Vertex secures TGA approval for young children with cystic fibrosis, while next-gen treatment on the horizon

Pharma News: Vertex Pharmaceuticals has received approval from the Therapeutic Goods Administration (TGA) to extend the use of Trikafta to include children aged 2 -5 with cystic fibrosis (CF) with at least one F508del mutation. This expansion awaits consideration on the Pharmaceutical Benefits Advisory Committee (PBAC) agenda for March 2024. Simultaneously, Vertex’s next-generation CF treatment is offering a promising horizon for advancing CF care.

Cystic fibrosis, a condition typically diagnosed at birth, leads to a progressive decline in health and premature death. Paediatric patients often exhibit early structural lung damage, even before the onset of recognisable respiratory symptoms.

Sabrina Barbic, Senior Country Manager for Australia and New Zealand at Vertex Pharmaceuticals, expressed enthusiasm for the expanded approval, stating, “We welcome the TGA’s decision to expand the indication of Trikafta to children as young as 2 years. Trikafta is on the agenda of the March 2024 PBAC meeting, and we remain committed to ensuring all those who can benefit from the medicine can get access to it as quickly as possible.”

Jo Armstrong, CEO of Cystic Fibrosis Australia, emphasised the importance of patient voices, urging individuals to contribute comments to the PBAC. She stated, “Act now to ensure your voice is heard in the extension process for Trikafta. Every comment counts, and your contribution can make a significant difference. Your voice carries the weight of collective advocacy, contributing to positive outcomes for our community.”

Looking ahead, Vertex is on the brink of introducing a next-gen CF treatment named Vanza, a once-daily triple CFTR modulator regimen consisting of vanzacaftor, tezacaftor and deutivacaftor. The promising data supporting Vanza’s efficacy comes from two trials, SKYLINE 102 and SKYLINE 103, comparing it head-to-head against Trikafta among CF patients aged 12 and above.

Notably, Vanza demonstrated noninferiority* to Trikafta, while showing superiority in the proportion of patients with sweat chloride (SwCl) below 60 mmol/L (the diagnostic threshold for CF) and below 30 mmol/L (the carrier level threshold), compared to Trikafta.

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*Absolute change from baseline in percent predicted forced expiratory volume in one second through week 24