Advocacy Update, April 2025

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 to those with CF aged two to five years of age with responsive mutations.

The Committee considered that commencing Trikafta in children aged two to five years of age would be the best strategy to prevent deterioration, preserve normal lung function, and prevent any meaningful decline.

Following the Committee’s recommendation, Pharmac advised that the next step for the application was assessment but no indication was given as to when that assessment would be undertaken. Pharmac’s Application Tracker currently shows the status of the application as “working to compare options”. In March 2025, Medsafe approved the use of Trikafta for this age group.

Given the Medsafe approval and the high priority funding recommendation, CFNZ urges Pharmac and Vertex to act without delay to make Trikafta available to this young age group and give the more than 50 children who would be eligible the benefit of this life-changing medicine.

ALYFTREK – once daily CFTR modulator for CF

Vertex has applied to Medsafe for approval of the use of vanzacaftor/tezacaftor/deutivacaftor (ALYFTREK) a once daily CFTR modulator therapy for those people with CF aged six years and older who have at least one F508del mutation or other responsive mutation. The application is currently undergoing initial evaluation.

Vertex applied to Pharmac in November 2024 for funding of ALYFTREK. The current status of the application on Pharmac’s Application Tracker is “seeking clinical advice”. Alyftrek is currently an agenda item for the May Pharmacology and Therapeutics Advisory Committee (PTAC) meeting.

The approval and funding of ALYFTREK in New Zealand would widen eligibility for access to a medicine treating the cause of CF by the addition of a further 31 mutations not responsive to other CFTR modulator therapies.

Australia’s Pharmaceutical Benefits Committee (PBAC) will consider the funding of ALYFTREK for those aged 6 years and older at its meeting in July.