The European Commission as approved use of Vertex' Symkevi (tezacaftor/ivacaftor) with Kalydeco (ivacaftor) for eligible children with cystic fibrosis (CF) aged 6-11 years.
The combination therapy offers another treatment option for CF patients who are homozygous for F508del in this age group, and is the only medicine to treat the underlying cause of CF in this patient group.
Specifically, approval covers use of the combination in patients aged six years and older who have two copies of the F508del mutation in the CF transmembrane conductance regulator (CFTR) gene or one copy of the F508del mutation and one copy of one of 14 mutations in the CFTR gene that result in residual CFTR activity: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T.
“With this approval, children with CF in Europe ages 6 to 11 years with the most common mutation, F508del, have a new treatment option and children with certain residual function mutations will, for the first time, have a treatment option available that addresses the underlying cause of their CF,” said Reshma Kewalramani, chief executive and president at Vertex.
In Europe, the medicine is already approved for the treatment of people with CF ages 12 years and older who have either two copies of the F508del mutation in the CFTR gene, or one copy of the F508del mutation and a copy of one of the following 14 mutations in which the CFTR gene results in residual CFTR protein activity: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T.
CF accounts for 9,500 hospital admissions and over 100,000 hospital bed days a year. A third of these are used by children under 15.