Synthetic Pharmaceutical Ivacaftor Powder CAS 873054-44-5 Vx-770

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The Introductction of Ivacaftor :

Ivacaftor is a new therapeutic agent that acts at the cystic fibrosis transmembrane conductance regulator (CFTR) channel to alter activity. It is approved for use in patients 6 years and older with cystic fibrosis who have at least 1 G551D mutation in the CFTR gene. It is unlike any other current pharmacologic agent for cystic fibrosis in that it specifically targets the gene defect associated with cystic fibrosis as opposed to treating resulting symptomology. Mucoactive agents, antibiotics, inhaled beta agonists, and other anti-inflammatory agents are currently the mainstay of cystic fibrosis treatment but can be associated with several side effects in addition to cumbersome frequency of administration. Ivacaftor's oral dosing regimen offers a more convenient treatment option.

The Application & Function of Ivacaftor:

the treatment of cystic fibrosis (CF) in patients who have the G551D mutation of the CF transmembrane regulator (CFTR) and are at least 6 years old. Ivacaftor (also known as VX-770) is a CFTR potentiator that increases the open probability of CFTR, thus increasing chloride secretion particularly in the 5% of CF patients with the G551D/F508 gating/ processing mutation. Ivacaftor was discovered by medicinal chemistry optimization of a lead scaffold identified through high-throughput screening of a 228,000 compound collection. In cultured bronchial epithelial cells from a CF patient with F508del, ivacaftor increased chloride secretion (EC50=81 nM). Preparation of ivacaftor is accomplished via a multistep synthesis oftwointermediates, 4-oxo-1,4-dihydroquinoline-3-carboxylic acid and 5-amino-di-tert-butylphenyl methyl carbonate, which are coupled using propanes phosphonic acid anhydride (T3P) to afford the amide; deprotection of the phenole ethen provides ivacaftor.
UsesIvacaftor (VX-770, Kalydeco) is a potentiator of CFTR targeting G551D-CFTR and F508del-CFTR with EC50 of 100 nM and 25 nM, respectively

Uses: Ivacaftor is used in the treatment of cystic fibrosis.

The COA of Ivacaftor: