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Elexacaftor-Tezacaftor-Ivacaftor: The First Triple-Combination Cystic Fibrosis Transmembrane Conductance Regulator Modulating Therapy.

Authors
  • Ridley, Kaden
  • Condren, Michelle
Type
Published Article
Journal
The journal of pediatric pharmacology and therapeutics : JPPT : the official journal of PPAG
Publication Date
Jan 01, 2020
Volume
25
Issue
3
Pages
192–197
Identifiers
DOI: 10.5863/1551-6776-25.3.192
PMID: 32265602
Source
Medline
Keywords
Language
English
License
Unknown

Abstract

Elexacaftor-tezacaftor-ivacaftor is a newly approved triple-combination cystic fibrosis transmembrane conductance regulator (CFTR) modulating therapy that contains 2 correctors and a potentiator of the CFTR channel. Its labeled indication for use is for persons 12 years of age and older with at least 1 F508del mutation for the CFTR gene. This drug combination provides potential therapy to many patients who had previously been excluded from CFTR modulation therapy due to the nature of their genetic mutations. The efficacy demonstrated in clinical trials surpasses the currently available therapies related to lung function, quality of life, sweat chloride reduction, and reducing exacerbations. The most common adverse events seen in clinical trials included rash and headache, and laboratory monitoring is recommended to evaluate liver function. Continued evaluation of patient data is needed to confirm its long-term safety and efficacy. Elexacaftor-tezacaftor-ivacaftor is a monumental and encouraging therapy for cystic fibrosis; however, approximately 10% of the CF population are not candidates for this or any other CFTR modulation therapy. Copyright Pediatric Pharmacy Association. All rights reserved. For permissions, email: [email protected] 2020.

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