FDA expands approval of Orkambi for young children with cystic fibrosis

The FDA expanded the approval of lumacaftor and ivacaftor to include the treatment of children with cystic fibrosis aged 1 year to younger than 2 years, according to a manufacturer-issued press release.
This approval applies to children who are homozygous for the F508del mutation (F/F genotype) in the CFTR gene.
Harboring two copies of the F508del mutation leads to a deficiency of the CFTR protein at the cell surface. The agent combines lumacaftor, which targets the defect of the F508del-CFTR protein that inhibits processing and trafficking, thereby increasing the amount of the protein at the

The FDA expanded the approval of lumacaftor and ivacaftor to include the treatment of children with cystic fibrosis aged 1 year to younger than 2 years, according to a manufacturer-issued press release.
This approval applies to children who are homozygous for the F508del mutation (F/F genotype) in the CFTR gene.
Harboring two copies of the F508del mutation leads to a deficiency of the CFTR protein at the cell surface. The agent combines lumacaftor, which targets the defect of the F508del-CFTR protein that inhibits processing and trafficking, thereby increasing the amount of the protein at the