‘An absolute game-changer’: Children under 12 to receive life-saving drug

Hundreds of children with cystic fibrosis will have subsidised access to the life-changing medication Trikafta from May after a year-long campaign to subsidise the drug for people under 12.

The groundbreaking medication was added to the Pharmaceutical Benefits Scheme (PBS) in April last year, but only for those 12 years and over. Federal Health Minister Mark Butler will announce on Thursday the extension of the scheme to include six to 11-year-olds, reducing the cost to parents from nearly $22,000 a month to just $30 a script.

Harry Killoran with mum Sally and sisters Tilly and Sophie. The whole family will benefit from changes to the PBS.

Flavio Brancaleone

“We know Trikafta changes lives and that’s why the government moved as quickly as possible to make this treatment available for young Australians,” Butler said. “Children 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.”

About 2500 children are born each year with cystic fibrosis, an inherited disease affecting cells that produce saliva, sweat, tears and mucus. The mutation causes mucus in the lungs and other parts of the body to become thick and sticky, resulting in persistent infections which over time cause irreversible damage to the lungs, organs and digestive system.

Harry Killoran is one of about 500 children who will benefit from the subsidised medication.

His daily treatment for cystic fibrosis includes taking up to 30 tablets, 20 minutes on a nebuliser and 30 minutes of physiotherapy, but that will change dramatically when he turns six and becomes eligible for Trikafta in August.

‘We’re just so excited that he’ll be able to access this drug, which will change his life.’

Sally, mother of Harry Killoran who has cystic fibrosis

His mother Sally said the medication would help level the playing field for the five-year-old, for whom playing sport is vital for maintaining lung health, and see her son grow to old age.

“He won’t be missing out on play dates and time away from school, and he’ll be able to thrive like every other five-year-old boy,” she said. “We’re just so excited that he’ll be able to access this drug, which will change his life.”

Until the introduction of Trikafta, the life expectancy of people with cystic fibrosis was in the 40s, but studies suggest children with early access to the drug can expect to live into their 70s, according to Bernadette Prentice, a paediatric respiratory physician at Sydney Children’s Hospital.

“Parents who get told their little babies have cystic fibrosis … they’re told this is a life-limiting condition. We don’t have to tell them that anymore,” she said.

“They can expect their children to live long, healthy lives that are unlimited by treatments.

“It is an absolute game changer.”

Trikafta, manufactured by Vertex Pharmaceuticals, works by correcting the abnormal protein responsible for producing the sticky mucus affecting the lungs of cystic fibrosis patients. The corrected protein is sent to the surface of the lungs, allowing normal mucus to be created and the lungs to be cleared.

Patient lung function can improve within a week of taking Trikafta tablets, Prentice said.

Cystic Fibrosis Australia chief executive Jo Armstrong said the past few weeks had been an “emotional roller-coaster” as the Pharmaceutical Benefits Advisory Committee considered adding the drug.

“This has been something that the community has been campaigning for all year, and we’re really delighted that this has been fast-tracked and made available,” she said.

Armstrong said the lobby group would continue to push for therapies for children who are ineligible to receive Trikafta, including those with a rare form of the gene mutation or who don’t respond to the therapy.

“It’s a great step of progress, but we still have a long way to go to make sure that everybody has the right therapies that they need,” she said.

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