Ivacaftor-d4 (VX-770-d4) is the deuterium labeled-Ivacaftor (HY-13017). Ivacaftor is a potent and orally active CFTR potentiator, targeting G551D-CFTR and F508del-CFTR with EC₅₀s of 100 nM and 25 nM, respectively.

Ivacaftor (10 μM) 使 ABCB4-G535D 的 PC 分泌活性增加 3 倍，ABCB4-G536R 增加 13.7 倍，ABCB4-S1076C 增加 6.7 倍，ABCB4-S1176L 增加 9.4 倍，ABCB4-增加 5.7 倍 G1178S。 Ivacaftor 纠正了 ABCB4 突变体的功能缺陷。
与 R1162X CFTR 细胞相比，Ivacaftor (10 μM) 显着增加 W1282X 表达细胞中的 CFTR 活性^{。
Ivacaftor 对测试的 160 个靶标没有显着活性，包括 GABA_A 苯二氮卓受体。 Ivacaftor 增加氯化物分泌，EC₅₀ 值为 0.236 ± 0.200 μM，与 F508del HBEs 相比，效力提高 10 倍。
在重组细胞中，Ivacaftor 在 F508del 加工突变和 G551D 门控突变中增加 CFTR 通道开放概率 (Po)。 Ivacaftor 在温度校正的 F508del-FRT 细胞中增加毛喉素刺激的 I_T 约 6 倍，EC₅₀ 为 25 nM[4]。}

西域 has not independently confirmed the accuracy of these methods. They are for reference only.

Ivacaftor (1-200 mg/kg, 口服) 在大鼠体内表现出良好的口服生物利用度。

西域 has not independently confirmed the accuracy of these methods. They are for reference only.

Ivacaftor (1-200 mg/kg, 口服) 在大鼠体内表现出良好的口服生物利用度。

西域 has not independently confirmed the accuracy of these methods. They are for reference only.

• . Delaunay JL, et al. Functional defect of variants in the adenosine triphosphate-binding sites of ABCB4 and their rescue by the cystic fibrosis transmembrane conductance regulator potentiator, ivacaftor (VX-770). Hepatology. 2017 Feb;65(2):560-570  [Content Brief]

  . Mutyam V, et al. Therapeutic benefit observed with the CFTR potentiator, ivacaftor, in a CF patient homozygous for the W1282X CFTR nonsense mutation. J Cyst Fibros. 2017 Jan;16(1):24-29  [Content Brief]

  . Hadida S, et al. Discovery of N-(2,4-di-tert-butyl-5-hydroxyphenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide (VX-770, ivacaftor), a potent and orally bioavailable CFTR potentiator. J Med Chem. 2014 Dec 11;57(23):9776-9  [Content Brief]

  [4]. Van Goor F, et al. Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770. Proc Natl Acad Sci U S A. 2009 Nov 3;106(44):18825-30.  [Content Brief]