Ivacaftor / Tezacaftor / Elexacaftor

What it is and how it works

Elexacaftor and tezacaftor are CFTR correctors that bind different sites on the CFTR protein, improving its processing and trafficking to the cell surface; ivacaftor is a CFTR potentiator that increases the channel-open probability. Together they increase chloride transport in patients with responsive CFTR mutations, including F508del.

Indications

Treatment of cystic fibrosis in patients (from the age specified for each product) who have at least one F508del mutation or another responsive CFTR mutation.

Dosage and administration (summary)

Adults and children 12 years and over: two tablets (each elexacaftor 100 mg / tezacaftor 50 mg / ivacaftor 75 mg) in the morning and one ivacaftor 150 mg tablet in the evening, about 12 hours apart, with fat-containing food; dose reduction is required in moderate hepatic impairment and with moderate or strong CYP3A inhibitors.

Contraindications

Hypersensitivity to the active substances or to any excipient; co-administration with strong CYP3A inducers is not recommended.

Warnings and precautions

Elevated transaminases and serious liver injury, including hepatic failure, have been reported; check liver function before starting, regularly during the first year and annually thereafter. Rash, depression and other mood changes may occur. Non-congenital lens opacities have been reported in children, so baseline and follow-up eye examinations are recommended.

Pregnancy

Limited data; use in pregnancy only if clearly needed.

Storage and supply

Store below 30 °C in the original package.

Source: reference summary prepared from the EU SmPC/EPAR, BNF and WHO Model Formulary. The approved product information in Iran governs.