Drug class

Cystic Fibrosis Transmemb. Conduct. Reg. (CFTR) Potentiators

Cystic fibrosis transmembrane conductance regulator (CFTR) potentiators are a targeted class of small-molecule CFTR modulators developed to improve defective chloride and bicarbonate transport across epithelial membranes. Read more about Cystic Fibrosis Transmemb. Conduct. Reg. (CFTR) Potentiators

6 drugs in this class 1 manufacturers

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Drugs in Cystic Fibrosis Transmemb. Conduct. Reg. (CFTR) Potentiators

Showing 1–6 of 6 drugs

KALYDECO (ivacaftor)

CYSTIC FIBROSIS TRANSMEMB. CONDUCT. REG. (CFTR) POTENTIATORS · CYSTIC FIBROSIS WITH RESPONSIVE CFTR MUTATION
VERTEX PHARMACEUTICAL

ORKAMBI (lumacaftor/ivacaftor)

CYSTIC FIBROSIS TRANSMEMB. CONDUCT. REG. (CFTR) POTENTIATORS · CYSTIC FIBROSIS WITH HOMOZYGOUS F508DEL MUTATION IN CFTR GENE
VERTEX PHARMACEUTICAL

SYMDEKO (tezacaftor/ivacaftor)

CYSTIC FIBROSIS TRANSMEMB. CONDUCT. REG. (CFTR) POTENTIATORS · CYSTIC FIBROSIS WITH HOMOZYGOUS F508DEL MUTATION IN CFTR GENE ·+1
VERTEX PHARMACEUTICAL

ORKAMBI (lumacaftor/ivacaftor)

CYSTIC FIBROSIS TRANSMEMB. CONDUCT. REG. (CFTR) POTENTIATORS · CYSTIC FIBROSIS WITH HOMOZYGOUS F508DEL MUTATION IN CFTR GENE
VERTEX PHARMACEUTICAL
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About Cystic Fibrosis Transmemb. Conduct. Reg. (CFTR) Potentiators.

Cystic fibrosis transmembrane conductance regulator (CFTR) potentiators are a targeted class of small-molecule CFTR modulators developed to improve defective chloride and bicarbonate transport across epithelial membranes.

They act directly on CFTR ion channels localized at the cell surface by increasing their open-channel probability, thereby facilitating ion conductance and mucosal hydration.

This therapeutic class is primarily indicated for the treatment of cystic fibrosis in patients harboring responsive CFTR gene mutations—notably gating defects or residual function variants—either as monotherapy or combined with CFTR correctors to restore channel function.