TGA clears expanded access to cystic fibrosis therapy following PBAC backing

Cystic Fibrosis Australia and Vertex Pharmaceuticals have welcomed the Therapeutic Goods Administration’s (TGA) approval of Trikafta’s (elexacaftor/tezacaftor/ivacaftor and ivacaftor) expanded use for the treatment of people living with cystic fibrosis (CF).

The updated indication now includes children aged two years and older who have at least one CFTR (cystic fibrosis transmembrane conductance regulator) gene mutation responsive to treatment, either through clinical or in vitro evidence. This broader eligibility comes just weeks after the Pharmaceutical Benefits Advisory Committee (PBAC) recommended listing the new indication on the Pharmaceutical Benefits Scheme (PBS).

“This milestone significantly broadens access to this life-changing therapy for children and adults across the CF community, including those living with ultra-rare mutations,” said Jo Armstrong, CEO of Cystic Fibrosis Australia.

Importantly, the revised Product Information (PI), expected to be available in the coming week, will allow healthcare professionals to consider the use of Trikafta for patients with non-Class I mutations not specifically listed. Use may be considered where the potential benefits outweigh the risks and under close clinical supervision, provided the patient does not harbour two Class I mutations.

Kasia Siwek, Medical Director for Australia and New Zealand at Vertex Pharmaceuticals, said “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.”

Armstrong further added, “As we continue advocating for access to essential treatments, we urge the community to submit consumer comments to the PBAC [by 28 May] in support of Alyftrek ahead of its upcoming review [at the July PBAC meeting].”

Vertex’s Alyftrek combines vanzacaftor, tezacaftor and deutivacaftor. It represents the fifth CFTR modulator in the company’s pipeline, offers a once-daily alternative to Trikafta, and extends therapeutic efficacy in 31 additional mutations.

In two Phase 3 studies involving patients aged 12 and older, Alyftrek was non-inferior to Trikafta in improving lung function (ppFEV1) over 24 weeks. Additionally, Alyftrek outperformed Trikafta on a key secondary endpoint – reduction in sweat chloride levels. For younger patients aged 6 to 11 years, efficacy was extrapolated from these trials and supported by pharmacokinetic data.

CF is a rare, life-shortening genetic disease affecting more 3,800 people in Australia.

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