FDA Approval Expands Orkambi Use in Pediatric Patients with Cystic Fibrosis

The expanded approval was based on data from a previously announced Phase 3 study
The expanded approval was based on data from a previously announced Phase 3 study

Vertex announced that the Food and Drug Administration (FDA) has approved Orkambi (lumacaftor and ivacaftor) tablets for use in children with cystic fibrosis aged 6–11 years who have 2 copies of theF508del mutation. 

Previously, Orkambi was approved for use in patients aged ≥12 years with 2 copies of the F508del mutation. The expanded approval was based on data from a previously announced open-label Phase 3 clinical safety study of Orkambi.  

Related Articles

Orkambi combines lumacaftor and ivacaftor, a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator. Lumacaftor improves the conformational stability of F508del-CFTR, resulting in increased processing and trafficking of mature protein to the cell surface. Ivacaftor is a CFTR potentiator that facilitates increased chloride transport by potentiating the channel-open probability (or gating) of the CFTR protein at the cell surface. 

Orkambi is available as 100mg/125mg and 200mg/125mg strength tablets in 112-count boxes containing a 4-week supply. Each weekly carton contains 7 daily blister strips with 4 tablets per strip.

For more information call (877) 634-8789 or visit Orkambi.com.

Loading links....