Orkambi offers new hope for Cystic Fibrosis sufferers

22 Jul 2020

Posted August 22, 2018

A recommendation to list the Cystic Fibrosis drug Orkambi on the Pharmaceutical Benefits Scheme (PBS) has been welcomed by the Federal Member for Mayo Rebekha Sharkie.

The recommendation is fourth time lucky for sponsors of the drug who have made three unsuccessful attempts to have Orkambi recommended for listing by the PBS Advisory Committee.

“There is still one step to go which is why I used Question Time today to ask Health Minister Greg Hunt when the Government is going to officially list Orkambi,” Rebekha said.

"I was so pleased to hear that the Minister himself had spoken to the company that supplies Orkambi and on the first day of the Advisory Committee's announcement had directed his department to start the listing process.

“This drug has the potential to change the lives of so many older children with Cystic Fibrosis, including many young people in Mayo.”

Minister Hunt told Question Time that it had taken four months to list another Cystic Fibrosis drug, Kalydeco, on the PBS once it was recommended by the Advisory Committee "but we will try to deliver at a faster pace".

Along with other drugs for life-threatening conditions such as breast cancer, the Minister said the Government would "move heaven and earth to do this as quickly as possible because I want this medicine to be in the hands of patients at the earliest possible time".

Rebekha has been advocating for the listing of the drug on behalf of local residents since she came into office in 2016.

She lodged a motion in Federal Parliament in September last year calling on the Government to “expedite” a PBS Advisory Committee review of the Orkambi application and for the Health Minister to intervene in the stand-off with manufacturers over the cost of the drug.

The Government needs to give approval for recommended drugs to be placed on the PBS but it cannot intervene in the assessment decisions of the committee.

Orkambi is a treatment that is proven to improve lung function in older children with Cystic Fibrosis, a genetic condition that affects the lungs and digestive system, but it currently costs up to $250,000 per patient per year.

“The effectiveness and safety of Orkambi are long-standing,” Rebekha said.

“I understand cost was the main reason the PBS Advisory Committee did not recommend adding Orkambi to the PBS in previous applications so I welcome the latest recommendation for approval.”

Stirling father Ed Grue also welcomes the news.

His son Will has Cystic Fibrosis and the family hopes Orkambi will make a difference to Will's quality of life.

“This drug has great potential and it could be a life changer for Will,” Mr Grue said.

“We are really appreciative of what the Federal Parliament is doing and we appreciate Rebekha taking this up for us as well.”

Cystic Fibrosis affects over 3000 people in Australia and over one million people carry the recessive gene that causes the condition.

When a person is diagnosed with Cystic Fibrosis, they are sentenced to a life of frequent hospital stays and ongoing physiotherapy.

Cystic Fibrosis causes a sufferer’s mucus glands to secrete large amounts of mucus which can clog lungs.

Bacteria can become trapped in the lungs, leading to repeated infections and blockages.

It causes irreversible lung damage. Lung transplants are a common measure, but they are not a cure.

Orkambi is a treatment that is proven to improve lung function.

Clinical trials have shown a reduction in lung damage by at levels 40% less than normal sufferers of Cystic Fibrosis.

It reduces instances of chest infection and hospital stays.

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