The move is expected to benefit more than 300 children each year and save families hundreds of thousands of dollars.
The Pharmaceutical Benefits Scheme (PBS) listing for elexacaftor/tezacaftor/ivacaftor and ivacaftor (Trikafta) has been expanded to include younger children.
Federal health minister Mark Butler made the announcement this week, saying the listing had been expanded to include children aged two to five years who had at least one F508del mutation on the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
This is the most common CF-causing mutation worldwide. In Australia, a baby is born with cystic fibrosis every four days.
The drug was first expanded by the Albanese Government to treat children aged six to 11 years on 1 May 2023. The inclusion of 2- to 5-year-olds will provide for earlier access to this life-changing treatment for children living with cystic fibrosis.
It is expected the listing will benefit an additional 330 children each year. Without subsidy, Trikafta might cost families more than $250,000 per year. On the PBS, they will pay no more than $31.60 per prescription, or $7.70 if they have a concession card.
“Trikafta is a life changing drug for children living with cystic fibrosis,” said Mr Butler.
“This treatment was far too expensive for most families to contemplate. Now, thanks to our world-leading PBS system, families with a diagnosis of CF can literally breathe easier.
“For kids who reap the full benefit of these treatments, they will be able to live and play, and plan for long, happy lives, just like their friends who weren’t born with a faulty gene.
“I want to acknowledge all those parents and families that have campaigned and called for this. Your advocacy and your voices have been heard, loud and clear.”
The decision was welcomed by Cystic Fibrosis Australia’s CEO Jo Armstrong, who said they had been campaigning tirelessly for Trikafta to be made available and subsidised for younger kids, so that the families can afford it.
“Today we have the great news that the life changing medication Trikafta will be made available from 1 August for children aged two to five,” she said.
“A huge thank you to Minister Butler and the Department of Health, and Vertex Pharmaceuticals for making this available.
“As a community we can celebrate: our voices have been heard and another 330 Aussie kids will now have the opportunity to live healthier, longer lives. It’s not a cure, but it’s a great step in the right direction.”
Dr Andrew Tai, paediatric respiratory and sleep medicine physician and head of department at the Women’s and Children’s Hospital Adelaide, also said it was welcome news.
“Given it is a progressive disease, it is important to treat children with CF as early in life as possible,” he said.
“With this expanded reimbursement of Trikafta, we can now treat more children as young as two years with a medicine that addresses the underlying cause of the disease. This is very welcome news for these children, their families and the CF community.”
In a statement, Vertex Pharmaceuticals said the latest reimbursement would result in more than 90% of the 3700 people living with CF in Australia qualifying for reimbursed access to a medicine that treats the underlying cause of their disease.
“It marks further progress towards our mission of providing a treatment for all people living with CF regardless of age or genotype,” said Sabrina Barbic, the company’s senior country manager, Australia and New Zealand.
Trikafta has been available on the PBS since 1 April 2022 for the treatment of CF in people aged 12 years and older who have at least one F508del mutation in the CFTR gene. It was approved by the Australian Therapeutic Goods Administration (TGA) for those aged two to five years in January this year.
