Vertex Pharma's Flagship Cystic Fibrosis Drug Scores FDA Approval For Infants

by Vandana Singh, Benzinga Editor

September 2, 2022 1:45 PM | 1 min read | Make a Comment

The FDA has approved Vertex Pharmaceuticals Incorporated's VRTX Orkambi (lumacaftor/ivacaftor) for expanded use to include children with cystic fibrosis (CF) ages 12 to <24 months.
The approval covers infants who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene.
Also see: FDA Lifts Clinical Hold On Vertex Pharma's Stem Cell-Derived Diabetes Treatment Trial.
Orkambi was previously approved by the FDA for use in people with CF ages two years and older with two copies of the F508del mutation.
Orkambi was first approved in 2015 in the U.S. and is now available in more than 30 countries.
Price Action: VRTX shares are down 2.23% at $283.74 on the last check Friday.