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Vertex Pharmaceuticals Cystic Fibrosis Drug Kalydeco Approved for Infants as Young as 1 Month Old

NEW YORK – The US Food and Drug Administration has approved Vertex Pharmaceuticals' cystic fibrosis drug Kalydeco (ivacaftor) for patients as young as 1 month old, the biotechnology company said Wednesday.

Just last week, Boston-based Vertex announced the FDA expanded indications for its cystic fibrosis drug Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for patients aged 2 to 5 years old.

Kalydeco, which was already approved in the US and UK for patients 4 months and older, is an oral medication for patients with mutations in the CFTR gene that lead to abnormally functioning CFTR protein at the cell surface. Kalydeco is designed to facilitate CFTR proteins' ability to transport salt and water across the cell membrane.

The latest FDA approval was supported by a Phase III trial that demonstrated a safety profile for Kalydeco in younger children similar to that observed in older children and adults.

"Treating the underlying cause of cystic fibrosis as early as possible is important, and this approval, the first for a CFTR modulator in this age group, means families will now have a medicine for eligible infants," Carmen Bozic, chief medical officer and executive VP of global medicines development and medical affairs at Vertex, said in a statement.