エリグルスタット酒石酸塩

エリグルスタット酒石酸塩 化学特性,用途語,生産方法

• 効能

  ライソゾーム病治療薬, グルコシルセラミド合成阻害薬

• 商品名

  サデルガ (サノフィ)

• 説明

  Eliglustat tartrate, developed and marketed by Genzyme Corporation (a subsidiary of Sanofi), was approved by the US FDA in August 2014 for the treatment of nonneuropathic (type 1) Gaucher disease (GD1) in both treatment-naive and treatment-experienced adult patients. It is the first oral treatment to be approved for first-line use in patients with Gaucher disease type 1, which is a rare lysosomal storage disease characterized by accumulation of lipid glucosylceramide (GL-1) due to insufficient production of the enzyme glucosylceramidase. Clinical complications include hepatosplenomegaly, anemia, thrombocytopenia, and bone involvement. Eliglustat is a specific inhibitor of glucosylceramide synthase with an IC50 of 10 ng/mL and acts as substrate reduction therapy for GD1; it has demonstrated non-inferiority to enzyme replacement therapy, which is the current standard of care, in Phase III trials.

• 使用

  Eliglustat Tartrate functions as an oral agent for treatment of Gaucher disease type 1, and lysosomal storage disorders in human. It also inhibits UDP-glucosylceramide synthase in mice thereby preventing Gaucher disease-associated with B-cell malignancy.

• 定義

  ChEBI: A tartrate that is the hemitartrate salt of eliglustat. A ceramide glucosyltransferase inhibitor used (as its tartrate salt) for treatment of Gaucher's disease.

• in vitro

  Eliglustat tartrate (Eliglustat hemitartrate) shows good potency with an IC50 of 24 nM and specificity against the target enzyme. Incubating K562 or B16/F10 cells for 72 h with increasing amounts of Genz-112638 (0.6-1000 nM) results in a dose-dependent reduction of both GM1 and GM3 cell surface levels. The mean IC50 value for inhibiting the cell surface presentation of GM1 in K562 cells was 24 nM (range 14-34 nM), and that for GM3 in B16/F10 cells was 29 nM (range 12-48 nM).

エリグルスタット酒石酸塩 生産企業

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