FDA expanded approval of Vertex Pharmaceuticals’ cystic fibrosis (CF) therapy Kalydeco (ivacaftor) to include use in children aged at least 12 months, but younger than 24 months who carry at least one responsive CFTR mutation, making it the only medicine to treat CF in this age group.  

Kalydeco’s new approval was supported by data from the ongoing late-stage Arrival study of 25 children with mutations in the CTFR gene. The results of the trial, unveiled in December 2017, showed the safety profile of the therapy to be similar to that seen in trials with older patients.

Reshma Kewalramani, Vertex CMO commented, “with today’s approval, parents and physicians now have a medicine to treat the underlying cause of CF in patients as young as one year of age.” 

(Source: Vertex Pharmaceuticals)