Dialog Box

Breaking News | PBAC Recommendation

Breaking News

We are delighted to announce that the Pharmaceutical Benefits Advisory Committee (PBAC) has today recommended Kalydeco® for children aged 4 to 12 months with a gating (class III) CFTR gene mutation.

The PBAC has also recommended that access to Kalydeco® be extended to include an additional 92 CFTR gene mutations.

There are approximately 100 people in Australia that will benefit from this decision.

The recommendation now heads to the Federal Government for review and a decision as to when it will be added to the PBS.

We know that people living with CF cannot wait any longer, and we urge all stakeholders to not delay this important listing on the PBS.

The sooner Kalydeco® is available, the sooner benefits can be realised for some of the youngest people with CF, and for those living with rare gene mutations who currently cannot access a modulator therapy.

Thank you to all of our community who advocated for this important change.

Today we celebrate this great news, but we remain resolute to keep moving forward and advocating for all people with CF to have access to effective treatments.

CFTR gene mutations that produce CFTR protein and are responsible for ivacaftor.

About CF and Kalydeco®

CF is caused by changes in the CFTR gene, a gene that is responsible for building a protein which controls the movement of salt in and out of cells. These changes can be thought of as ‘spelling mistakes’ in the CFTR gene, which impacts the gene’s ability to function and create the CFTR protein. 

Kalydeco® works by binding to the defective CFTR protein at the surface and helps the movement of chloride and water in and out of the cells, thinning the mucus in the lungs and other organs. Around 9.5% of the CF population in Australia currently have access to this medication, with this potential listing looking to expand eligibility beyond gating mutations to include those who have conductance and other residual function mutations in the CFTR gene.

This medication was originally listed on the Pharmaceutical Benefits Scheme (PBS) in Australia in 2014. Access was more recently expanded to include children aged 12 – 24 months in August 2019.


11 December 2023
Category: News