CF- Tezacaftor/Ivacaftor DI

Question 1

Tezacaftor/Ivacaftor brand name

Answer 1

Symdeko

Question 2

Tezacaftor MoA

Answer 2

Same as lumacaftor! Works to fix the defective CFTR protein so it can move to the proper place on the airway cell surface

Question 3

Is tezacaftor a CFTR potentiator or corrector?

Answer 3

Corrector!

Question 4

What is ivacaftor present for in Symdeko?

Answer 4

CFTR potentiator; just there to help the tezacaftor better by facilitating opening of the chloride channel

Question 5

What class of CF mutation does Symdeko work on?

Answer 5

Class II

Question 6

Symdeko indication

Answer 6

Patients ≥6 years who have 2 copies of the F508del mutation OR ≥1 mutation in the CFTR gene responsible to Symdeko