Aussie kids get free access to life-changing cystic fibrosis drug

From today (February 7), children aged two to five in Australia will have immediate and free access to Kalydeco (ivacaftor) – a life-changing drug that addresses the causes of cystic fibrosis rather than just the symptoms.

Previously, only children six years of age and older with the G551D or other class III gating mutations had subsidized access to Kalydeco, from US biotech firm Vertex Pharmaceuticals (Nasdaq: VRTX).

Without government subsidy, the treatment would cost each patient around A$300,000 ($230,770) per year.

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 Kalydeco treatment, many children can experience an improved quality of life with reductions in respiratory and gastrointestinal complications, improved lung function and fewer hospitalizations. The Turnbull government is making sure that this life-changing drug is no longer out of reach for Australian families that need it.

The announcement today follows a recommendation from the Pharmaceutical Benefits Advisory Committee (PBAC) that subsidized access to Kalydeco should be extended to children aged two to five years old.

Kalydeco is due to be formally added to the Pharmaceutical Benefits Scheme (PBS) on May 1, 2017. Until then, Vertex has agreed to provide the drug free of charge for children aged two to five.

From May 1, 2017, the drug will cost A$6.30 for all concessional patients and A$38.80 for general patients.