Posted by Michael Wonder on 31 Jul 2018
Vertex receives European CHMP positive opinion for Symkevi (tezacaftor/ivacaftor) for people with cystic fibrosis aged 12 and older with certain mutations in the CFTR gene
27 July 2018 - If approved, Symkevi (tezacaftor/ivacaftor)will be Vertex’s third medicine to treat the CFTR protein defect in patients with cystic fibrosis – a rare life-shortening disease.
Vertex Pharmaceuticals today announced that the EMA CHMP adopted a positive opinion for Symkevi (tezacaftor/ivacaftor) in a combination regimen with ivacaftor (Kalydeco) for the treatment of people with cystic fibrosis aged 12 and older who either have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, or one copy of the F508del mutation and a copy of one of the following 14 mutations in which the CFTR protein shows residual activity: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T.
The regulatory submission was supported by results from two pivotal Phase 3 studies, EVOLVE and EXPAND, published in the New England Journal of Medicine in November 2017.
