12 March 2019 - - The Therapeutic Goods Administration of Australia has granted registration to Symdeko (tezacaftor/ivacaftor and ivacaftor) for the treatment of people with cystic fibrosis 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 gene that is responsive to tezacaftor/ivacaftor based on in vitrodata and/or clinical evidence, US-based Vertex Pharmaceuticals Inc said.

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.

The TGA's decision is based on results from two pivotal Phase 3 studies, EVOLVE and EXPAND, published in the New England Journal of Medicine in November 2017.

Results showed treatment with tezacaftor/ivacaftor in combination with ivacaftor provides benefits across different CF populations, including statistically significant improvements in lung function, as determined by absolute change from baseline in % predicted forced expiratory volume in one second (ppFEV1), with a generally well tolerated safety profile and a lack of increased respiratory adverse events compared to placebo.

The improvements in lung function showed a mean absolute change in ppFEV1 compared to placebo of 4.0 %age points (P