FDA approves Symdeko for use in children with cystic fibrosis who have two copies of the F508del-CFTR mutation or who at least one mutation in the CFTR gene

Vertex Pharmaceuticals Incorporated announced the FDA approved Symdeko (tezacaftor/ivacaftor and ivacaftor) for use in children with cystic fibrosis ages 6 through 11 years who have two copies of the F508del-CFTR mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to Symdeko. It was previously approved by the FDA for use in patients with cystic fibrosis 12 years and older with two copies of the F508del mutation or one copy of a responsive mutation in the U.S.

An additional dosage strength of Symdeko tablets is now available (tezacaftor 50 mg/ivacaftor 75 mg and ivacaftor 75 mg) in connection with this approval. Vertex completed a 24-week Phase III open-label, multicenter study to evaluate the pharmacokinetics, safety, and tolerability of tezacaftor/ivacaftor and ivacaftor in children ages 6 through 11 years in the U.S. and Canada. The regimen was generally well tolerated, and safety data were similar to what was observed in previous studies of patients aged 12 years and older. The full data from this study will be published later this year.