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ivacaftor + lumacaftor + ivacaftor + tezacaftor + ivacaftor + elexacaftor + tezacaftor + ivacaftor + vanzacaftor + tezacaftor + deutivacaftor
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- Unknown
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- Small Molecules
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- Oral
01
Overview
This entry refers to a comprehensive set of cystic fibrosis transmembrane conductance regulator (CFTR) modulators, including both approved therapies and investigational combinations. The list encompasses ivacaftor (a CFTR potentiator) and various combination regimens: lumacaftor/ivacaftor, tezacaftor/ivacaftor, elexacaftor/tezacaftor/ivacaftor, and the next-generation triple combination vanzacaftor/tezacaftor/deutivacaftor. These agents are designed to address the underlying protein defect in cystic fibrosis by either improving the gating of the CFTR channel at the cell surface (potentiators) or by acting as pharmacological chaperones (correctors) to increase the processing and trafficking of defective CFTR proteins to the plasma membrane. Developed primarily by Vertex Pharmaceuticals, these therapies are tailored to specific CFTR mutations, such as the common F508del mutation.
- Brand names
- OrkambiSymkevi
- Other names
- CFTR modulators
02
Targets
CFTR (Cystic fibrosis transmembrane conductance regulator)
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