The Medical Letter on Drugs and Therapeutics
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1491
Lumacaftor/Ivacaftor (Orkambi) for Cystic Fibrosis
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The FDA has approved a fixed-dose combination of lumacaftor and ivacaftor (Orkambi – Vertex) for oral treatment of cystic fibrosis (CF) in patients ≥12 years old who are homozygous for the F508del mutation. About 50% of patients in the US with CF are homozygous for the F508del (also called Phe508del) mutation. This is the first approved indication for lumacaftor; ivacaftor is available alone as Kalydeco for treatment of CF in patients ≥2 years old with other mutations.1 Orkambi is the first drug to be approved in the US for treatment of patients with the F508del mutation.

MECHANISM OF ACTION — The cystic fibrosis transmembrane conductance regulator (CFTR) protein functions as a regulated chloride channel. The F508del mutation causes CFTR protein misfolding, a reduced quantity of CFTR protein at the cell surface, and decreased stability of the ... more      

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Title: Lumacaftor Ivacaftor (Orkambi) for Cystic Fibrosis
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