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 language | English (US) |
|---|---|
| Pages (from-to) | 725-732 |
| Number of pages | 8 |
| Journal | Expert Review of Respiratory Medicine |
| Volume | 12 |
| Issue number | 9 |
| DOIs | |
| State | Published - Sep 2 2018 |
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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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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 journal › Article
TY - JOUR
T1 - Tezacaftor for the treatment of cystic fibrosis
AU - Sala, Marc A
AU - Jain, Manu
PY - 2018/9/2
Y1 - 2018/9/2
N2 - 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.
AB - 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.
KW - CFTR
KW - Cystic fibrosis
KW - tezacaftor
UR - http://www.scopus.com/inward/record.url?scp=85051985934&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85051985934&partnerID=8YFLogxK
U2 - 10.1080/17476348.2018.1507741
DO - 10.1080/17476348.2018.1507741
M3 - Article
C2 - 30073878
AN - SCOPUS:85051985934
VL - 12
SP - 725
EP - 732
JO - Expert Review of Respiratory Medicine
JF - Expert Review of Respiratory Medicine
SN - 1747-6348
IS - 9
ER -