CF, a rare and life-shortening genetic disease, that affects around 75,000 people across the globe. It is a progressive and multi-system disease that damages the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract.
The label of the combined drug has been expanded to treat CF aged between six and 11 years who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
It can also be used to treat patients with 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.
Vertex will immediately offer the combined drug with a new indication in Germany. Under long-term reimbursement agreements, Vertex will shortly launch Symkevi plus Kalydeco in countries such as the UK, Denmark and the Republic of Ireland.
Vertex will coordinate with relevant authorities in other European countries to provide the new treatment for all patients.
Vertex CEO and president Reshma Kewalramani said: “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.
“This approval brings us closer to our ultimate goal of providing medicines for all people with CF.”
In Europe, Symkevi plus Kalydeco already secured approval to treat people with CF aged between 12 years and older who have either two copies of the F508del mutation in the CFTR gene or a copy of one of the14 mutations in which the CFTR gene results in residual CFTR protein activity.
In June, Vertex secured EC approval for its Kalydeco (ivacaftor) to treat children and adolescents with cystic fibrosis (CF).