Profile of tezacaftor/ivacaftor combination and its potential in the treatment of cystic fibrosis

Cystic fibrosis (CF) is a life-limiting autosomal recessive disease caused by dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) ion channel. Management of CF has traditionally relied upon managing complications of CFTR protein dysfunction and this has led to a steady impr...

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Bibliographic Details
Published in:Therapeutics and clinical risk management 2019-01, Vol.15, p.1029-1040
Main Authors: Shiferaw, Dejene, Faruqi, Shoaib
Format: Article
Language:English
Subjects:
CFTR modulators
CFTR potentiators
Chloride
Cystic Fibrosis
Defects
Ivacaftor
Mutation
Proteins
Review
Teaching hospitals
Tezacaftor
triple therapy
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Summary:Cystic fibrosis (CF) is a life-limiting autosomal recessive disease caused by dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) ion channel. Management of CF has traditionally relied upon managing complications of CFTR protein dysfunction and this has led to a steady improvement in survival of CF patients. However, the landscape of CF care has changed substantially over the last decade with the discovery of CFTR modulators that aim to increase or potentially restore the function of the disease-causing CFTR protein. This narrative review summarizes the development of CFTR therapies so far with emphasis on tezacaftor/ivacaftor combination therapy. We have also summarized the Phase II results of triple combination therapy which promises an effective CFTR modulator therapy for more than 90% of CF patients.
ISSN:1176-6336
1178-203X
1178-203X
DOI:10.2147/TCRM.S165027