Vertex Pharmaceuticals : Gets FDA Priority Review of Expanded Trikafta Age Range

By Colin Kellaher

Vertex Pharmaceuticals Inc. on Tuesday said the U.S. Food and Drug Administration granted priority review to its application for an expanded age range for the cystic-fibrosis drug Trikafta.

The Boston drug maker is seeking approval to include children ages six through 11 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 based on in-vitro data.

Trikafta is currently approved for patients ages 12 and older who have at least one copy of the F508del mutation in CFTR gene or another mutation that is responsive to treatment with the drug. Vertex said approval of the expanded age range could potentially benefit about 1,500 children.

The FDA grants priority review to medicines that have the potential to provide significant improvements in the treatment of a serious disease, and the designation shortens the review period. Vertex said the agency set a target action date of June 8 for the application.

Write to Colin Kellaher at [e-mail]

(END) Dow Jones Newswires

01-26-21 0910ET