CF- Lumacaftor/Ivacaftor DI Flashcards
1
Q
Lumacaftor/Ivacaftor brand name
A
Orkambi
2
Q
Lumacaftor MoA
A
Fixes the defective CFTR protein so it can move to the proper place on the airway cell surface
3
Q
Is Lumacaftor a CFTR potentiator or corrector?
A
Corrector
4
Q
Orkambi works on what CF mutation class?
A
Class II
5
Q
Class II mutation
A
Protein is made but misfolded, so it never reaches the cell surface
6
Q
Orkambi indication
A
Patients ≥2 years who have 2 copies of the F508del CFTR mutation (homozygous)
7
Q
Role of ivacaftor in Orkambi
A
CFTR potentiator, just there to open the chloride channel and make the lumacaftor work better
