Australia Approves Trikafta for Patients, 12 and Older, With Common Mutation

Health regulators in Australia have approved Trikafta (elexacaftor/tezacaftor/ivacaftor) to treat people with cystic fibrosis (CF), ages 12 years and older, who have at least one copy of the F508del mutation in the disease-causing gene, Vertex Pharmaceuticals, the therapy’s developer, announced.

“We are delighted” that Trikafta is now open to “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,” Reshma Kewalramani, MD, CEO and president of Vertex, said in a press release.

“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,” Kewalramani added.

Trikafta, a triple combination and oral therapy, is already approved for CF patients 12  and older with at least one copy of the F508del mutation — the most common mutation resulting in CF — in the U.S. and in the European Union, where the therapy is marketed under the brand name Kaftrio.

With the approval issued by the Australian Therapeutic Goods Administration (TGA), the medication will become available in a country where about 3,500 people are affected by CF. Of those, it is estimated that some 750 will be eligible to be treated with Trikafta.

The therapy is a modulator of the CFTR (cystic fibrosis transmembrane conductance regulator) gene, which provides the instructions necessary for cells to make the CFTR protein. This protein is an ion channel, and controls the movement of charged ions across cell membranes.

Specifically, elexacaftor and tezacaftor work as correctors, binding to the faulty CFTR protein to help it fold correctly. This way, cells can shuttle more of it to the membrane instead of degrading the protein. Ivacaftor — also marketed separately as Kalydeco — is a potentiator, meaning its binding to the CFTR protein holds the channel open so that more ions can pass through.

CF “is a condition that significantly affects not only the patient, but also those who care for them, with people living with cystic fibrosis spending multiple hours every day on treatment and requiring daily care from a family member or loved one,” said John Wilson, MD, head of the Cystic Fibrosis Service of Alfred Health in Melbourne.

“The approval of any new treatment option for people living with cystic fibrosis is always welcome news. 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,” Wilson added.

The TGA’s decision was based in part on the results of four global Phase 3 studies, which included various trial sites and patients in Australia.