Vertex wins FDA label expansion for cystic fibrosis drug to add younger children
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Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) announced Friday that the FDA approved the expanded use of its cystic fibrosis therapy Orkambi to include certain children aged 12 â 24 months.
A multi-organ disorder, CF is caused by a defective and/or missing CFTR protein due to mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

The latest approval allows the use of Orkambi in CF patients as young as 12 months who have two copies of the F508del mutation in the CFTR gene, a common genetic defect linked to a vast majority of CF cases. 

âTreating children with cystic fibrosis as early in life as possible is critically important, because early treatment has the potential to slow the progression of this devastating disease,â noted Carmen Bozic, Chief Medical Officer of Vertex (VRTX).
Orkambi, also known as lumacaftor/ivacaftor, was previously indicated in the U.S. for CF patients aged two years and older with two copies of the F508del mutation in the CFTR gene. The FDA first approved the treatment in 2015 for CF patients aged 12 years and older.