While Vertex Pharmaceuticals’ application is still to clear the PBAC hurdle, hope is high for about 130 Australian children.
The Therapeutic Goods Administration has approved the expanded use of Vertex Pharmaceuticals’ Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) cystic fibrosis therapy to include children aged two to five years with at least one F508del mutation in the CFTR gene.
Trikafta was previously approved for use in people with cystic fibrosis six years and older who have at least one F508del mutation.
This latest approval by the TGA was supported by results from a 24-week Phase III open-label study, which evaluated the safety, pharmacokinetics and efficacy of TRIKAFTA® in children aged two to five.
Vertex, a global biotech with reach into Australia, said in its company announcement that the TGA approval would benefit “approximately 130 children living with CF” by treating “for the first time” the underlying causes of their disease.
“We welcome the TGA’s decision to expand the indication of Trikafta to children as young as two years,” said Sabrina Barbic, Vertex’s senior country manager for Australia and New Zealand.
“Trikafta is on the agenda of the March 2024 Pharmaceutical Benefits Advisory Committee meeting, and we remain committed to ensuring all those who can benefit from the medicine can get access to it as quickly as possible.”
