Vertex: FDA OKs Trikafta for Some Children Ages 2 Through 5 With Cystic Fibrosis With Certain Mutations

By Colin Kellaher

Vertex Pharmaceuticals Inc. on Wednesday said the U.S. Food and Drug Administration approved the expanded use of its Trikafta cystic-fibrosis drug in certain children between the ages of two through five who have the rare, life-shortening genetic disease.

The Boston drugmaker said the approval covers patients who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator, CFTR, gene, or a mutation in the CFTR gene that is responsive to Trikafta based on in-vitro data.

The FDA in 2019 approved Trikafta for patients 12 years and older with the most common cystic fibrosis mutation, and the agency expanded the approval in 2021 to include children ages six through 11.

Vertex said the latest approval gives about 900 children with cystic fibrosis their first medicine to treat the underlying cause of their disease.

Write to Colin Kellaher at colin.kellaher@wsj.com

(END) Dow Jones Newswires

April 26, 2023 14:18 ET (18:18 GMT)

Vertex Pharmaceuticals (NASDAQ:VRTX)
Historical Stock Chart
From Nov 2023 to Dec 2023