Miglustat
Brand names,
Miglustat
Analogs
Miglustat
Brand Names Mixture
Miglustat
Chemical_Formula
C10H21NO4
Miglustat
RX_link
http://www.rxlist.com/cgi/generic3/zavesca.htm
Miglustat
fda sheet
Miglustat
msds (material safety sheet)
Miglustat
Synthesis Reference
No information avaliable
Miglustat
Molecular Weight
219.278 g/mol
Miglustat
Melting Point
169 - 172 oC
Miglustat
H2O Solubility
Highly soluble in water (>1000 mg/mL as a free base).
Miglustat
State
Solid
Miglustat
LogP
-1.087
Miglustat
Dosage Forms
Capsules (100-mg) for oral administration
Miglustat
Indication
For the treatment of adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option (e.g. due to constraints such as allergy, hypersensitivity, or poor venous access).
Miglustat
Pharmacology
Miglustat is an N-alkylated imino sugar, a synthetic analogue of D-glucose. Miglustat is an inhibitor of the enzyme glucosylceramide synthase, which is a glucosyl transferase enzyme responsible for the first step in the synthesis of most glycosphingolipids.
Miglustat
Absorption
Mean oral bioavailability is 97%.
Miglustat
side effects and Toxicity
Miglustat has been administered at doses of up to 3000 mg/day (approximately 10 times the recommended starting dose administered to Gaucher patients) for up to six months in Human Immunodeficiency Virus (HIV)-positive patients. Adverse events observed in the HIV studies included granulocytopenia, dizziness, and paresthesia. Leukopenia and neutropenia have also been observed in a similar group of patients receiving 800 mg/day or above.
Miglustat
Patient Information
Miglustat
Organisms Affected
Humans and other mammals