Posted by Michael Wonder on 04 May 2020
Vertex receives European CHMP positive opinion for Kalydeco (ivacaftor) for children and adolescents with cystic fibrosis between the ages 6 months and 18 years with the R117H mutation in the CFTR gene
1 May 2020 - If approved, Kalydeco (ivacaftor) will be the first and only medicine in Europe to treat the underlying cause of cystic fibrosis in patients with the R117H mutation, the most common residual function mutation, as young as 6 months of age.
Vertex Pharmaceuticals today announced that the EMA's CHMP adopted a positive opinion for the label extension of Kalydeco (ivacaftor), to include the treatment of children and adolescents with cystic fibrosis, ages 6 months and older weighing at least 5 kg who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
