Trikafta for 2-5 year olds
Pharmac’s Respiratory Advisory Committee has recommended a high priority for funding of Trikafta to those people with CF aged two to five years of age with responsive mutations. Trikafta is manufactured by Vertex Pharmaceuticals (Vertex) and is currently funded by Pharmac for those aged 6 years and over.
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. The next step for the application is assessment.
You can read the minutes of the Respiratory Advisory Committee here:
https://pharmac.govt.nz/assets/2024-08-28-Respiratory-Advisory-Committee-Record-Web-final.pdf
Kalydeco
Pharmac’s current criteria for access to Kalydeco requires that those with cystic fibrosis causing mutation(s) must also have a sweat chloride level of at least 60mmol/L. However, this is not the case for access to Trikafta.
Pharmac’s Respiratory Advisory Committee has recommended a change to the criteria for access to Kalydeco to align with that for Trikafta. This would mean that there would no longer be a requirement to have both cystic fibrosis causing mutations and a sweat chloride level of at least 60mmol/L. Pharmac has yet to accept this recommendation.
There has been no progress to date with the application to widen access to Kalydeco to include mutations approved as responsive by the US FDA, as is the case for Trikafta. The application was given a high priority by the Respiratory Advisory Committee in April 2022 but still awaits assessment.
Expanded Access to Trikafta
Vertex has announced that the US Food & Drug Administration (FDA) has approved expanded use of Trikafta for people with CF aged two years and over with at least one F508del or another responsive CFTR mutation. This approval adds an additional 94 non-F508del mutations to the approved list.
Pharmac’s criteria for access to Trikafta in New Zealand is linked to the FDA approved mutation list, meaning expanded eligibility for Kiwis with CF aged six years and older.
You can read the Vertex release, including the full list of additional mutations, here:
https://news.vrtx.com/news-releases/news-release-details/vertex-announces-us-fda-approval-trikafta
ALYFTREK – once daily CFTR modulator for CF
Vertex has announced that the US FDA has approved 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 approval provides for an additional 31 mutations not responsive to other CFTR modulator therapies.
You can read the Vertex release here:
ALYFTREK in New Zealand
Vertex has applied to Medsafe for approval of the use of ALYFTREK in New Zealand. The application is currently undergoing initial evaluation. CFNZ has written to Medsafe seeking priority for consideration of the application.
