The Medical Letter on Drugs and Therapeutics
Tezacaftor/Ivacaftor (Symdeko) for Cystic Fibrosis
Subscribers: Log in to read full article.  Not a subscriber?  Subscribe or purchase article.

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      

The Medical Letter is a subscriber-funded nonprofit organization that publishes critical appraisals of new prescription drugs and comparative reviews of drugs for common diseases.

Would you like to read the rest of this article?  Gain access below.

Subscriptions to The Medical Letter on Drugs and Therapeutics include:
  • Print version published and mailed biweekly (26 issues/year)
  • Unlimited online access to current and past issues (1988 - present)
  • Mobile App for iOS, Android, and Kindle Fire
  • FREE online per issue CME/CE
Purchase this article:
Title: Tezacaftor Ivacaftor (Symdeko) for Cystic Fibrosis
Article code: 1558b
 Electronic, downloadable article - $45
Gain access through your organization
Ask your librarian to consider an Institutional Subscription to The Medical Letter.
Recommend to your librarian