US FDA approves Vertex’s Orkambi for cystic fibrosis in children
Pharmaceutical Technology
SEPTEMBER 5, 2022
The treatment is indicated for CF patients who are homozygous for the F508del mutation (F/F genotype) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The latest approval for use in children aged 12 to below 24 months is based on an open-label, multicentre Phase III clinical trial for 24 weeks.
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