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All three medicines are CFTR modulators.

CFTR (cystic fibrosis transmembrane conductance regulator) proteins help control the movement of salt and water in and out of cells. In people with cystic fibrosis, these proteins do not work properly, causing thick, sticky mucus to build up in the body.

These medicines help the faulty CFTR protein work more normally, which makes mucus thinner and less sticky. This helps reduce damage caused by cystic fibrosis and improves overall health.

• Trikafta combines three active ingredients (elexacaftor, tezacaftor and ivacaftor) to treat the underlying cause of cystic fibrosis. It improves lung function, supports weight gain, and reduces the risk of hospitalisation.
• Kalydeco (ivacaftor) works for people with specific mutations by improving how the CFTR protein functions, helping clear mucus and reduce symptoms.
• Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) is a newer CFTR modulator that works in a similar way to Trikafta and provides an additional treatment option for people with eligible mutations.

These medicines do not cure cystic fibrosis, but they treat the underlying cause of the condition and can help people live longer, healthier lives.

Yes. Pharmac is funding these medicines without age‑specific funding restrictions. This means people with eligible mutations can access treatment as soon as it is clinically appropriate, regardless of age.

Trikafta currently has Medsafe approval for use from two years of age, and Alyftrek from six years of age.

If a medicine is prescribed outside its Medsafe‑approved use, this is known as use for an unapproved indication. In these cases, the decision to prescribe is made by the treating clinician in discussion with the patient or their families.

While it remains at a prescriber’s clinical discretion to determine whether a medicine may be appropriate, if they consider it could benefit a person, they are able to prescribe it under Section 25 of the Medicines Act 1981(external link). In these circumstances, the treatment would be funded.

Pharmac heard from people with cystic fibrosis, their families, health professionals and advocacy groups during consultation.

Many people told us that these medicines are life‑changing, and that earlier access is important, especially for children, to help prevent long‑term damage and improve quality of life. There was strong support for removing age restrictions so treatment can start as soon as it is clinically appropriate.

Some people asked for:

• Clearer and fairer access rules
• Eligibility criteria that cover the widest possible range of genetic mutations, including rare ones
• Fewer barriers to getting treatment, particularly for people living outside major centres
• Better information and support for families and clinicians

What we changed in response

• We simplified and aligned the eligibility rules across Trikafta, Kalydeco and Alyftrek, removing criteria that are no longer clinically relevant.
• We updated the criteria to ensure the broadest possible range of eligible mutations is covered and will continue to keep mutation lists up to date.
• We simplified diagnostic requirements, so access is clearer and more consistent across the three medicines.
• We confirmed that applications can be made by any relevant health practitioner, not just specialists.

Some suggestions, such as changing how these medicines are dispensed, could not be made at this time. However, Pharmac will continue working with Health New Zealand to monitor access and consider improvements over time.

We’re grateful to everyone who shared their views. Their feedback helped shape this decision and improve access for people with cystic fibrosis and their families.