The Medical Letter on Drugs and Therapeutics
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1558
Tezacaftor/Ivacaftor (Symdeko) for Cystic Fibrosis
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The FDA has approved the fixed-dose combination of tezacaftor and ivacaftor (Symdeko – Vertex) for oral treatment of cystic fibrosis (CF) in patients ≥12 years old who are homozygous for the F508del (also called Phe508del or ΔF508) mutation or have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to the combination. About 50% of CF patients in the US are homozygous for the F508del mutation. This is the first approved indication for tezacaftor. Ivacaftor is also available in combination with lumacaftor as Orkambi for treatment of CF in F508del-homozygous patients ≥2 years old and alone as Kalydeco for treatment of CF in patients ≥12 months old with other sensitive mutations.1,2

MECHANISM OF ACTION — The CFTR protein functions as a regulated chloride channel. In CF, mutations ... more      

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Title: Tezacaftor/Ivacaftor (Symdeko) for Cystic Fibrosis
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