Vertex pharmaceuticals incorporated announces fda approves trikafta to treat the underlying cause of cystic fibrosis in people ages 12 and older who have at least one f508del mutation

Vertex pharmaceuticals incorporated announced the u.s. food and drug administration (fda) has approved trikafta™ (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of cystic fibrosis (cf) in people ages 12 years and older who have at least one f508del mutation in the cystic fibrosis transmembrane conductance regulator (cftr) gene, the most common cf-causing mutation. with this approval, for the first time, approximately 6,000 people with cf ages 12 years and older who have one f508del mutation and one minimal function mutation (f/mf) have a medicine that targets the underlying cause of their cf. additionally, approximately 12,000 people with one or two f508del mutations who are currently eligible for one of vertex’s three other fda-approved cf medicines are now also eligible for trikafta. vertex has submitted a marketing authorization application (maa) to the european medicines agency (ema) for the elexacaftor/tezacaftor/ivacaftor combination regimen. vertex is currently evaluating elexacaftor/tezacaftor/ivacaftor in people ages 6 through 11 with f/mf and f/f cf mutations in an ongoing phase 3 study and is committed to evaluating elexacaftor/tezacaftor/ivacaftor in children <6 years of age as part of planned future studies.
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