Vertex receives TGA Approval for Expanded Indication of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for People with Cystic Fibrosis

Vertex Pharmaceuticals today announced that the Australian Therapeutic Goods Administration (TGA) has approved the expanded use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of people with cystic fibrosis (CF).

With this approval, the indication has been expanded to include patients aged 2 years and older who meet the diagnostic criteria for CF and who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive based on clinical or in vitro evidence.

There are over 2,000 known CFTR gene mutations, about half of which cause cystic fibrosis[ii]. Today’s approval extends the medicine’s indication to include an additional 271 mutations[iii].

We welcome the TGA’s decision to expand the indication of TRIKAFTA to include people living with CF with a responsive mutation, including those with ultra rare mutations. It marks further progress towards our mission of providing a treatment for all people living with CF regardless of age or genotype,” said Kasia Siwek, Medical Director, Australia and New Zealand, Vertex Pharmaceuticals.

The indication expansion was supported by a combination of clinical data, real world evidence and in vitro data.

PBS Information: TRIKAFTA is available on the Pharmaceutical Benefits Scheme (PBS) for patients aged 2 years and older with at least one F508del mutation in the CFTR gene. The medicine is not currently funded for those with other ‘responsive mutations’ as per this new indication. Refer to PBS Schedule for full authority information.

[i] Data on file based on April 2025 analysis of ACFDR 2023 data

[ii] CFTR2 database https://cftr2.org/welcome

[iii] TRIKAFTA Product Information. 8 May 2025