PBAC approve Vertex’s cystic fibrosis treatment ORKAMBI®

Vertex Pharmaceuticals Limited announces that it has received the Pharmaceutical Benefits Advisory Committee’s (PBAC) recommendation for ORKAMBI® (lumacaftor/ivacaftor) to be listed on the Pharmaceutical Benefits Scheme (PBS) in Australia for people ages six and over with cystic fibrosis (CF) who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Lumacaftor/ivacaftor is the first medicine to treat the underlying cause of CF in people ages six and older who have two copies of the F508del mutation.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the build-up of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the mid-to-late 20s.

In people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface.

ORKAMBI® is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface. Lumacaftor/ivacaftor is available as tablets and is typically taken twice per day.

Many thousands of patients worldwide are already receiving lumacaftor/ivacaftor in countries where it is reimbursed including Austria, Denmark, Germany, Ireland, Italy, Sweden, the Netherlands and the U.S.

Simon Bedson, International General Manager at Vertex said, “We’re very pleased that lumacaftor/ivacaftor received a positive PBAC recommendation for CF patients in Australia, which brings those who have long been waiting closer to receiving this important medicine. We welcome the PBAC decision and are now working with the Australian Government to finalize the agreement as quickly as possible to make lumacaftor/ivacaftor available to patients.”