Posted by Michael Wonder on 01 Nov 2018
Vertex announces European authorisation for third cystic fibrosis medicine Symkevi (tezacaftor/ivacaftor), to be used in combination with ivacaftor (Kalydeco), for people with CF aged 12 and older with certain mutations in the CFTR gene
1 November 2018 - First medicine in the EU to treat the CFTR protein defect in patients who have one copy of the F508del mutation and one copy of one of 14 mutations that result in residual CFTR activity.
Vertex Pharmaceuticals today announced that the European Commission has granted Marketing Authorisation for Symkevi (tezacaftor/ivacaftor) in a combination regimen with ivacaftor (Kalydeco) for the treatment of people with cystic fibrosis (CF) 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. In addition, the European Medicines Agency’s Committee for Orphan Medicinal Products recently recommended the maintenance of orphan designation for tezacaftor/ivacaftor in combination with ivacaftor.
The EU marketing authorisation was based on results from two pivotal Phase 3 studies, EVOLVE and EXPAND, published in the New England Journal of Medicine in November 2017.
