Vertex Pharma (VRTX) Receives Approval for SYMDEKO in Australia to Treat Underlying Cause of CF
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the Therapeutic Goods Administration (TGA) 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.
“We are delighted that the Therapeutic Goods Administration in Australia recognized the safety profile and efficacy of SYMDEKO®. This approval brings us one step closer to our future goal of bringing treatment to all people living with CF,” said Reshma Kewalramani, M.D., Executive Vice President and Chief Medical Officer at Vertex. “This new medicine is an especially important treatment option for patients with residual function mutations and those with two copies of the F508del mutation who either never started or discontinued ORKAMBI® (lumacaftor/ivacaftor).”
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 percent 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 percentage points (P<0.0001) and 6.8 percentage points (P<0.0001) in EVOLVE and EXPAND respectively. The most common adverse reactions (≥10% incidence) experienced by patients who received tezacaftor/ivacaftor in combination with ivacaftor in pooled, placebo-controlled Phase 3 studies were headache and nasopharyngitis.
Tezacaftor/ivacaftor in combination with ivacaftor was approved by the U.S. Food and Drug Administration (FDA) in February 2018, by Health Canada in June 2018 and by the European Commission in October 2018.