FDA Approval Expands Orkambi Use in Pediatric Patients with Cystic Fibrosis
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.
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.