Vertex secures TGA approval for Cystic Fibrosis therapy

Pharma News: Vertex Pharmaceuticals secured the Australian Therapeutic Goods Administration (TGA) approval of its Cystic Fibrosis drug.

TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor), triple-combination drug, is indicated for people with Cystic Fibrosis (CF) ages 12 years and older who have at least one F508del mutation in the CF 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.

Professor John Wilson AM, Head, Cystic Fibrosis Service, Alfred Health said “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.

“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.”

Lauren Carey, Country Manager Australia & New Zealand, Vertex Pharmaceuticals, said “We think the announcement will be well received by Australians living with cystic fibrosis. This approval by the TGA is the first important step in the process of seeking reimbursed access to TRIKAFTA.”

Trikafta’s first full year on the global market proved a huge success during which its $3.9 billion revenue globally increased Vertex’s total sales by 55% to $6.2 billion.

At this year’s J.P. Morgan healthcare conference, Vertex CEO Dr Reshma Kewalramani said the company was actively scouting for “tools and technology” to acquire, specifically mid- and late-stage assets to boost its pipeline beyond CF.