The Food and Drug Administration (FDA) has approved Orkambi (lumacaftor/ivacaftor; Vertex) for use in children 2 to 5 years old with cystic fibrosis (CF) who have 2 copies of the F508del-CFTR mutation. The treatment was previously only approved for patients ≥6 years.

The approval was based on data from a 24-week, open-label, Phase 3 study in patients aged 2 to 5 years (N=60) which showed a similar safety profile to that in patients ≥6 years. In addition, improvements in sweat chloride were observed at week 24 in this age group (mean absolute change from baseline was -31.7mmol/L; 95% CI: -35.7, -27.6). The efficacy and safety of Orkambi has not been established in children younger than 2 years old. 

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“Research suggests Orkambi could impact CF outcomes in patients as young as 2 years old,” said Dr John McNamara, Medical Director of the cystic fibrosis program at Children’s Minnesota hospital and lead study researcher. “This approval is a significant development that enables physicians to begin treating the underlying cause of the disease in this population earlier than ever before.”

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Vertex is also introducing Orkambi oral granules in 2 dosage strengths (lumacaftor 100mg/ivacaftor 125mg and lumacaftor 150mg/ivacaftor 188mg) for weight-based dosing. The granules can be mixed with soft food or liquid and should be ingested within 1 hour of mixing. The oral granules are expected to be available in 2 to 4 weeks in cartons containing 56 unit-dose packets.

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