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.