Products
Elexacaftor was approved in the United States in 2019 and in many countries in 2020 as a fixed-dose combination with tezacaftor and ivacaftor in film-coated tablet form (Trikafta). In the morning dose, all three active ingredients are included. In the evening dose, only ivacaftor.
Structure and properties
Elexacaftor (C26H34F3N7O4S, Mr = 597.7 g/mol) exists as a white crystalline powder that is practically insoluble in water.
Effects
Elexacaftor (ATC R07AX32) is a so-called CFTR corrector. It binds to the CFTR protein and enhances cellular processing and transport of F508del-CFTR to the cell surface. This increases the opening probability of the CFTR channel.
Indications
For the treatment of patients with cystic fibrosis 12 years of age and older who are either homozygous for the F508del mutation in the CFTR gene or heterozygous for the F508del mutation in the CFTR gene and have a minimal function mutation.
Dosage
According to the SmPC. Tablets are taken in the morning and evening with a fat-containing meal. Caution: as mentioned above, the morning and evening doses contain different active ingredients. Foods containing grapefruit should be avoided.
Contraindications
- Hypersensitivity
For complete precautions, see the drug label.
Interactions
Elexacaftor is a substrate of CYP3A4 and corresponding interactions with inducers and inhibitors are possible.
Adverse effects
The most common potential adverse effects of the combination include headache, diarrhea, and upper respiratory tract infection.