Tezacaftor for the treatment of cystic fibrosis

Marc A Sala, Manu Jain*

*Corresponding author for this work

Research output: Contribution to journalArticle

8 Citations (Scopus)

Abstract

Introduction: Cystic fibrosis (CF) is the most common, life-limiting autosomal recessive disease in Caucasians, and is caused by defects in production of the CFTR ion channel. Until recently, there were no available treatments targeting the disease-causing defects in CFTR but newly developed CFTR modulators are changing the course of disease in CF. The newest modulator, tezacaftor, is a CFTR corrector that was recently approved by the FDA to be used in combination with the first approved CFTR potentiator, ivacaftor. Areas covered: A detailed review of the clinical trials and published literature, focusing on safety and efficacy, leading to the approval of tezacaftor in CF. Expert commentary: Recent trials have demonstrated that the combination of tezacaftor–ivacaftor is a slightly superior combination to its predecessor, lumacaftor–ivacaftor, with respect to an increase in FEV1, adverse event profile, and drug–drug interactions. It is also approved for a large number of non-F508del, residual function mutations that are predicted to respond based on in vitro testing. The horizon for continued improvements in CFTR-targeted treatments is promising, with three-drug combinations currently in Phase 3 clinical trials, and other drugs with novel mechanisms of action being studied. Within the next 5 years, the vast majority of patients with CF are expected to have a modulator approved for their genotype.

Original languageEnglish (US)
Pages (from-to)725-732
Number of pages8
JournalExpert Review of Respiratory Medicine
Volume12
Issue number9
DOIs
StatePublished - Sep 2 2018

Fingerprint

Cystic Fibrosis
Phase III Clinical Trials
Drug Combinations
Therapeutics
Ion Channels
Genotype
Clinical Trials
Safety
Mutation
Pharmaceutical Preparations

Keywords

  • CFTR
  • Cystic fibrosis
  • tezacaftor

ASJC Scopus subject areas

  • Immunology and Allergy
  • Pulmonary and Respiratory Medicine
  • Public Health, Environmental and Occupational Health

Cite this

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title = "Tezacaftor for the treatment of cystic fibrosis",
abstract = "Introduction: Cystic fibrosis (CF) is the most common, life-limiting autosomal recessive disease in Caucasians, and is caused by defects in production of the CFTR ion channel. Until recently, there were no available treatments targeting the disease-causing defects in CFTR but newly developed CFTR modulators are changing the course of disease in CF. The newest modulator, tezacaftor, is a CFTR corrector that was recently approved by the FDA to be used in combination with the first approved CFTR potentiator, ivacaftor. Areas covered: A detailed review of the clinical trials and published literature, focusing on safety and efficacy, leading to the approval of tezacaftor in CF. Expert commentary: Recent trials have demonstrated that the combination of tezacaftor–ivacaftor is a slightly superior combination to its predecessor, lumacaftor–ivacaftor, with respect to an increase in FEV1, adverse event profile, and drug–drug interactions. It is also approved for a large number of non-F508del, residual function mutations that are predicted to respond based on in vitro testing. The horizon for continued improvements in CFTR-targeted treatments is promising, with three-drug combinations currently in Phase 3 clinical trials, and other drugs with novel mechanisms of action being studied. Within the next 5 years, the vast majority of patients with CF are expected to have a modulator approved for their genotype.",
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Tezacaftor for the treatment of cystic fibrosis. / Sala, Marc A; Jain, Manu.

In: Expert Review of Respiratory Medicine, Vol. 12, No. 9, 02.09.2018, p. 725-732.

Research output: Contribution to journalArticle

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