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Positive PBAC Recommendation for Trikafta and Kalydeco Expansion

At its March 2025 meeting, the Pharmaceutical Benefits Advisory Committee (PBAC) reviewed two proposed extensions to existing cystic fibrosis treatments. Both have been recommended for listing:

Trikafta: Proposed Extension to include children aged 2 years and older with a responsive CFTR gene mutation.

This recommendation has the potential to give 45 children access to a CFTR modulator therapy for the first time. Additionally, approximately 200 individuals currently using Symdeko or Kalydeco may become eligible to transition to Trikafta, offering broader treatment options.

Kalydeco: Proposed Extension to include infants aged 1 to 4 months with at least one CFTR gene mutation responsive to Ivacaftor.

This expansion would enable early intervention for approximately 10 babies each year—providing a crucial opportunity to impact health outcomes in the earliest months of life.

More information here.

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