Orkambi Shows Long-term Safety, Efficacy in Children Ages 2-5

Orkambi Shows Long-term Safety, Efficacy in Children Ages 2-5

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Orkambi Shows Long-term Safety, Efficacy in Children Ages 2-5

Long-term use of Orkambi (lumacaftor/ivacaftor), an approved cystic fibrosis (CF) treatment, is safe and effective for children between ages 2 and 5 with two copies of the disease-causing F508del mutation in the CFTR gene, a study reported. It draws on results from a Phase 3 open-label extension trial (NCT03125395) showing that Orkambi’s safety and efficacy could be maintained for up to 120 weeks (about 2.3 years), well beyond the 24 weeks of treatment that supported…

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