N-BUTYLDEOXYNOJIRIMYCIN Struktur

N-BUTYLDEOXYNOJIRIMYCIN

Bezeichnung:N-BUTYLDEOXYNOJIRIMYCIN

CAS-Nr72599-27-0

Englisch Name:N-BUTYLDEOXYNOJIRIMYCIN

CBNumberCB0125257

SummenformelC10H21NO4

Molgewicht219.28

MOL-Datei72599-27-0.mol

N-BUTYLDEOXYNOJIRIMYCIN physikalisch-chemischer Eigenschaften

Schmelzpunkt	126-127℃
alpha	D25 -15.9° (c = 0.77 in water)
Siedepunkt	394.7±42.0 °C(Predicted)
Dichte	1.234
RTECS-Nr.	TN4350150
storage temp.	2-8°C
Löslichkeit	DMSO 44 mg/mL (200.66 mM) Ethanol 22 mg/mL (100.33 mM) Water 44 mg/mL (200.66 mM)
Aggregatzustand	Powder
pka	13.72±0.70(Predicted)
Farbe	White
Wasserlöslichkeit	Soluble in water at 10mg/ml
InChIKey	UQRORFVVSGFNRO-UTINFBMNSA-N

Sicherheit

WGK Germany	3
HS Code	2933399990

Gefahreninformationscode (GHS)

N-BUTYLDEOXYNOJIRIMYCIN Chemische Eigenschaften,Einsatz,Produktion Methoden

Beschreibung
Miglustat is an N-alkylated iminosugar, launched as an oral treatment for mild to moderate type 1 Gaucher’s disease in adult patients for whom enzyme replacement therapy is not a therapeutic option. It is readily synthesized from D-glucose in three steps by first converting to N-butylglucamine via reductive amination with butylamine, followed by a microbial oxidation to an aminofuranose intermediate and subsequent reductive cyclization. Type 1 Gaucher’s disease is a metabolic disorder caused by the lysosomal accumulation of certain glycosphingolipids (GSLs) as a result of deficiency in their degradation. Enlargement of the liver and spleen, low blood platelet and bone lesions are among the key symptoms of this disease. Miglustat acts by inhibiting glucosylceramide synthase, a glucosyl transferase enzyme in the biosynthesis of most GSLs, which results in the lowering of GSLs to a level that can be effectively cleared. Up to 50% reduction in liver and splenocyte GSL levels are achieved in mice by long-term administration of Miglustat (600– 1800 mg/kg/day for 118 days). Miglustat, dosed at 50 and 100 mg in Gaucher patients, exhibits dose proportionate pharmacokinetics (t_max=2.5 h, t_1/2=6 to 7 h) and ＞90% oral bioavailability. Steady-state plasma levels are reached after 4–6 weeks of treatment. Miglustat is not significantly metabolized in humans and the major route of excretion is renal. In clinical trials, efficacy was demonstrated by significant reductions in liver and spleen volumes (12 and 19%, respectively) at 12 months and increase in hemoglobin and platelet count (0.91 g/dL and 13.6×10⁹/l, respectively) at 24 months. Miglustat is generally well tolerated by patients and the most common side effects are diarrhea and weight loss.
Originator
G.D. Searle (Pfizer) (US)
Verwenden
An alpha-glucosidase Inhibitor
Verwenden
Treatment of glycolipid storage diseases.
Definition
ChEBI: A hydroxypiperidine that is deoxynojirimycin in which the amino hydrogen is replaced by a butyl group.
Trademarks
Zavesca(Actelion);Vevesca.
Allgemeine Beschreibung
N-Butyldeoxynojirimycin is an alkylated product of imino sugar deoxynojirimycin.
Biologische Aktivität
Orally active α -glucosidase I and II and ceramide-specific glycosyltransferase inhibitor. Rescues trafficking-deficient F508del-CTFR in human airway epithelial cells via inhibition of ER α -glucosidases I and II. Also has broad spectrum antiviral activity.
Biochem/physiol Actions
N-Butyldeoxynojirimycin is an inhibitor of glucosyltransferase and α-glucosidases. N-Butyldeoxynojirimycin, also known as misglustat, reduces glycolipid levels by substrate reduction therapy (SRT) and is effectively used for the treatment of glycosphingolipid lysosomal storage disorder, Gaucher disease.

N-BUTYLDEOXYNOJIRIMYCIN Anbieter Lieferant Produzent Hersteller Vertrieb Händler.

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