RARE Daily

Vertex Reaches Reimbursement Agreement in Australia for Cystic Fibrosis Treatment

March 28, 2022

Rare Daily Staff

After protracted discussion, Vertex Pharmaceuticals reached a reimbursement agreement with the Australian Pharmaceutical Benefits Scheme for Trikafta, its treatment for cystic fibrosis in people ages 12 years and older who have at least one F508del mutation in the CFTR gene, the most common CF-causing mutation worldwide.

“Today’s announcement is a significant milestone in ensuring Australians living with CF receive timely and sustainable access to Trikafta,” said Ludovic Fenaux, senior vice president, Vertex International.

With the reimbursement agreement in place, more than 2,200 people with CF will have PBS-funded access to Trifakta, including more than 700 who will now have access to a CFTR modulator therapy for the first time.

Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) was approved by the Australian Therapeutic Goods Administration in March 2021 based on the results of four global phase 3 clinical trials, which included multiple Australian trial sites and patients.

Cystic fibrosis (CF) is a rare, life-shortening, genetic disease affecting approximately 3,500 people in Australia. It is caused by a defective and/or missing CFTR protein, resulting from mutations in the CFTR gene. Up to 90 percent of people living with CF have at least one F508del mutation. CFTR mutations lead to CF by causing the CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the early 30s.

“As a genetic disease, cystic fibrosis is a prime candidate for precision medicine. Now, with PBS listing of Trikafta, eligible Australians living with CF ages 12 years and older can broadly access a therapy that treats the underlying cause of their disease. Clinicians across Australia will be excited about this most welcome news,” said Professor John Wilson AM, Head, Cystic Fibrosis Service, Alfred Health Australia.

Australia now joins the list of 30 countries where the triple combination therapy is approved and reimbursed including Denmark, Finland, France, Germany, Italy, Ireland, Israel, Poland, Spain, Switzerland and the countries within the U.K.

Photo: Ludovic Fenaux, senior vice president, Vertex International

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