"目录号: HY-14852
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Tafamidis(Fx1006A)是转甲状腺素蛋白动力稳定剂，能抑制淀粉样级连反应。
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Description
Tafamidis(Fx1006A) is a potent and selective transthyretin kinetic stabilizer that inhibits the amyloid cascade.Target: OthersTafamidis is a drug for the amelioration of transthyretin-related hereditary amyloidosis.Tafamidis functions by kinetic stabilization of the correctly folded tetrameric form of the transthyretin (TTR) protein. Tafamidis binds selectively and with negative cooperativity (K(d)s ~2 nM and ~200 nM) to the two normally unoccupied thyroxine-binding sites of the tetramer, and kinetically stabilizes TTR. Patient-derived amyloidogenic variants of TTR, including kinetically and thermodynamically less stable mutants, are also stabilized by tafamidis binding. The crystal structure of tafamidis-bound TTR suggests that binding stabilizes the weaker dimer-dimer interface against dissociation, the rate-limiting step of amyloidogenesis [1].
Clinical Trial
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Phase 2
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Familial Amyloid Polyneuropathy-ATTR-PN
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Phase 2-Phase 3
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Transthyretin-associated Amyloidosis With Polyneuropathy
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Phase 2
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Familial Amyloid Polyneuropathy
January 2007
Phase 2-Phase 3
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Pfizer
Mild Hepatic Dysfunction-Moderate Hepatic Dysfunction
November 2010
Phase 1
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Transthyretin Familial Amyloid Polyneuropathy
November 2011
Phase 3
NCT02791230
Pfizer
Transthyretin (TTR) Amyloid Cardiomyopathy
June 13, 2016
Phase 3
NCT02217813
Pfizer
Healthy Volunteers
October 2014
Phase 1
NCT00935012
Pfizer
TTR-CM
September 22, 2009
Phase 3
NCT02697864
Pfizer
Healthy
April 2016
Phase 1
NCT02746926
Pfizer
Healthy
April 2016
Phase 1
NCT01655511
Pfizer
TTR Cardiomyopathy
July 2012
Phase 1
NCT01369836
Pfizer
Healthy
July 2011
Phase 1
NCT01994889
Pfizer
Transthyretin (TTR) Amyloid Cardiomyopathy
December 9, 2013
Phase 3
NCT00925002
Pfizer
ATTR-PN
August 5, 2009
Phase 3
NCT02406560
Pfizer
Healthy Volunteers
April 2015
Phase 1
NCT02534740
Pfizer
Healthy
September 2015
Phase 1
NCT02189330
Pfizer
Healthy Volunteers
May 2014
Phase 1
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References
[1].Bulawa, C.E., et al., Tafamidis, a potent and selective transthyretin kinetic stabilizer that inhibits the amyloid cascade. Proc Natl Acad Sci U S A, 2012. 109(24): p. 9629-34.