Products
Tezacaftor was approved in the US and EU in 2018 and in many countries in 2019 in fixed combination with previously registered ivacaftor in film-coated tablet form (Symdeko). In 2020, a fixed combination with tezacaftor, elexacaftor, and ivacaftor was approved (Trikafta).
Structure and properties
Tezacaftor (C26H27F3N2O6, Mr = 520.5 g/mol) exists as a white powder that is practically insoluble in water. It is an indole derivative.
Effects
Tezacaftor (ATC R07AX31) facilitates cellular processing and transport of normal and mutant CFTR. This increases the number of functional proteins on the cell surface and improves chloride transport.
Indications
For the treatment of patients with cystic fibrosis who are homozygous for the F508del mutation or are heterozygous for the F508del mutation and have a mutation in the CFTR gene as defined in the SmPC.
Dosage
According to the SmPC. Tablets are taken morning and evening 12 hours apart with a fat-containing meal.
Contraindications
- Hypersensitivity
For complete precautions, see the drug label.
Interactions
Tezacaftor is a substrate of CYP3A and corresponding interactions are possible.
Adverse effects
The most common potential adverse effects include nasopharyngitis, headache, dizziness, sinus congestion, and nausea. These statements are for the fixed-dose combination.