1152311-62-0 Usage
Description
VX-661, also known as Tezacaftor, is an oral medication developed by Vertex Pharmaceuticals for the treatment of cystic fibrosis (CF). It is designed to promote the maturation of delta F508 mutants of the cystic fibrosis transmembrane conductance regulator (CFTR), a class of CFTR mutation characterized by impaired processing of misfolded CFTR proteins and reduced accumulation of the protein at the cell surface. VX-661 facilitates the trafficking of CFTR to the epithelial cell membrane and is intended for use in combination with the CFTR potentiator ivacaftor to stimulate both CFTR accumulation and opening at the apical epithelial surface.
Uses
Used in Cystic Fibrosis Treatment:
VX-661 is used as a combination therapy with Ivacaftor for the treatment of patients with cystic fibrosis. It is approved in the U.S. and Canada for CF patients, ages 12 and older, who have two copies of the F508del mutation in the CFTR gene and one minimal function mutation. The combination therapy formulation, known as Symdeko (tezacaftor plus ivacaftor), is also approved and marketed in the EU as Symkevi. VX-661 is not approved as a stand-alone treatment but is used in conjunction with Ivacaftor to enhance the therapeutic outcomes for CF patients.
Biochem/physiol Actions
VX-661 is another cystic fibrosis transmembrane conductance regulator (CFTR) corrector in development for the treatment of cystic fibrosis.
in vitro
VX-661, is CFTR modulator that is potentially useful for treatment of cystic fibrosis. VX-661 corrects F508del-CFTR trafficking and increases F508del-CFTR protein activity in vitro.
references
[1] s. donaldson, j. pilewski, m. griese, q. dong, p.-s. lee, for the vx11–661-101 study group. ws7.3 vx-661, an investigational cftr corrector, in combination with ivacaftor, a cftr potentiator, in patients with cf and homozygous for the f508del-cftr mutation: interim analysis. journal of cystic fibrosis, volume 12, supplement 1, june 2013, page s14
Check Digit Verification of cas no
The CAS Registry Mumber 1152311-62-0 includes 10 digits separated into 3 groups by hyphens. The first part of the number,starting from the left, has 7 digits, 1,1,5,2,3,1 and 1 respectively; the second part has 2 digits, 6 and 2 respectively.
Calculate Digit Verification of CAS Registry Number 1152311-62:
(9*1)+(8*1)+(7*5)+(6*2)+(5*3)+(4*1)+(3*1)+(2*6)+(1*2)=100
100 % 10 = 0
So 1152311-62-0 is a valid CAS Registry Number.
InChI:InChI=1S/C26H27F3N2O6/c1-24(2,13-33)22-8-14-7-18(17(27)10-19(14)31(22)11-16(34)12-32)30-23(35)25(5-6-25)15-3-4-20-21(9-15)37-26(28,29)36-20/h3-4,7-10,16,32-34H,5-6,11-13H2,1-2H3,(H,30,35)/t16-/m1/s1
1152311-62-0Relevant articles and documents
Pharmaceutical compositions for the treatment of cystic fibrosis transmembrane conductance regulator mediated diseases
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, (2021/04/21)
The present invention features compositions comprising a plurality of therapeutic agents wherein the presence of one therapeutic agent enhances the properties of at least one other therapeutic agent. In one embodiment, the therapeutic agents are cystic fibrosis transmembrane conductance regulators (CFTR) such as a CFTR corrector or CFTR potentiator for the treatment of CFTR mediated diseases such as cystic fibrosis. Methods and kits thereof are also disclosed.
NOVEL PROCESSES FOR PREPARATION OF TEZACAFTOR
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, (2021/08/14)
The present invention generally relates to processes for preparation of Tezacaftor and pharmaceutical composition comprising the same. The present invention also encompasses novel intermediates of tezacaftor, processes for its preparation and use of said intermediates in the preparation of tezacaftor.
METHODS OF TREATMENT FOR CYSTIC FIBROSIS
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, (2020/06/05)
This application describes methods of treating cystic fibrosis or a CFTR mediated disease comprising administering Compound I or a pharmaceutically acceptable salt thereof. (I) The application also describes pharmaceutical compositions comprising Compound I or a pharmaceutically acceptable salt thereof and optionally comprising one or more additional CFTR modulating agents.