FDA New Drug and Biologic Approvals, 2018 Midyear Review

Mary L Windle, PharmD

Disclosures

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.

Mechanisms:

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.

Dosage:

  • 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.

References:

Comments

3090D553-9492-4563-8681-AD288FA52ACE
Comments on Medscape are moderated and should be professional in tone and on topic. You must declare any conflicts of interest related to your comments and responses. Please see our Commenting Guide for further information. We reserve the right to remove posts at our sole discretion.
Post as:

processing....