5 October 2018

Hundreds of Australian children living with cystic fibrosis (CF) now have greater access to a life-changing medication, thanks to an international collaboration in clinical trials, supported by Children’s Health Queensland’s respiratory physician Professor Claire Wainwright.

From 1 October 2018, Orkambi® became available on the Pharmaceutical Benefits Scheme (PBS).

CF is a life-long genetic condition that causes the body to produce abnormal amounts of thick, sticky mucous within the lungs, airways and digestive system. This leads to impairment of the digestive functions of the pancreas and traps bacteria in the lungs resulting in recurrent infections, and irreversible damage. There is currently no cure.

Clinical trials have shown that Orkambi® can slow the rate of lung function decline and reduce the number of infections in patients with CF – who have two copies of the F508del mutation – the most common form of CF in Australia. With the support of the Children’s Hospital Foundation, Prof Wainwright and her team continue to undertake research into new therapeutic approaches to improve the health outcomes of children with CF.

“It’s very exciting that children with CF – who carry two copies of the F508del CTFR mutation in Australia will now have access to Orkambi®. This medicine can reduce the frequency of chesty episodes, and reduce the need for hospital admission.

It also slows down the deterioration in the function of the lungs that is seen in people with CF over time, which is a great opportunity to see improved outcomes for children with CF,” Professor Claire Wainwright said.

It is predicted that around 200 children with CF in Queensland, and approximately the same number of adults, will now have access to this potentially life-changing medicine.

Each year around 80 babies with Cystic Fibrosis are born in Australia, about half have two copies of the f508del mutation.

Professor Wainwright’s research Fellowship was funded by the Children’s Hospital Foundation.

For more information about the PBS listing of Orkambi, see here.