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Orkambi, ivacaftor/lumacaftor

Also known as: ivacaftor/VX-809

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Company Vertex Pharmaceuticals Inc.
DescriptionFixed-dose combination of Kalydeco ivacaftor, a small molecule potentiator of cystic fibrosis transmembrane conductance regulator (CFTR), and lumacaftor (VX-809), a small molecule CFTR corrector
Molecular Target Cystic fibrosis transmembrane conductance regulator (CFTR)
Mechanism of ActionCFTR stabilizer
Therapeutic ModalitySmall molecule: Combination
Latest Stage of DevelopmentMarketed
Standard IndicationCystic fibrosis (CF)
Indication DetailsTreat cystic fibrosis (CF) in patients ages 6-11 with 2 copies of the delta F508 CFTR mutation; Treat cystic fibrosis (CF) in patients with 2 copies of the delta F508 CFTR mutation
Regulatory Designation U.S. - Breakthrough Therapy (Treat cystic fibrosis (CF) in patients with 2 copies of the delta F508 CFTR mutation);
U.S. - Orphan Drug (Treat cystic fibrosis (CF) in patients with 2 copies of the delta F508 CFTR mutation);
U.S. - Priority Review (Treat cystic fibrosis (CF) in patients with 2 copies of the delta F508 CFTR mutation)
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