Cystic fibrosis sufferer Natalie Wineti has been taking Trikafta for three months. Photo / Andrew Warner
Renee Smith can now enjoy “a lot more days on this Earth” as the “life-changing” drug Trikafta became publicly available in New Zealand on Saturday.
The 38-year-old says she is looking forward to no longer having coughing attacks, walking without becoming breathless easily and waking up feeling energetic.
“It gives us a longer life ... a life where we don’t feel like we have to breathe through a bloody straw.”
Originally from Rotorua, Smith and her twin sister Natalie Wineti have cystic fibrosis - an ultimately terminal condition that produces thick and sticky mucous and mainly affects a person’s lungs and digestive system.
Trikafta treats its underlying cause, but at $330,000 per year, the drug has been out of reach for many families as it has previously not been publicly funded in New Zealand.
In the past year, several Bay of Plenty families have spoken out about the need for Trikafta to be publicly funded, including Rotorua mother Trish Daniels, who feared her 19-year-old son would not make it to his 21st birthday without it, and Tauranga father Glenn Ford, who was considering moving to where it is funded overseas.
In December, Pharmac announced funding for Trikafta was proposed to start on April 1.
In March, Pharmac confirmed the funding of Trikafta for people with cystic fibrosis, aged six and above, who met certain eligibility criteria.
Smith, who lives in Wellington with her partner, said she was “really happy” when she found out the “life-changing” drug would be funded.
Smith, a drug and alcohol counsellor, said having cystic fibrosis was “really challenging”.
She would become breathless “very easily” and it was a struggle to walk to the bus stop in the mornings. She also could not walk far without having “a coughing attack”.
Smith said she and her partner loved going on bush walks, but due to her condition, she had to walk slowly and rest a lot.
“I know it [Trikafta] is going to be helpful for me, because then I can enjoy those long walks without being tired.”
She said the drug would allow her to have “a lot more days on this Earth”, and her goal was to have a family of her own.
In October, Natalie Wineti told the Rotorua Daily Post she was considering moving to Australia, where the drug was funded.
However, just before Christmas, Wineti found out she had been approved for Trikafta through a managed access programme with the drug’s manufacturer and supplier Vertex.
After driving to Waikato Hospital to pick it up, “I couldn’t believe that I finally had the miracle drug in my hand”.
Wineti said she started “purging” within the first five hours of taking her first dose, “completely emptying the lungs”.
But within three days, she went from having a “persistent cough” to “no cough at all”.
“I can walk and run and breathe like a normal person.”
Wineti said she had “so much more energy” and was no longer waking up with severe migraines, joint pain and breathing difficulties.
Asked what her future looked like, Wineti said being able to stay in New Zealand was the “biggest relief”. She is married with a 10-year-old son and two stepchildren.
“I’m happy in my job. I don’t have to worry about uprooting my whānau to make that move to Australia ... we can just stay put.”
She said she was “absolutely over the moon” for the cystic fibrosis community, as they could finally access the drug and have a “quality of life”.
“I never thought that I would be able to live the life I’m living now in terms of my health ... I’m thriving.”
Smith said she cried “tears of joy” when Wineti told her she was approved for Trikafta. However, she had never applied for the managed access programme as she understood she would have to be “really, really unwell” to be eligible for it.
Whakatāne brothers Ashley and Troy Watson shared a similar story in April 2022, as Ashley was eligible for the programme but Troy was not.
In November, Troy was also approved for the programme.
Since starting on Trikafta, Troy said he had noticed a “huge improvement” in his health.
“I think the whole cystic fibrosis community ... will benefit greatly from it [Trikafta].”
Cystic Fibrosis New Zealand chief executive Lisa Burns said funding Trikafta was “nothing short of momentous” and it was a “significant milestone” for those who were eligible.
“With a current life expectancy of 31 in New Zealand, Trikafta could give people with cystic fibrosis benefits equivalent to 27 more years, which opens opportunities for a future that they never thought would be possible.”
Burns said it was “delighted” New Zealand was one of the few countries in the world with “such extensive access to Trikafta, which includes rare mutations”.
The priority was to ensure all people with cystic fibrosis who could benefit from modulator therapy had access to one that worked for them.
“This is so important for our community, to ensure equity of access and that no one is left behind.
“Our cystic fibrosis community won’t be able to just take a tablet and walk away. In many cases, they’ll need to maintain existing treatments including exercise, and they’ll need ongoing support as they transition through some big life changes.”
Pharmac’s director of operations Lisa Williams said the agency expected about 370 people to be eligible for funded access to Trikafta.
“Trikafta has not been shown to work for all people with cystic fibrosis, and the eligibility criteria target funded treatment to those that it will work for.”
Pharmac also funded another cystic fibrosis treatment, ivacaftor (Kalydeco), which was used by about 7 to 8 per cent of people with cystic fibrosis.
“Some of these people will change to Trikafta, but others will remain on Kalydeco as Trikafta won’t work for their specific gene mutations.”
This meant about 90 per cent of people with cystic fibrosis in New Zealand will have mutations responsive to these targeted therapies, she said.
“Having a medicine with this level of investment secured for our communities is a big deal, and we’re grateful to everyone who has been a part of this process.”
In a media release on March 7, Williams acknowledged the time people had put into advocating for those living with cystic fibrosis and those who had shared their personal stories.
“We have heard them all, and we know there will be a lot of people relieved to hear that we are funding Trikafta.”
According to the Pharmac website, prescribers will need to apply for a special authority on behalf of people diagnosed with cystic fibrosis.
Prescribers will need to confirm the person meets six certain access criteria, including the patient having either two cystic fibrosis-causing mutations in the cystic fibrosis transmembrane regulator gene or a sweat chloride value of at least 60 millimoles per litre.
Once an application has been approved, people can go to any Te Whatu Ora - Health New Zealand inpatient hospital pharmacy to collect Trikafta.
Vertex Australia and New Zealand senior country manager Sabrina Barbic said it was “delighted” eligible New Zealanders with cystic fibrosis could access Trikafta on the pharmaceutical schedule from April 1.
Patients who were on Trikafta through Vertex’s managed access programme would continue to receive their medicine until this date. From then, their healthcare practitioner would work with them to transition them to Pharmac’s supply with no break from treatment.
The 19-year-old disappeared without her wallet or mobile phone.