Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the Australian Therapeutic Goods Administration (TGA) has approved the use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for people with cystic fibrosis (CF) ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the most common CF-causing mutation worldwide. Up to 90% of people living with CF worldwide have at least one F508del mutation.
“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,” said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. “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.”
CF affects approximately 3,500 people in Australia. It is caused by a defective and/or missing CFTR protein resulting from mutations in the CFTR gene.
“Cystic fibrosis is a complex, progressive, devastating disease that causes severe damage to the lungs, digestive system and other organs in the body. It 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 Professor John Wilson AM, Head, Cystic Fibrosis Service, Alfred Health. “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.”
The TGA approval of TRIKAFTA was based on the results of four global Phase 3 studies, which included multiple trial sites and patients from Australia.