Vertex Pharmaceuticals announced the U.S. Food and Drug Administration approved the expanded use of Trikafta to include children with cystic fibrosis, or CF, ages 2 through 5 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator gene or a mutation in the CFTR gene that is responsive to Trikafta based on in vitro data. Trikafta was previously approved by the FDA for use in people with CF 6 years and older with at least one F508del mutation or a mutation in the CFTR gene that is responsive to Trikafta based on in vitro data, the company noted.
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