What is the difference between Symdeko and Orkambi?
Symdeko (tezacaftor/ivacaftor and ivacaftor) and Orkambi (ivacaftor and lumacaftor) are both used to treat patients with cystic fibrosis. The two medicines differ in their ingredients and dosage forms, and the age groups and genetic mutations they treat.
Symdeko is supplied as co-packaged tablets:
- tezacaftor 100 mg/ivacaftor 150 mg and ivacaftor 150 mg
Orkambi is supplied as tablets and oral granules:
- lumacaftor 100 mg / ivacaftor 125 mg tablets
- lumacaftor 200 mg / ivacaftor 125 mg tablets
- lumacaftor 75 mg / ivacaftor 94 mg oral granules in unit-dose packets
- lumacaftor 100 mg / ivacaftor 125 mg oral granules in unit-dose packets
- lumacaftor 150 mg / ivacaftor 188 mg oral granules in unit-dose packets
Both medicines contain ivacaftor (Kalydeco) a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator.
Symdeko is approved for the treatment of patients with cystic fibrosis
- aged 6 years and older who are homozygous for the F508del mutation or who have at least one mutation in the CFTR gene that is responsive to Symdeko.
Orkambi is approved for the treatment of patients with cystic fibrosis
- aged 1 year and older who have two copies of the F508del mutation in their CFTR gene.
Orkambi should not be used in patients other than those who have two copies of the F508del mutation in their CFTR gene.
Kalydeco (ivacaftor) is approved for the treatment patients with cystic fibrosis
- aged 4 months and older who have one mutation in the CFTR gene that is responsive to ivacaftor.
Symdeko, Orkambi and Kalydeco are all manufactured by Vertex Pharmaceuticals.
Read next
How does Symdeko work?
Symdeko contains ivacaftor and tezacaftor. Ivacaftor allows the defective CFTR protein to work better, improving the exchange of chloride ions across cell membranes within the airways. Tezacaftor moves the defective CFTR protein to the cell membrane so that it can facilitate the transfer of chloride ions across the membrane. Ivacaftor boost the function of the defective CFTR protein and tezacaftor corrects its location. This combined approach helps maintain a good balance of salt and water within the lungs and provides superior treatment compared to either drug alone. Continue reading
What is an orphan drug?
Orphan drugs are medicines developed to help treat, prevent or diagnose rare “orphan” diseases, which are conditions that each affect fewer than 200,000 people in the U.S. Continue reading
How does Kalydeco work?
Kalydeco specifically targets the underlying cause of cystic fibrosis (CF) and is approved for adults, children, and infants aged 4 months and older with CF with one or more specific mutations in their CFTR gene that is responsive to Kalydeco. It allows the defective CFTR protein to work better and increases salt and water movement within the airways. This thins the mucus and makes it easier to cough out. Continue reading
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