Therapy Areas: Hereditary Disorders
Vertex Pharmaceuticals (Europe) receives positive opinion from European watchdog for Kalydeco
23 October 2018 -

UK-based Vertex Pharmaceuticals (Europe) has received a positive opinion from the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) for Kalydeco (ivacaftor) intended to treat patients with cystic fibrosis aged 12 to less than 24 months, it was reported on Monday.

The committee has recommended the product to treat cystic fibrosis patients in the age category who have at least one of the following nine mutations in their cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

The submission was supported by data from the ongoing Phase 3 open-label safety study (ARRIVAL) of children with CF aged 12 to

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