Vanzacaftor, tezacaftor and deutivacaftor FDA Approval Status
Last updated by Judith Stewart, BPharm on July 17, 2024.
FDA Approved: No
Generic name: vanzacaftor, tezacaftor and deutivacaftor
Company: Vertex Pharmaceuticals Incorporated
Treatment for: Cystic Fibrosis
Vanzacaftor/tezacaftor/deutivacaftor is a next-in-class, once daily triple combination therapy (vanza triple) in development for the treatment of cystic fibrosis.
- Vanzacaftor/tezacaftor/deutivacaftor is intended for use in people ages 6 years and older with cystic fibrosis who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene responsive to vanza triple.
- Cystic fibrosis (CF) 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.
- Vanzacaftor and and tezacaftor are designed to increase the amount of CFTR protein at the cell surface by targeting the processing and trafficking defect of the CFTR protein. 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). Vanzacaftor and deutivacaftor are not yet contained in any approved combination CF treatments.
- Vanzacaftor/tezacaftor/deutivacaftor has been granted Fast Track and Orphan Drug Designations from the U.S. Food and Drug Administration for the treatment of cystic fibrosis.
Development timeline for vanzacaftor, tezacaftor and deutivacaftor
Further information
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