Cystic Fibrosis: Tauranga boy could soon access Trikafta drug

Tauranga boy Riaan Hamilton, 4, has cystic fibrosis. He could soon access the drug Trikafta.

Tauranga boy Riaan Hamilton spent ‘pretty much 90%’ of last winter on antibiotics. The 4-year-old has cystic fibrosis and has been hospitalised several times. Now, his future looks different after Pharmac announced a ‘game-changing’ proposal to fund the drug, Trikafta, that treats the condition for young children like Riaan.

It has been an “emotional couple of weeks” for the Hamilton family as they think about Riaan living a longer, healthier life.

Riaan Hamilton, 4, has cystic fibrosis – a condition that produces thick and sticky mucus and mainly affects the lungs and digestive system. It is ultimately terminal.

There is no cure for the condition that affects about 500 New Zealanders, but Trikafta treats its underlying cause and makes it more manageable.

Pharmac announced provisional Trikafta funding from April 1 for children aged 2 to 5 with cystic fibrosis.

Pharmac started funding the drug for people aged 6 and up in April 2023. Recipients have said it has helped them walk without struggling to breathe and start new careers.

Pharmac’s Pharmacology and Therapeutics Advisory Committee previously said Trikafta could add 27 years to someone’s life.

Riaan’s mother, Khali Hamilton, said she understood Riaan had the genetic mutation that made him eligible for Trikafta.

Hamilton felt “a bit apprehensive” when she first heard the funding news because it was provisional.

“I was reassured it’s just a process that needs to be done, and then the emotions came that he has the opportunity to add that 27 years onto his life and reduce the risk of [lung] scarring and everything else that comes along with CF when they’re not on Trikafta.”

Hamilton said having Trikafta meant the family would have “less stress and worry”.

“He hates treatment. He gets anxious at the hospital, even if we drive past ... ”

Hamilton previously said Riaan went to hospital every time he had a cough and had to have a suction tube put down his throat.

She said Trikafta would allow her son to live a higher quality of life.

Hamilton said Riaan had returned to daycare after having “such a good summer”.

“He absolutely loves swimming. He’s such a water kid.”

They spent time at the beach and pools.

Tauranga boy Riaan Hamilton.

Riaan now has a younger sister, who turns 1 on February 7.

“Riaan just absolutely adores her, loves having a little sister around.”

Hamilton said Riaan had a “really bad winter” and was on antibiotics “pretty much 90%” of the time.

“He also had grown a specific bug pseudomonas and that ... was two months of, morning and evening, antibiotic nebulisers.”

Hamilton said Trikafta would reduce or remove the need for hospital admissions and treatments.

She planned to submit feedback on Pharmac’s proposal.

Children to gain ‘best possible start in life’

A Cystic Fibrosis New Zealand statement said extending access to Trikafta to younger children meant families could look forward to fewer hospitalisations, improved health, and the possibility of “a long and fruitful future”.

The charity’s executive lead, Simone Brown, said the proposal was “game-changing”.

“It will give our young children living with CF the best possible start in life, and hope to parents who have been nervously awaiting this moment.”

Brown said the organisation knew of at least five families who had moved to Australia or elsewhere overseas to access Trikafta for their young children.

“This proposal will enable those with young children with CF to remain in New Zealand and provide the potential for those who have already left to reunite their families.”

Brown thanked Pharmac and supplier Vertex for providing a range of treatments that reflected individual needs.

“It is very welcome news, and we couldn’t be happier.”

A Pharmac statement on January 22 said its proposal included widening access to Trikafta for everyone with eligible mutations, and funding a new treatment, Alyftrek.

Trikafta had Medsafe regulatory approval for use in people aged 2 and older, and Alyftrek for children aged 6 and older.

About 35 people were expected to benefit in the first year, increasing to 47 people after five years, it said.

Pharmac pharmaceuticals director Adrienne Martin said Trikafta had already changed “hundreds” of lives.

“We are now proposing to fund Trikafta for more people so it can be used as soon as clinically appropriate, regardless of age.”

Martin said cystic fibrosis started to cause harm very early in life.

“Funding these medicines for all age groups would help more young children with cystic fibrosis live longer, healthier lives.”

Martin said funding the treatments would benefit the health system because cystic fibrosis sufferers would visit the hospital less and need less treatment.

Pharmac is seeking feedback on the proposal, which can be submitted online. Consultation closes on February 11.

Megan Wilson is a health and general news reporter for the Bay of Plenty Times and Rotorua Daily Post. She has been a journalist since 2021.