| Name | Elexacaftor |
| Description | Elexacaftor (VX-445) is a cystic fibrosis transmembrane conductance regulator (CFTR) corrector. It promotes the processing and trafficking of CFTR, increases the amount of CFTR on the cell surface, and improves the processing and trafficking of Phe508del CFTR protein. |
| In vitro | Elexacaftor (VX-445) has the potential to treat cystic fibrosis and it also is a next-generation cystic fibrosis transmembrane conductance regulator (CFTR) corrector designed to restore Phe508del CFTR protein function. VX-445-Tezacaftor-VX-770 significantly improves Phe508del CFTR protein processing, trafficking, chloride transport. To a greater extent than any two of these agents in dual combination[2]. |
| In vivo | METHODS: Patients with the Phe508del–Phe508del genotype underwent a 4-week run-in of tezacaftor and ivacaftor and were randomized to receive either elexacaftor (VX-445) (200 mg orally daily) plus tezacaftor (100 mg daily) and ivacaftor (150 mg daily) or matching placebo plus tezacaftor and ivacaftor for 4 weeks.
RESULTS Treatment with elexacaftor (VX-445)-tezacaftor-ivacaftor significantly increased the percentage predicted FEV1 by up to 13.8 percentage points in patients with the Phe508del-MF genotype and up to 11.0 percentage points in patients with the Phe508del-Phe508del genotype who were already receiving tezacaftor-ivacaftor.[2] |
| Storage | store at low temperature,keep away from direct sunlight | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice. |
| Solubility Information | DMSO : 125 mg/mL (209.15 mM), Sonication is recommended.
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| Keywords | Fibrosis | Autophagy | CFTR | Cystic fibrosis transmembrane conductance regulator | Inhibitor | Elexacaftor | Cystic | VX 445 | inhibit | VX445 |
| Inhibitors Related | Stavudine | Xylitol | Myricetin | Sodium 4-phenylbutyrate | Hydroxychloroquine | Guanidine hydrochloride | Taurine | Curcumin | Oxyresveratrol | Paeonol | Naringin | Gefitinib |
| Related Compound Libraries | Bioactive Compound Library | Membrane Protein-targeted Compound Library | EMA Approved Drug Library | Anti-Cancer Clinical Compound Library | Drug Repurposing Compound Library | FDA-Approved Drug Library | Anti-Cancer Approved Drug Library | Bioactive Compounds Library Max | Ion Channel Targeted Library | Anti-Cancer Drug Library |
United States
