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# Vertex Gains Expanded Use for Cystic Fibrosis Drug
- URL: https://www.fdaweb.com/vertex-gains-expanded-use-for-cystic-fibrosis-drug/
- Published: 2023-04-26T12:00:00.000Z
- Updated: 2026-09-14T18:27:45.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5154306

FDA has approved a Vertex Pharmaceuticals supplemental NDA for the expanded use of Trikafta (elexacaftor/tezacaftor/ivacaftor) to include children with cystic fibrosis (CF) aged two through five years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTRgene that is responsive to Trikafta. Previously, the drug was approved in this CF patient population in those aged six years and older.

Approval was based on data from a 24-week Phase 3 open-label study which enrolled 75 children aged two through five years with CF. “The regimen was generally well tolerated, with a safety profile consistent with that observed in older age groups, and led to improvements in sweat chloride concentration, a measure of CFTR function, and lung function,” the company says.

Trikafta is described by Vertex as an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface. “Elexacaftor and tezacaftor work together to increase the amount of mature protein at the cell surface,” it says. “Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. The combined actions of elexacaftor, tezacaftor and ivacaftor help hydrate and clear mucus from the airways.”