Vertex Announces Reimbursement of Cystic Fibrosis Medicines SYMDEKO (tezacaftor/ivacaftor and ivacaftor) for Eligible Patients Ages 12 and Older, and ORKAMBI (lumacaftor/ivacaftor) in Children Ages 2 to 5, With Certain CFTR Mutations in Australia
Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced that SYMDEKO (tezacaftor/ivacaftor and ivacaftor) is reimbursed in Australia for people with cystic fibrosis (CF) ages 12 years and older who are homozygous for the F508del mutation or who have one copy of the F508del mutation and another responsive residual function (RF) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. People with CF who have one copy of the F508del mutation and another responsive RF mutation in the CFTR gene will have access to a medicine for the cause of their CF for the first time. In addition, ORKAMBI (lumacaftor/ivacaftor) is now also reimbursed for the treatment of children with CF ages 2 to 5 who have two copies of the F508del mutation in the CFTR gene. Patients over the age of 6 have already been able to access ORKAMBI in Australia since October 2018.
Following previously received positive recommendations from the Pharmaceutical Benefits Advisory Committee (PBAC), eligible patients in Australia will be able to access both medicines immediately, and the medicines will be listed on the Pharmaceutical Benefits Scheme (PBS) from December 1st.
“We are pleased that SYMDEKO and ORKAMBI will be made available immediately to eligible cystic fibrosis patients in Australia. We appreciate that the PBAC has recognized the value of these medicines to patients and thank the Department of Health and the Minister for Health in Australia for their strong engagement and collaboration to finalize the agreement,” said Ludovic Fenaux, Senior Vice President, Vertex International.
Vertex’s CF medicines are reimbursed in 17 countries around the world including Austria, Denmark, Germany, the Republic of Ireland, Italy, the Netherlands, Sweden and the U.S.
Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins 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 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 early 30s.