Alyftrek FDA Approval History
Last updated by Judith Stewart, BPharm on Dec 28, 2024.
FDA Approved: Yes (First approved December 20, 2024)
Brand name: Alyftrek
Generic name: deutivacaftor, tezacaftor and vanzacaftor
Company: Vertex Pharmaceuticals Incorporated
Treatment for: Cystic Fibrosis
Alyftrek (deutivacaftor, tezacaftor and vanzacaftor) is a next-in-class, triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator for use in the treatment of cystic fibrosis.
- Alyftrek is indicated for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation or another responsive mutation in the CFTR gene.
If the patient’s genotype is unknown, an FDA-cleared CF mutation test should be used to confirm the presence of at least one indicated mutation. - Cystic fibrosis is a rare, genetic disease caused by mutations in the CFTR gene, which leads to decreased quantity and/or function of the CFTR protein channel at the cell surface, resulting in the poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients.
- Alyftrek contains three drugs which work together to increase the quantity and function of CFTR at the cell surface, resulting in increased CFTR activity.
- tezacaftor and vanzacaftor bind to different sites on the CFTR protein and have an additive effect in facilitating the cellular processing and trafficking of select mutant forms of CFTR (including F508del-CFTR) to increase the amount of CFTR protein delivered to the cell surface compared to either molecule alone.
- deutivacaftor is a potentiator designed to keep CFTR proteins at the cell surface open longer to improve the flow of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways. - Tezacaftor is contained in the approved combination CF treatments Symdeko (ivacaftor/tezacaftor and ivacaftor) and Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor). Alyftrek is the first FDA approved combination CF treatment that contains either vanzacaftor or deutivacaftor.
- Alyftrek tablets are administered orally, once daily, with fat-containing food.
- Alyftrek comes with a Boxed Warning for drug-induced liver injury and liver failure. Warnings and precautions include serious and potentially fatal drug-induced liver injury and liver failure; hypersensitivity reactions, including anaphylaxis; drug interactions with CYP3A inducers and CYP3A inhibitors; and cataracts.
- Common adverse reactions include cough, nasopharyngitis, upper respiratory tract infection, headache, oropharyngeal pain, influenza, fatigue, increased ALT, rash, increased AST, and sinus congestion.
Development timeline for Alyftrek
Further information
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