FDA approval for Kalydeco to treat eligible infants with cystic fibrosis ages 1 month and older.-Vertex Pharma
Vertex Pharmaceuticals Incorporated announced the FDA approved Kalydeco (ivacaftor) for use in children with cystic fibrosis (CF) ages 1 month to less than four months old who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to Kalydeco based on clinical and/or in vitro assay data.
Kalydeco is already approved in the U.S. and EU for the treatment of CF in patients ages four months and older.
“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,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex.
The approval was supported by a cohort in the Phase III, 24-week, open-label study to evaluate the safety, pharmacokinetics and pharmacodynamics of ivacaftor in subjects with CF who are less than 24 months of age and have an ivacaftor-responsive CFTR mutation. This cohort demonstrated a safety profile similar to that observed in older children and adults.
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