Lumacaftor-ivacaftor (Orkambi) for cystic fibrosis: behind the ‘breakthrough’

Abstract

Lumacaftor-ivacaftor (Orkambi) was recently approved by the Food and Drug Administration (FDA) in the USA to treat patients at least 12 years old who have cystic fibrosis due to two copies of the F508del ( Phe508del ) mutation in the cystic fibrosis transmembrane conductance regulator ( CFTR ) gene. Lumacaftor-ivacaftor has been designated a ‘breakthrough’ and is slated to cost $259 000 per year per patient, but close scrutiny of the data behind this new medication reveals modest improvements in outcomes that are not exclusively benefited by lumacaftor-ivacaftor, and the $259 000 cost is questionable and problematic. Providers considering this therapy should have a candid and easily-understandable discussion with their patients about what the data show for lumacaftor-ivacaftor so their patients can make an informed decision about taking it. This article aims to help this process.