生物活性

• 描述Eliglustat is a selective inhibitor of glucosylceramide synthase that is extensively metabolized by CYP2D6 and, to a lesser extent by CYP3A4; it is also an inhibitor of the P-gp transporter^[3]. Eliglustat is an oral inhibitor of glucosylceramide synthase which is used in the therapy of type 1 Gaucher disease^[4]. Eliglustat inhibits glucosylceramide synthase, thereby decreasing production of the substrate glucosylceramide and reducing its accumulation^[5]. Administration of eliglustat to Swiss CD-1 mice at 0, 10, 25 or 75 mg/kg/day for 104 weeks by dietary admixture did not influence survival or bodyweight evolution, or produce any clinical indication of poor condition. Eliglustat was not carcinogenic to mice or rats in standard lifetime bioassays^[6].
• 临床研究

• NCT号	适应症或疾病	临床期	招募状态	预计完成时间	地点
  NCT01074944	Gaucher Disease	Phase 3	Completed	-	-
  NCT00358150	Gaucher Disease, Type 1 ... more >> Cerebroside Lipidosis Syndrome Glucocerebrosidase Deficiency Disease Glucosylceramide Beta-Glucosidase Deficiency Disease Gaucher Disease, Non-Neuronopathic Form Collapse <<	Phase 2	Completed	-	United States, New York ... more >> New York University New York, New York, United States New York, New York, United States Argentina Aprillus Asistencia e Investigación Buenos Aires, Argentina Hospital de Oncologia Maria Curie Buenos Aires, Argentina IMAI Buenos Aires, Argentina Instituto Argentino de Diagnostico y Tratamiento (IADT) Buenos Aires, Argentina Buenos Aires, Argentina Hospital Ramos Mejia Ciudad Autonoma de Buenos Aires, Argentina Israel Rambam Medical Center Haifa, Israel Haifa, Israel Sha'are Zedek Medical Centre Jerusalem, Israel Jerusalem, Israel Italy Universita degli Studi di Milano Milano, Italy Mexico Instituto Mexicano del Seguro Social D.f., Mexico Mexico City, Mexico Russian Federation Hematology Research Center of Ministry of Healthcare of the Russian Federation Moscow, Russian Federation Moscow, Russian Federation Collapse <<
  NCT00943111	-	-	Completed	-	-

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• 参考文献

• [1] Shayman JA. ELIGLUSTAT TARTRATE: Glucosylceramide Synthase Inhibitor Treatment of Type 1 Gaucher Disease. Drugs Future. 2010 Aug 1;35(8):613-620.

  [2] Yew NS, Zhao H, et al. Increased hepatic insulin action in diet-induced obese mice following inhibition of glucosylceramide synthase. PLoS One. 2010 Jun 21;5(6):e11239.

  [3] Almeida-Calpe A, López de Frutos L, Medrano-Engay B, García-García CB, Ribate MP, Giraldo P. Metabolizing profile of the cytochrome pathway CYP2D6, CYP3A4 and the ABCB 1 transporter in Spanish patients affected by Gaucher disease. Chem Biol Interact. 2021 Aug 25;345:109527

  [4] LiverTox: Clinical and Research Information on Drug-Induced Liver Injury [Internet]. Bethesda (MD): National Institute of Diabetes and Digestive and Kidney Diseases; 2012–. Eliglustat. 2018 Mar 5

  [5] Balwani M, Burrow TA, Charrow J, Goker-Alpan O, Kaplan P, Kishnani PS, Mistry P, Ruskin J, Weinreb N. Recommendations for the use of eliglustat in the treatment of adults with Gaucher disease type 1 in the United States. Mol Genet Metab. 2016 Feb;117(2):95-103

  [6] Dagher R, Watzinger M, Chevalier G, Thirion-Delalande C, Gervais F, Forster R. Carcinogenicity testing of eliglustat in mice and rats. Regul Toxicol Pharmacol. 2015 Oct;73(1):401-12

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