Vertex wins greenlight for Kalydeco in infants

16 August 2018

The US Food and Drug Administration (FDA) has approved Vertex Pharmaceuticals' (Nasdaq: VRTX) Kalydeco (ivacaftor) for use in cystic fibrosis (CF) patients under the age of 24 months.

The approval was issued following data obtained in the ongoing open-label Phase III ARRIVAL trial, investigating the drug's safety profile in patients aged 12 to 24 months. According to Vertex, this trial showed a safety profile consistent with the safety profile of Kalydeco in older children, with no severe adverse effects and no patients having withdrawn from the study.

The most common side-effects of Kalydeco in patients under two years old are coughs (73% of patients), fever (37%) and a runny nose (32%).

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