Name: Ivacaftor (VX-770)
Brand: Selleck Chemicals
SKU: S1144
Availability: InStock
Rating: 5 (279 reviews)

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Quality Control

Batch: Purity: 99.97%

99.97

Products Often Used Together with Ivacaftor (VX-770)

Tezacaftor (VX-661)

Tezacaftor specifically targets F508del CFTR whereas, this compound targets both G551D-CFTR and F508del-CFTR.

VX-445 (Elexacaftor)

Elexacaftor exhibits multiplicative synergy with it in potentiating Class III and IV CFTR mutations.

CFTRinh-172

This compound is a CFTR potentiator, whereas CFTRinh-172 is an CFTR inhibitor.

Icenticaftor (QBW251)

It and Icenticaftor are small-molecule channel potentiators of CFTR that are under clinical trials for their roles in chronic obstructive pulmonary disease.

Related Targets	CRM1 CD markers AChR Calcium Channel Sodium Channel Potassium Channel GABA Receptor TRP Channel ATPase GluR
Other CFTR Inhibitors	Tezacaftor (VX-661) VX-445 (Elexacaftor) Vanzacaftor (VX-121) VX-561(CTP-656) CFTRinh-172 GLPG1837 GlyH-101 FDL169 Galicaftor (ABBV-2222) IOWH032

Cell Culture, Treatment & Working Concentration

Cell Lines	Assay Type	Concentration	Incubation Time	Activity Description	PMID
HBE	Function Assay	10 μM	10 min	augments CFTR-dependent ion transport	24106801
CFBE41o-	Function Assay	10 µM		induces robust increases in anion transport	22768130
HBE	Function Assay	10 µM		augments CFTR-dependent anion transport activity	22768130
HBE	Function Assay	10 µM	24 h	induces a modest but significant increase in ASL depth	22768130
HBE	Function Assay	10 µM		potentiates CFTR-dependent Isc, regardless of prior administration of CSE	22768130
HBE	Function Assay	10 µM		partially restores depletion of ASL depth in CSE treated monolayers	22768130
mouse NIH-3T3 cells	Function assay		30 mins	Potentiation of human CFTR F508del mutant expressed in mouse NIH-3T3 cells after 30 mins by fluorescent voltage sensing optical assay, EC50 = 0.003 μM.	25441013
human bronchial epithelial cells	Function assay			Potentiation of human CFTR F508del/G551D mutant in human bronchial epithelial cells by Ussing chambers recording technique, EC50 = 0.022 μM.	25441013
human CFBE41o cells	Function assay		10 mins	Potentiation of CFTR F508del mutant (unknown origin) expressed in human CFBE41o cells incubated for 10 mins in presence of forskolin measured for 7 secs by YFP halide assay, EC50 = 0.126 μM.	29148763
human bronchial epithelial cells	Function assay			Potentiation of human CFTR F508del mutant in human bronchial epithelial cells by Ussing chambers recording technique, EC50 = 0.236 μM.	25441013
HEK293 cells	Function assay		10 mins	Potentiation of CFTR G551D mutant (unknown origin) expressed in HEK293 cells incubated for 10 mins in presence of forskolin measured for 2 mins by YFP halide assay, EC50 = 1.3 μM.	29148763
NRK-49F cells	Function assay			Inhibition of TGF-beta1-induced total collagen accumulation in rat NRK-49F cells, IC50 = 4 μM.	25467157
DAOY cells	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for DAOY cells	29435139
NB-EBc1 cells	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for NB-EBc1 cells	29435139
MG 63 (6-TG R) cells	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for MG 63 (6-TG R) cells	29435139
RD cells	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for RD cells	29435139
SK-N-MC cells	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for SK-N-MC cells	29435139
Saos-2 cells	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for Saos-2 cells	29435139
Click to View More Cell Line Experimental Data

Solubility

In vitro
Batch:

DMSO : 79 mg/mL (201.27 mM)
(Moisture-contaminated DMSO may reduce solubility. Use fresh, anhydrous DMSO.)

Water : Insoluble

Ethanol : Insoluble

Molarity Calculator

Mass	Concentration	Volume	Molecular Weight
Mass value Mass unit	Concentration value Concentration unit	Volume value Volume unit	Molecular weight (g/mol)

Dilution Calculator Molecular Weight Calculator

In vivo batch selection In vivo Batch:
Homogeneous suspension	CMC-NA	≥5mg/ml	Taking the 1 mL working solution as an example, add 5 mg of this product to 1 ml of CMC-Na solution, mix evenly to obtain a homogeneous suspension with a final concentration of 5 mg/ml.
Homogeneous suspension	CMC-NA	≥5mg/ml	Taking the 1 mL working solution as an example, add 5 mg of this product to 1 ml of CMC-Na solution, mix evenly to obtain a homogeneous suspension with a final concentration of 5 mg/ml.
Clear solution	5%DMSO 1 40%PEG300 2 5%Tween80 3 50%ddH2O Validated by Selleck labs. Should you need adjustments to this formulation, contact our sales team for custom testing.	5.500mg/ml (14.01mM)	Taking the 1 mL working solution as an example, add 50 μL of 110 mg/ml clarified DMSO stock solution to 400 μL of PEG300, mix evenly to clarify it; add 50 μL of Tween80 to the above system, mix evenly to make it clear; then continue to add 500 μL of ddH2O to adjust the volume to 1 mL. The mixed solution should be used immediately for optimal results.
Homogeneous suspension	CMC-NA	≥5mg/ml	Taking the 1 mL working solution as an example, add 5 mg of this product to 1 ml of CMC-Na solution, mix evenly to obtain a homogeneous suspension with a final concentration of 5 mg/ml.
Homogeneous suspension	CMC-NA	≥5mg/ml	Taking the 1 mL working solution as an example, add 5 mg of this product to 1 ml of CMC-Na solution, mix evenly to obtain a homogeneous suspension with a final concentration of 5 mg/ml.
Homogeneous suspension	CMC-NA	≥5mg/ml	Taking the 1 mL working solution as an example, add 5 mg of this product to 1 ml of CMC-Na solution, mix evenly to obtain a homogeneous suspension with a final concentration of 5 mg/ml.

