The Pharmaceutical Benefits Advisory Committee recommendation of Kalydeco and rejection of Orkambi

Page last updated: 27 April 2017

The Government is listing a breakthrough medicine for the treatment of cystic fibrosis on the Pharmaceutical Benefits Scheme (PBS) from 1 May 2017 for young children.

Cystic fibrosis is the most common life threatening condition affecting young people in Australia.

Extending access to this medicine is important news for Australian families.

Kalydeco (containing ivacaftor) has been subsidised on the PBS since 2014 for the treatment of cystic fibrosis patients aged six years and over, who have a class III (gating) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

In January 2017, the Pharmaceutical Benefits Advisory Committee (PBAC) recommended this listing be extended to allow PBS subsidised treatment to commence at age two. The extended PBS listing for Kalydeco will take effect from 1 May 2017. Until then, Vertex Pharmaceuticals Australia Pty Ltd (Vertex) has agreed to provide the drug free-of-charge to eligible children.

Orkambi (containing lumacaftor with ivacaftor) is another treatment for cystic fibrosis in patients aged 12 years and over. Orkambi is a treatment for a different genetic mutation in the CFTR gene than that which Kalydeco treats. Orkambi not supported for PBS subsidy by the PBAC in March 2016 and November 2016.

While the PBAC acknowledged the clinical benefits of Orkambi in the short-term, the impact of treatment with Orkambi on improvements in long-term lung function and survival was not clear based on the evidence presented by Vertex.

Vertex has lodged a new submission for Orkambi to be considered by the PBAC at its July 2017 meeting. The outcomes from that meeting will be publicly available from 18 August 2017.

The Australian Government cannot list a medicine on the PBS without a positive recommendation from the PBAC – an expert advisory body that is established under legislation and independent from Government.

The PBAC meeting outcomes are publicly available and can be found on the PBS website. A PBAC decision not to recommend listing does not necessarily represent a final PBAC view about the merits of a medicine.