The Food and Drug Administration (FDA) has approved Symdeko (tezacaftor/ivacaftor and ivacaftor; Vertex Pharmaceuticals) for the treatment of patients aged ≥6 years with cystic fibrosis (CF) who are homozygous for the F508del mutation or who have at least 1 mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor. Previously, the treatment has only been approved for patients ≥12 years old.

The expanded indication was based on extrapolated efficacy data from 3 double blind, placebo controlled phase 3 trials in patients aged ≥12 years with CF. Additionally, the Company evaluated the safety and tolerability of Symdeko in a 24-week open label, multicenter phase 3 study in 70 CF patients aged 6 through 11 years. The safety profile was similar to that observed in prior clinical trials of patients aged ≥12 years. The Company will publish full study results later this year.

“We’ve seen the clinical impact of Symdeko in people with CF aged 12 years and above, and this approval marks a crucial milestone for patients ages 6 through 11 years who may benefit from CFTR modulation, enabling us to treat the basic defect in CF at an earlier stage of disease,” said Seth Walker, MD, University Hospitals of Cleveland, Cleveland Medical Center, Rainbow Babies and Children’s Hospital. “Symdeko is an important treatment option for eligible people with CF who either never started or have discontinued another CFTR modulator.”

Symdeko is co-packaged as: tezacaftor 50mg/ivacaftor 75mg fixed dose combination tablets and ivacaftor 75mg strength tablets; or tezacaftor 100mg/ivacaftor 150mg fixed dose combination tablets and ivacaftor 150mg strength tablets in 56-count cartons (4-week supply). Dosing in pediatric patients <12 years old is based on weight. 

For more information visit symdeko.com.

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This article originally appeared on MPR