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

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      

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: Lumacaftor Ivacaftor (Orkambi) for Cystic Fibrosis
Article code: 1491b
 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