In vivo Formulation Calculator (Clear solution)

Step 1: Enter information below (Recommended: An additional animal making an allowance for loss during the experiment)

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Working concentration: mg/ml;

Method for preparing DMSO master liquid: mg drug pre-dissolved in μL DMSO ( Master liquid concentration mg/mL, Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug. )

Method for preparing in vivo formulation: Take μL DMSO master liquid, next addμL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH₂O, mix and clarify.

Method for preparing in vivo formulation: Take μL DMSO master liquid, next add μL Corn oil, mix and clarify.

Note: 1. Please make sure the liquid is clear before adding the next solvent.
2. Be sure to add the solvent(s) in order. You must ensure that the solution obtained, in the previous addition, is a clear solution before proceeding to add the next solvent. Physical methods such
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Chemical Information, Storage & Stability

Molecular Weight	392.49	Formula	C₂₄H₂₈N₂O₃	Storage (From the date of receipt)	3 years-20°Cpowder
CAS No.	873054-44-5	Download SDF		Storage of Stock Solutions	1 year-80°Cin solvent 1 month-20°Cin solvent
Synonyms	VX-770			Smiles	CC(C)(C)C1=CC(=C(C=C1NC(=O)C2=CNC3=CC=CC=C3C2=O)O)C(C)(C)C

Mechanism of Action

Features	The first potent and orally available CFTR potentiator to enter human clinical trials.
Targets/IC₅₀/Ki	F508del-CFTR (Fisher rat thyroid cells) 25 nM(EC50) G551D-CFTR (Fisher rat thyroid cells) 100 nM(EC50)
In vitro	Ivacaftor (VX-770) (10 μM) significantly increases the forskolin-stimulated Cl^- secretion (I_T) by ~4-fold with an EC50 of 100 nM in the recombinant Fisher rat thyroid (FRT) cells expressing G551D gating mutation of CFTR, and by ~6-fold with an EC50 of 25 nM in the recombinant cells expressing temperature-corrected F508del processing mutation of CFTR. Consistent with the increases in the forskolin-stimulated I_T, this compound increases the open probability (P_o) of G551D-, F508del-, and wild-type CFTR by ~6-fold, ~5-fold and ~2-fold, respectively, indicating that it acts directly on CFTR to increase its gating activity. In primary cultured human CF bronchial epithelia (HBE) carrying the G551D and F508del CFTR mutations, Ivacaftor potently increases the forskolin-stimulated I_T by ~10-fold from 5% to a maximum level of 48% of that measured in non-CF HBE, with an EC50 of 236 nM displaying ~70-fold more potency compared with the commonly used CFTR potentiator genistein, which has an EC50 of 16 μM. In HBE with F508del homozygous CFTR, it causes a significant increase in the forskolin-stimulated I_T with an EC50 of 22 nM, to a less extent from 4% to 16% of non-CF HBE compared with the effect in G551D/F508del HBE. Due to CFTR potentiation, this compound inhibits excessive ENaC-mediated Na⁺ and fluid absorption with an IC50 of 43 nM, and decreases the response, resulting in an increase in the surface fluid and cilia beat frequency (CBF) in G551D/F508del HBE.
Kinase Assay	Ussing Chamber Recordings
Kinase Assay	The effect of Ivacaftor (VX-770) on CFTR-mediated Cl^- secretion is characterized by measuring the CFTR-mediated I_T in chambers using recombinant Fisher rat thyroid (FRT) cells expressing G551D, or F508del CFTR. Cells are grown on Costar Snapwell cell culture inserts maintained at 37 °C before recording. The cell culture inserts are mounted into an Ussing chamber to record I_T in the voltage-clamp mode (V_hold = 0 mV). For FRT cells, the basolateral membrane is a basolateral to apical Cl^- gradient is established. The basolateral bath solution contains 135 mM NaCl, 1.2 mM CaCl₂, 1.2 mM MgCl₂, 2.4 mM K₂HPO₄, 0.6 mM KHPO₄, 10 mM N-2-hydroxyethylpiperazine-N
References	[1] https://pubmed.ncbi.nlm.nih.gov/19846789/ [2] https://pubmed.ncbi.nlm.nih.gov/25441013/

Applications

Methods	Biomarkers	Images	PMID
Western blot	PPARγ / pERK NLRP3 Rδf508		30498130
Immunofluorescence	F-actin		30498130

Clinical Trial Information

(data from https://clinicaltrials.gov, updated on 2024-05-22)

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT06331000	Not yet recruiting	Cystic Fibrosis	University Hospital Strasbourg France	March 2024	--
NCT05519020	Recruiting	Cystic Fibrosis	Sheffield Teaching Hospitals NHS Foundation Trust	July 27 2022	--
NCT04254705	Withdrawn	Cystic Fibrosis	Universitaire Ziekenhuizen KU Leuven\|Vertex Pharmaceuticals Incorporated\|KU Leuven\|University of Lisbon	March 1 2020	Not Applicable
NCT03085485	Completed	Chronic Obstructive Pulmonary Disease\|Chronic Bronchitis	University of Alabama at Birmingham\|National Heart Lung and Blood Institute (NHLBI)\|Vertex Pharmaceuticals Incorporated	March 16 2017	Phase 2

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Ivacaftor (VX-770) CFTR potentiator

Chemical Structure

Molecular Weight: 392.49