Dialog Box

Rare Mutations Update

Cystic Fibrosis Community Care is pleased to share that Vertex Pharmaceuticals have recently submitted an application to the Australian Therapeutic Goods Administration (TGA) to add a number of non-F508del CFTR mutations that are responsive to Trikafta.

This follows on the footsteps of an announcement in 2023, where Vertex submitted an application to add 200 non-F508del CFTR mutations to the Trikafta license in Europe.

The list of responsive mutations submitted in Australia are not yet known at this stage, but we hope the list will be similar to those involved with the European process.

This marks the start of the process in Australia, and if approved by the TGA, the process then moves to the Pharmaceutical Benefits Advisory Committee (PBAC). Should this result in a positive PBS listing, it will mean an increased number of people with CF could receive a medicine that treats the underlying cause of their illness for the first time.

CFCC welcomes this development and recognises it is an important step in ensuring that no one is left behind.

We will continue to monitor the TGA process and will keep the community informed with any new developments.

08 May 2024
Category: News