Australian TGA approves Vertex’s Trikafta for cystic fibrosis treatment

The Australian Therapeutic Goods Administration (TGA) has approved the use of Vertex Pharmaceutical’s Trikafta (elexacaftor, tezacaftor, ivacaftor and ivacaftor) for people with cystic fibrosis (CF) aged 12 years and above.

Trikafta is indicated for individuals with at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

A progressive, multi-system disease, CF affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract.

It is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. The majority of people with CF have at least one F508del mutation.

The latest development is based on the results of four global phase III studies in various trial sites and patients from Australia.

Vertex CEO and president Reshma Kewalramani said: “We are delighted the TGA has approved TRIKAFTA for eligible people living with CF in Australia and will continue working with the Australian Government to bring this important medicine to patients as quickly as possible.

“It is our goal to develop and provide treatments for all people with CF around the world and today is another significant milestone on that journey.”

With this approval, approximately 750 individuals living with CF in Australia will be eligible for CFTR modulator therapy.

Alfred Health Cystic Fibrosis Service head professor John Wilson said: “Cystic fibrosis is a complex, progressive, devastating disease that causes severe damage to the lungs, digestive system and other organs in the body.”

“This new treatment is for patients ages 12 years and older with at least one F508del mutation and means more patients can potentially benefit from a medicine that targets the underlying cause of the disease for the first time.”

In January, the US Food and Drug Administration (FDA) accepted Vertex’s supplemental New Drug Application (sNDA) to expand the use of Trikafta for children aged six to 11 years old with at least one F508del mutation in the CFTR gene.