FDA approves KALYDECO (ivacaftor) as the first and only CFTR regulator to treat children with cystic fibrosis.

The U.S. Food and Drug Administration (FDA) has approved KALYDECO® for children with cystic fibrosis (CF) from 4 months to less than 6 months with at least one CFTR gene mutation, the pharmaceutical company Vertex announced today.
KALYDECO ® has been approved in the United States and the European Union to treat CF patients over 6 months.
, chief executive of Vertex, said: "Since KALYDECO's initial approval eight years ago, we have been advancing clinical development programmes with a view to treating cystic fibrosis as early as possible.
today's approval is testimony to our tireless efforts with the clinical and scientific community."
FDA approval is based on Phase III Open Label Security Queue (ARRIVAL), which consists of six children aged 4 months to less than 6 months with 10 mutations in the CFTR gene (G551D, G 178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H).
that the team study showed that its safety was similar to that observed in older children and adults.
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