Life-changing treatment approved for CF kids aged 6-11

A life-changing treatment is now available for hundreds of more children living with cystic fibrosis, following the Australian Therapeutic Goods Administration’s (TGA) approval of Trikafta for those aged 6-11 years old. 

Trikafta is a cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy, a treatment designed to correct the malfunctioning protein made by the CFTR gene. It works by improving the flow of chloride and water in these patients, as well as helping improve lung function and breathing. 

The treatment has been available for Australians aged 12 years and older since March 2021 and in April this year, it was listed on the Pharmaceutical Benefits Scheme (PBS) for that age group.

Dr Hiran Selvadurai, Head of Respiratory Medicine at The Children’s Hospital at Westmead (CHW), has seen a significant improvement in the older children who have received the life-changing therapy. 

 “The initial data has been promising and in the older children, there have been astonishing changes immediately. We’ve seen children not being admitted to hospital nor needing antibiotics anywhere near as frequently as they used to, and their lung function, growth, nutrition, mental health and wellbeing have all improved,” Dr Selvadurai said. 

“Their whole quality of life has improved significantly. These children are suddenly dreaming of a career and a future, whereas previously they were hindered by the fear of what CF would ultimately lead to. 

“Things are changing. CF is changing. The face of CF is changing. We’re all so very excited about it.” 

Now Trifakta is available for children aged five years and older, Dr Selvadurai said earlier intervention will help reduce the long-term impact of CF by preventing lung disease before scarring and inflammation takes hold. 

With this approval, about 500 children living with CF, like eight-year-old Liam, are now eligible for Trikafta. 

Diagnosed with CF, Liam has been seen by Dr Selvadurai at CHW since he was a month old. He takes tablets and has injections daily, with his family also making the return trip from Yerrinbool to Westmead at least once a month for treatment. 

For his parents, Sarah-Jean and Daniel, this life-changing treatment has given them new hope for his future.

“Liam has been through a great deal as you can imagine. It’s so exciting, we’re so very hopeful for Liam,” Sarah-Jean said. 

CF is the most common life-shortening genetic condition, with the median life expectancy significantly lower than the average Australian at 47 years old.  

But with this therapy, Dr Selvadurai said the hope is for these children to now live long and healthy lives. 

“We now expect our children will well and truly live into late adulthood; they won't die from CF but with CF. I love the implications of that ambition,” Dr Selvadurai said. 

“These children are going to live into late adulthood and have aspects of old age, but not the lung diseases that for many generations we have worried about with CF.”