Press release
CORRECTING and REPLACING Vertex Announces U.S. FDA Approval for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 6 through 11 With Certain Mutations
- With this approval approximately 1,500 children with one minimal function mutation and one F508del mutation have a medicine to treat the underlying cause
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n- With this approval approximately 1,500 children with one minimal function mutation and one F508del mutation have a medicine to treat the underlying cause of their disease for the first time -\n\n BOSTON--(BUSINESS WIRE)--\nFirst paragraph, third sentence of release should read: An additional dosage strength of TRIKAFTA tablets is now available (elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg and ivacaftor 75 mg)...(instead of An additional dosage strength of TRIKAFTA tablets is now available (elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 75 mg and ivacaftor 75 mg)...).\n\nThe updated release reads:\n\nVERTEX ANNOUNCES U.S. FDA APPROVAL FOR TRIKAFTA® (ELEXACAFTOR/TEZACAFTOR/IVACAFTOR AND IVACAFTOR) IN CHILDREN WITH CYSTIC FIBROSIS AGES 6 THROUGH 11 WITH CERTAIN MUTATIONS\n\n- With this approval approximately 1,500 children with one minimal function mutation and one F508del mutation have a medicine to treat the underlying cause of their disease for the first time -\n\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved expanded use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis (CF) ages 6 through 11 years 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 to TRIKAFTA based on in vitro data. TRIKAFTA was previously approved by the FDA for use in people with cystic fibrosis 12 years and older with at least one copy of the F508del mutation or one copy of a mutation that is responsive in vitro. An additional dosage strength of TRIKAFTA tablets is now available (elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg and ivacaftor 75 mg) in connection with this approval.\n\n“Today’s approval is a critical milestone in our efforts to deliver medicines that help treat the underlying cause of this devastating disease as early in life as possible,” said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. “We can now reach approximately 1,500 newly eligible children in the U.S., and we continue to pursue approval for this expanded indication in other countries.”\n\nVertex completed a 24-week Phase 3 open-label, multicenter study which enrolled 66 children ages 6 through 11 years old wi...