Posted by Michael Wonder on 12 Mar 2019
Vertex receives approval for Symdeko (tezacaftor/ivacaftor and ivacaftor) in Australia, to treat the underlying cause of cystic fibrosis in people aged 12 and older with certain CFTR gene mutations
12 March 2019 - A new treatment option for patients with two copies of the F508del mutation, the most common mutation in cystic fibrosis.
Vertex Pharmaceuticals Incorporated today announced that the Therapeutic Goods Administration of Australia has granted registration to Symdeko (tezacaftor/ivacaftor and ivacaftor) for the treatment of people with cystic fibrosis (CF) aged 12 years and older who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence. Mutations in the CFTR gene that produce CFTR protein responsive to Symdeko include F508del and mutations in which the CFTR protein shows residual function: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, 3849+10kbC→T, E56K, R74W, D110E, D110H, E193K, E831X, F1052V, K1060T, A1067T, F1074L and D1270N.
Symdeko will be considered for Australian reimbursement for eligible CF patients aged 12 years and older at the March meeting of the Pharmaceutical Benefits Advisory Committee.
