Trikafta for 2-5 year olds
Trikafta is manufactured by Vertex Pharmaceuticals (Vertex) and is currently funded by Pharmac for those people with CF aged six years and over with responsive mutations. In early 2024, Vertex applied to Medsafe and Pharmac for the approval and funding of Trikafta in New Zealand for those with CF aged two years and older who carry a genetic mutation responsive to the medicine.
Pharmac’s Respiratory Advisory Committee has now recommended a high priority for funding of Trikafta for this age group.
The Committee considered that the CF disease trajectory is generally set before the age of six and there is no biologic or evidence based reason to commence treatment at six years. The Committee also 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.
CFNZ is delighted that the Respiratory Committee has recognised that the best way to prevent deterioration and long term decline is to intervene early. Investing in Trikafta at this early age will give children with CF the greatest opportunity to stay well and live normal lives. It will also provide benefits across the health system though reduced treatments, hospital care and surgery. There will also be wider societal benefits through reducing the burden of care on parents and caregivers, reducing lost school time, and providing the potential for parents and caregivers to work.
Pharmac advises that the next step for the application is assessment. CFNZ would like to see this step take as little time as possible. Trikafta has already been subject to a comprehensive assessment process prior to funding for those aged six years and over. The extension to those aged two to five years of age should therefore be a simple incremental process completed very quickly.
Investing in medicines such as Trikafta that help to prevent long term damage and reduce the burden of ill-health on individuals and society are a core part of this Government’s reset for Pharmac.
Making Trikafta available to those aged two to five years therefore needs to happen as soon as practically possible to maximise these benefits.
You can read the minutes of Pharmac’s Respiratory Advisory Committee here:
https://pharmac.govt.nz/assets/2024-08-28-Respiratory-Advisory-Committee-Record-Web-final.pdf
Access to Kalydeco
Kalydeco is currently funded by Pharmac for those people with CF who carry one or more of nine specific CF mutations. In addition to carrying two CF mutations, the criteria require that an individual must also have a sweat chloride level of at least 60mmol/L. However, this is not the case for access to Trikafta.
As part of its consideration of the extension of Trikafta to those aged two to five years, 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 two cystic fibrosis causing mutations and a sweat chloride level of at least 60mmol/L. Pharmac has yet to accept this recommendation.
An application was made in July 2021 to widen access to Kalydeco from the existing nine CF mutations. In April 2022, the application was given a high priority for funding by Pharmac’s Respiratory Advisory Committee which recommended that access be widened to include those mutations approved as responsive by the US FDA, as is the case for Trikafta.
Pharmac has advised CFNZ that the application is still awaiting assessment.
Many of the people who would previously have benefited from a widening of access to Kalydeco are now able to access Trikafta. However, access to Kalydeco still needs to be widened to enable those who are unable to access or to tolerate Trikafta to have a suitable CFTR modulator therapy.
IN CFNZ’s view, waiting three years for an assessment to widen access to a medicine that is already funded by Pharmac is unacceptable. As with the recommended expansion of access to Trikafta for those aged two to five years, it should be a simple and rapid incremental process.
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.
Consent for the use of ALYFTREK by Medsafe may provide an option for some Kiwis with rare CF mutations for whom no other CFTR modulator therapies are available. CFNZ has written to Medsafe seeking priority for consideration of the application to enable its use in New Zealand as soon as practically possible.
Access to CFTR Modulators in Australia
Australia’s Pharmaceutical Benefits Advisory Committee (PBAC) will be considering two applications at its March 2025 meeting to widen access to CFTR modulator therapies.
PBAC will consider the extension of Trikafta to those people with CF aged two years or older with responsive mutations, and the extension of Kalydeco for those aged one to four months.
OTHER DEVELOPEMENTS
For those unable to benefit from CFTR modulator therapies, a Phase 1b trial is underway to assess the effectiveness of Recode Therapeutics’ inhaled therapy for people with CF. The trial is being carried out in the United States and Europe.
The inhaled therapy, RCT 2100, is expected to provide lung cells with a healthy version of CFTR messenger RNA (mRNA) to allow the cells to produce a functional version of CFTR, regardless of the CF-causing mutation.
You can read more about the trial of RCT2100 here:
https://cysticfibrosisnewstoday.com/news/first-us-patient-dosed-phase-1b-trial-cf-therapy-rct2100/
Vertex is also progressing the development of an inhaled mRNA therapy for those people who are unable to benefit from CFTR modulator therapies. The multiple ascending dose phase of the Phase 1 / 2 trial is underway with data expected during the first half of 2025.
You can read Vertex’s original release about VX-522 here:
