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Trikafta
Published
Product name
Trikafta
Active ingredient
Elexacaftor/tezacaftor/ivacaftor, and ivacaftor
Submission type
New chemical entity, New fixed dose combination
Decision
Approved
Decision date
Registration date
What this medicine was approved for
Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) was approved for the following therapeutic use:
Trikafta is indicated for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
How this medicine works
Elexacaftor and tezacaftor are cystic fibrosis transmembrane conductance regulator (CFTR) correctors that bind to different sites on the CFTR protein and have an additive effect in facilitating the cellular processing and trafficking of F508del-CFTR to increase the amount of CFTR protein delivered to the cell surface compared to either molecule alone. Ivacaftor potentiates the channel open probability (or gating) of the CFTR protein at the cell surface.The combined effect of elexacaftor, tezacaftor and ivacaftor is increased quantity and function of F508del-CFTR at the cell surface, resulting in increased CFTR activity as measured by CFTR mediated chloride transport. Clinical outcomes were consistent with in vitro results and indicate that a single F508del mutation is sufficient to result in a significant clinical response (see Clinical Efficacy section of the Product Information).
Why the TGA approved or did not approve this medicine
The decision was based on quality (chemistry and manufacturing), nonclinical (pharmacology and toxicology), clinical (pharmacology, safety and efficacy) and risk management plan information submitted by the sponsor. The benefit-risk profile of Trikafta was considered favourable for the therapeutic use approved.