New CF drug gets TGA approval

Cystic fibrosis

20 Mar 2019

The TGA has approved a new cystic fibrosis treatment tezacaftor/ivacaftor (Symdeko) for patients aged 12 and older.

Manufacturer Vertex announced  on 12 March that its drug (tezacaftor 100 mg/ivacaftor 150 mg tablet and ivacaftor 150 mg tablets) had been approved by the TGA for patients with F508del and residual function mutations in the CF transmembrane conductance regulator (CFTR) gene.

“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),” said Reshma Kewalramani, M.D., Executive Vice President and Chief Medical Officer at Vertex.

The drug now awaits consideration by the Pharmaceutical Benefits Advisory Committee at its March meeting, with a decision expected to be announced on April 25th.

In a statement, Vertex said the TGA’s decision was based on results from two pivotal Phase 3 studies, EVOLVE and EXPAND, published in the NEJM 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 25 residual function mutations in the CFTR gene that produce CFTR protein responsive to Symdeko include: 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.

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