Life-saving drug Orkambi available to Australians living with cystic fibrosis

Melissa Gibson of Mt Helena with her eight-year-old son Toby. Photo: David Baylis
Melissa Gibson of Mt Helena with her eight-year-old son Toby. Photo: David Baylis

THE lives of thousands of Australian children and adults living with cystic fibrosis (CF) will improve following today’s decision to make a revolutionary drug publically available.

Today the Pharmaceutical Benefits Advisory Committee (PBAC) announced a deal with Vertex Pharmaceuticals to make a life-saving drug called Orkambi accessible.

It was the pharmaceutical company’s fourth submission to have the expensive drug listed on the benefits scheme for patients 12 years and older, and its first submission for patients six to 11 years old.

Both submissions were accepted and the announcement was met by celebration from families across the country who had been campaigning for the move.

Prior to Orkambi being on the scheme, it cost $250,000 a year for patients.

Mt Helena parents Melissa and Justin Gibson rejoiced at the news, knowing their eight-year-old son Toby would now have more hope of living a longer life.

Toby, who was born with the rare genetic condition, has two copies of a gene mutation that Orkambi targets.

Mrs Gibson said the availability of the drug would be “life altering” for Toby, allowing him to experience a better quality of life through improved lung function and less hospital admissions, and prevent further lung damage.

Cystic Fibrosis Western Australia (CFWA) chief executive Nigel Barker said the decision was a culmination of years of advocacy by people whose lives had been impacted by CF to force an agreement between the government and Vertex.

“We are ecstatic that the Australian Health System is supporting better health outcomes and quality of life for those with rare diseases, but we’re also concerned that lives have been put on hold and lungs have incurred irreversible damage whilst we struggle to get a fair deal, years behind approval in the USA and Europe,” he said.

“There has to be a better way to fast track the process of drug approval in the future.”

CFWA president Caz Boyd, who was born with two copies of the gene mutation that Orkambi targets and underwent a lung transplant at 27, said the announcement was exciting.

“I am grateful that for a large group of youngsters living with CF their journey will be different to mine; if this drug was available 24 years ago I wouldn’t have needed a lung transplant and witnessed the lives of many childhood CF friends cut short by this insidious disease,” she said.

“Today’s announcement continues to reflect that Australia’s health system remains one of the best in the world.”