Orkambi® is now approved for use in children with cystic fibrosis ages 1-2 years who have two F508del mutations. This is the only modulator available to very young children with these mutations.
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The U.S. Food and Drug Administration has approved the use of Trikafta® (elexacaftor/tezacaftor/ivacaftor) for children ages 6 through 11 who have at least one copy of the F508del mutation or certain mutations in the CFTR gene that are responsive based on lab data.
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