Medical Professionals Reference (MPR)Trikafta Approved for Children with Cystic Fibrosis as Young as 2 Years

The approval was based on results from a 24-week open-label, two-part phase 3 trial that assessed Trikafta’s effectiveness and safety in 75 CF patients having at least one F508del mutation or a mutation known to be responsive to Trikafta. According to the findings, patients’ sweat chloride concentration and lung clearance index decreased in a clinically significant way. Trikafta therapy was typically well tolerated and commensurate with results seen in individuals who were older.