Vertex Pharmaceuticals has welcomed the TGA's expanded approval of TRIKAFTA (elexacaftor, tezacaftor, ivacaftor and ivacaftor) to include more people living with cystic fibrosis (CF).
Vertex said the approval means TRIKAFTA can be used to treat patients aged two 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.
Over 2,000 known CFTR gene mutations exist, about half of which cause cystic fibrosis. Today’s approval extends the medicine’s indication to include an additional 271 mutations.
“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, the medical director of Vertex Pharmaceuticals, Australia and New Zealand.
CF Together said the broad listing is testament to the "power of advocacy" and "the Australian medicine approval process becoming more flexible."
At its March 2025 meeting, the Pharmaceutical Benefits Advisory Committee recommended the indication for reimbursement via the PBS.
"CF Together welcomes this news for the more than 200 Australians who will now become eligible for TRIKAFTA for the first time. We know that fast access is vital; reducing infections, hospitalisations and increasing quality of life, and we urge all stakeholders to expedite this important listing on the PBS," said the organisation.
