber weighs these risks against the benefits for your specific situation. Source: FDA-approved prescribing information.

View full prescribing information on DailyMed (NLM) ↗

FDA-Approved Indications (1)

Cystic fibrosisOrphan Designation

Indicated for CF in patients aged 2+ with at least one F508del mutation.

Indications & Usage

1 INDICATIONS AND USAGE TRIKAFTA is indicated for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator ( CFTR ) gene or a mutation in the CFTR gene that is responsive based on clinical and/or in vitro data (see Table 6 ) [see Clinical Pharmacology (12.1) ] . If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to confirm the presence of at least one indicated mutation [see Clinical Pharmacology (12.1) ] . TRIKAFTA is a combination of ivacaftor, a CFTR potentiator, tezacaftor, and elexacaftor indicated for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one F508del mutation in the CFTR gene or a mutation in the CFTR gene that is responsive based on clinical and/or in vitro data. If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to confirm the presence of at least one indicated mutation. ( 1 )

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Trikafta
Vertex Pharmaceuticals
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Medical disclaimer: This information is for educational purposes only. Always consult a qualified healthcare provider before making treatment decisions. Data sourced from FDA and current as of our last update.