Aussies’ anxious wait for CF wonder drug

ADRUG that treats the underlying cause of cystic fibrosis in some patients is going back to the Pharmaceutical Benefits Advisory Committee for the third time.

Clinical trials in Australia and across the world provide evidence that Orkambi, a new generation drug for cystic fibrosis, improved lung function and reduced exacerbations, hospitalisation and antibiotic use in trial participants.

The drug is designed for those with the cystic fibrosis gene mutation F508del homozygous.

“Australia is a country that prides itself on an equitable and fair health system but sadly the relevant parties, the government’s PBAC and the multinational pharmaceutical company Vertex, have been unable to come to an agreement on costs for this life-changing drug,” the chief executive of Cystic Fibrosis Australia Nettie Burke said.

“We know everyone is doing everything they can at the negotiating table and the Federal Health Minister Greg Hunt in particular has been particularly supportive. But for the sake of so many people’s future we need to close that gap now.”

Where the current treatment arsenal for cystic fibrosis is limited to therapies that treat the symptoms and clinical manifestations of the disease, Orkambi is designed to treat the underlying cause of CF.

The 96-week trial outcomes showed that lung damage was slowed on average by 40%.

Exacerbations reduced by 39%, hospitalisations were reduced by 61% and antibiotic use by 56%.

Orkambi also had a positive flow-on effect on the mental health of people with CF leading to social inclusion.

“The PBAC must realise that Australians with CF need access to life-changing drugs,” Ms Burke said.

“Specifically and importantly in this instance, Orkambi is the first treatment that can prevent or at least slow down permanent, irreversible lung damage for people with CF who have two copies of F508del gene mutation.”