Kalydeco and Soliris – new treatments for cystic fibrosis and aHUS respectively – will be listed on the Pharmaceutical Benefits Scheme (PBS) from December 1.

Health Minister Peter Dutton said the Government had approved the listings of the medicines which will have significant health benefits for patients suffering these diseases.

Ivacaftor (Kalydeco) is a new treatment for cystic fibrosis in patients with a specific gene mutation and is the first medicine to treat the underlying cause of cystic fibrosis in these patients.

The PBS listing of ivacaftor will help more than 250 Australians affected by the cystic fibrosis G551D gene mutation. 

“The PBS subsidy of this medicine, which would otherwise cost approximately $300,000 a year per patient, will bring great relief to the patients and the families of people affected by this life threatening condition,” Mr Dutton said.

The Government has approved $174.5 million over the next four years to provide ivacaftor on the PBS.

Cystic fibrosis is the most common life threatening recessive genetic condition affecting young people in Australia. It affects organs such as the lungs and pancreas causing irreversible damage and can cause death.

“With this new treatment many patients can experience an improved quality of life with reductions in respiratory and gastrointestinal complications, improved lung function and fewer hospitalisations,” Mr Dutton said.

“Consistent with the advice of the Pharmaceutical Benefits Advisory Committee (PBAC) that this medicine could be listed on the PBS based on a pay-for-performance basis, all cystic fibrosis patients six years and older who have a G551D mutation in the CFTR gene will be treated with ivacaftor for as long as needed.

“I’m pleased to announce that the sponsor of ivacaftor, Vertex Pharmaceuticals, has agreed to proceed with the PBS listing as recommended by the PBAC,” Mr Dutton said.

For more details, go to: http://www.health.gov.au/internet/ministers/publishing.nsf/Content/health-mediarel-yr2014-dutton092.htm