CFTR Treatments

Question 1

Kayldeco (Vertex)

Answer 1

Ivacaftor
Potentiator = opens the CFTR channels in the cell membrane
Initially approved for G551D (class III mutation), now in use for 38 missense mutations
Children 4+ months
Costs $311,000

Question 2

Orkambi (Vertex)

Answer 2

Ivacaftor + Lumacaftor
Corrector + Potentiator = moves defective CFTR to the membrane and opens channels
Approved for homozygous deltaF508
2+ yrs and older
Side effects? Liver disease, interacts with hormonal birth control
Costs ~$250-$270

Question 3

Symdecko (Vertex)

Answer 3

Ivacaftor + Tezacaftor
Corrector + Potentiator = moves defective CFTR to the membrane and opens channels
Approved for homozygous deltaF508 OR heterozygous for 26 other mutations
Ages 6+
Costs $292,000

Question 4

Trikafta (Vertex)

Answer 4

Ivacaftor + Tezacaftor + Elexacaftor 
Corrector + Potentiator = moves defective CFTR to the membrane and opens channels
Approved for heterozygous deltaF508
Ages 12+ (trials ongoing) 
Costs $311,500