FDA New Drug and Biologic Approvals, 2018 Midyear Review

Mary L Windle, PharmD


August 01, 2018

In This Article

Cystic Fibrosis: Symdeko (tezacaftor/ivacaftor)

Indication: Indicated for cystic fibrosis (CF) in patients aged 12 years or older who are homozygous for the F508del mutation or who have at least 1 mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence.


Tezacaftor: CFTR corrector; increases amount of mature CFTR protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein.

Ivacaftor: CFTR potentiator; enhances function of the CFTR protein once it reaches the cell surface.


  • Copackaged as a fixed dose combination tablet of tezacaftor 100 mg and ivacaftor 150 mg PLUS a separate ivacaftor 150mg tablet.

  • Morning dose: One tezacaftor/ivacaftor 100-mg/150-mg fixed-dose tablet PO.

  • Evening dose: One ivacaftor 150-mg tablet PO.

  • Administer morning and evening doses ~12 hr apart.

Approval was based on the EVOLVE and EXPAND phase 3 trials. Results showed treatment with tezacaftor/ivacaftor and ivacaftor measured improvements across multiple disease measures, including lung function and pulmonary exacerbations compared with ivacaftor monotherapy.



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