Trikafta for 2-5 year olds
Trikafta is manufactured by Vertex Pharmaceuticals (Vertex) and is currently funded by Pharmac for those aged 6 years and over. In August 2024, Pharmac’s Respiratory Advisory Committee recommended a high priority for funding of Trikafta for 2 to 5 year olds.
Following the Committee’s recommendation, Pharmac advised that the next step for the application was assessment. CFNZ met with Pharmac and Vertex on 14 August 2025 and, at that meeting, Pharmac advised that assessment is underway and the application is on track for the September prioritisation meeting. Prioritisation is the process by which Pharmac compares the costs and benefits of funding one medicine against another.
Pharmac also advised CFNZ that it was struggling to identify the unmet need for this age group. CFNZ has provided further information to Pharmac and asked the CF community to share their experiences to illustrate that need and the impact of CF on their lives. CFNZ will coordinate this information and provide it to Pharmac in advance of the September prioritisation meeting.
The damage caused by CF starts very early in life. Trikafta treats the cause of CF and helps to prevent that damage occurring. The alternative medicines currently available treat only the symptoms of CF and, while they can help to slow decline, they cannot stop it or prevent damage occurring.
Preventing that damage is key to enabling young children with CF to have the opportunity to lead long and healthy lives. CFNZ urges Pharmac to ensure that its assessment and prioritisation process reflects the full benefit Trikafta would bring and makes an early decision to fund.
ALYFTREK – New Zealand
Vertex applied to Medsafe in July 2024 for approval of the use of vanzacaftor/tezacaftor/deutivacaftor (ALYFTREK) a once daily CFTR modulator therapy for people with CF aged six years and older who have at least one F508del mutation or other responsive mutation.
Vertex applied to Pharmac in November 2024 for funding of ALYFTREK. Pharmac’s Pharmacology and Therapeutics Advisory Committee (PTAC) considered ALYFTREK at its May 2025 meeting and its provisional recommendations have now been released. These are:
ALYFTREK adds a further 31 mutations to those already responsive to a CFTR modulator. Having another modulator available in New Zealand would ensure that all those with CF for whom there is an effective modulator have access to one. CFNZ asks that Pharmac and Vertex do all they can to make ALYFTREK available in New Zealand as soon as possible.
ALYTREK – Australia
Australia’s Pharmaceutical Benefits Committee (PBAC) considered the funding of ALYFTREK for those aged 6 years and older at its meeting in July 2025. PBAC deferred its recommendation on ALYFTREK pending as the Therapeutic Goods Administration (TGA) process is not yet complete. The TGA regulates the use of medicines in Australia.
Definition of a Rare Disorder
On 11 August 2025, Pharmac opened consultation on the definition of a rare disorder. Pharmac’s current definition is a disorder which affects less than 1 in 50,000 people in New Zealand. This current definition excludes CF and is also out of step with overseas’ jurisdictions and the New Zealand Rare Disorders Strategy.
Pharmac proposes that the definition be amended to define a rare disorder as one which affects less than 1 in 2,000 people in New Zealand. This would then include CF. CFNZ has made a submission to Pharmac supporting the change to 1 in 2,000 people but requesting that the wording of the definition is consistent with the definition in the Rare Disorders Strategy.
