Dunedin parents Tom and Lara Connor said the drug Trikafta, used to treat cystic fibrosis, meant their 9-year-old son Emerson could enjoy riding his bike, just like other children his age.
The couple are urging the government to expand funding so Emerson’s baby sister Aria, who also has cystic fibrosis, can receive the life-changing treatment sooner.
Since 2023, the government has approved and funded Trikafta in cystic fibrosis patients aged 6 years and older.
Last week, Medsafe granted consent for the drug’s use for children as young as 2, making 55 more patients eligible for the drug, and the government's drug -buying agency Pharmac is assessing a proposal to fund the drug’s expanded use.
Cystic fibrosis is a life-threatening genetic disorder that causes thick mucus to build up in the lungs, pancreas and other organs.
Without funding, a year of Trikafta treatment for one person could cost about $330,000.
Mr Connor said it had been a stressful wait for Emerson’s 6th birthday, after he was diagnosed with cystic fibrosis as a newborn, as was his 7-month-old sister Aria.
People with cystic fibrosis were more vulnerable to illness — a cold could become a hospital visit — and before Emerson turned 6, the family were unable to do many social activities.
"We could hear a cough from 100 metres away," Mr Connor said.
"[Emerson] felt left out, probably on a few things, but it's just about keeping him safe."
Emerson had often experienced severe fatigue and became exhausted by late afternoon, but after two days on Trikafta, "he was up with us on the street riding his bike at 6.30 at night", Mr Connor said.
"It was pretty life-changing for him."
While risks remained, the family were able to socialise more, and on Saturday they watched Emerson take part in a sailing event — possible only because of Trikafta, Mr Connor said.
"Emerson really enjoys social things.
"If it wasn't funded, it would definitely be hard to get him there ... he wouldn't handle the day and now he can."
The family hoped the government would extend Trikafta funding so Aria could receive the same benefits when she turned 2.
"There's still definitely side effects of Trikafta that affect Emerson, but in terms of his energy and everything, if we can get Aria on to the smaller doses of Trikafta, it's going to really improve how she feels, I have no doubt," Mr Connor said.
Seven-month-old Maya Palmer had also been diagnosed with cystic fibrosis as a newborn.
Her parents, Maddie and Mackenzie Palmer, of Dunedin, had considered moving the family to Australia, where the drug was funded for patients from 2 years old, but now hoped a funding change would allow them to stay in New Zealand.
"[At the moment] we have to do everything we can to protect Maya from getting sick for six years, which kind of means isolating her.
"To be able to have her treated from 2 years old means she can live a more carefree, actual childhood."
Maya had already been admitted to hospital with lung infections and while Trikafta was a relatively new drug, research had shown it increased patients’ quality of life and reduced the possibility of needing a lung transplant, Mrs Palmer said.
"To be able to save that damage in the long term would be literally life changing for Maya."
Last year, Pharmac’s respiratory advisory committee recommended the drug be funded.
The organisation will compare the application with other funding options and make a decision following consultation.
