Vertex pharmaceuticals incorporated receives european chmp positive opinion for kalydeco (ivacaftor) to treat eligible infants with cystic fibrosis as early as 6 months of age

Vertex pharmaceuticals (europe) limited announced that the european medicines agency’s (ema) committee for medicinal products for human use (chmp) adopted a positive opinion for kalydeco (ivacaftor), to include use in infants with cystic fibrosis (cf) ages 6 months to less than 12 months who have one of the following mutations in their cystic fibrosis transmembrane conductance regulator (cftr) gene: g551d, g1244e, g1349d, g178r, g551s, s1251n, s1255p, s549n or s549r. if the european commission issues a favorable adoption of the ema chmp opinion for the extension of indication, ivacaftor will be the first and only medicine approved in europe to treat the underlying cause of cf in patients ages 6 months to less than 12 months, who have specific mutations in the cftr gene. the submission was supported by data from the ongoing phase 3 open-label safety study (arrival) of children with cf ages 6 months to less than 12 months who have certain mutations in the cftr gene. the study showed a safety profile similar to that observed in previous phase 3 studies of older children and adults, and improvements in sweat chloride, a key secondary efficacy endpoint. ivacaftor is already approved in europe for the treatment of cf in patients ages 12 months and older who have one of the following mutations in the cftr gene: g551d, g1244e, g1349d, g178r, g551s, s1251n, s1255p, s549n or s549r. it is also approved for the treatment of cf in patients 18 years and older who have the r117h mutation in the cftr gene.
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