FDA approves expanded use of Trikafta (elexacaftor/tezacaftor/ivacaftor with ivacaftor) to include children with cystic fibrosis (CF) ages 6 to 11 years

Trikafta was previously approved by FDA for use in people age ≥12 years with ≥1 copy of F508del mutation or 1 copy of a mutation responsive in vitro. Phase 3 study (n=66) found regimen was generally well tolerated and safety data were similar to previous studies in ages ≥12 years

SPS commentary:

TRIKAFTA® is already approved for the treatment of patients ages 12 years and older with certain mutations in UK, as Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with Kalydeco (ivacaftor). Vertex has submitted applications for use of Trikafta/Kaftrio in children ages 6 through 11 years to the European Medicines Agency and the MHRA.

Biospace Inc.