ZAVESCA

País: Australia

Idioma: inglés

Fuente: Department of Health (Therapeutic Goods Administration)

Ingredientes activos:

Miglustat

Disponible desde:

Actelion Pharmaceuticals Australia Pty Limited

clase:

Medicine Registered

Ficha técnica

Zavesca
® miglustat  
PRODUCT INFORMATION 

ZAVESCA
®
 (MIGLUSTAT)  
100MG CAPSULES 

NAME OF THE MEDICINE 
Miglustat  

DESCRIPTION 
Miglustat is an orally active, non-peptide, N-alkylated imino sugar,
which is a synthetic 
analogue of D-glucose. It is a white to off-white crystalline solid
and is highly water 
soluble (›1000mg/mL as a free base). The chemical name of miglustat
is 1, 5-
(butylimino)-1,5-dideoxy-D-glucitol. 

Molecular structure 

N
OH
OH
HO
Me
HO

Molecular formula: C
10
H
21
NO
4
Molecular mass: 219.28  
CAS Number 72599-27-0 

Each capsule contains: 
Active: 100 mg miglustat  
Inactives: sodium starch glycollate, povidone, magnesium stearate  
Capsule shell: gelatin, titanium dioxide (E171), black printing ink 

PHARMACOLOGY 

PHARMACODYNAMICS 
TYPE 1 GAUCHER DISEASE 
Type 1 Gaucher disease is an inherited metabolic disorder caused by a
functional 
deficiency of β-glucocerebrosidase, the enzyme that mediates
the degradation of the 
glycosphingolipid; glucocerebroside.  The failure to degrade
glucocerebroside results in 
the lyosomal storage of this material within tissue macrophages,
leading to widespread 
pathology.  Macrophages containing stored glucocerebroside are
typically found in the 
liver, spleen, and bone marrow and occasionally in lung, kidney and
intestine.  
Secondary haematogical consequences include severe anaemia and 
thrombocytopenia in addition to the characteristic
progressive hepatosplenomegaly. 
Skeletal complications include osteonecrosis and osteopenia with
secondary 
pathological fractures and associated pain; all of which can cause
significant morbidity. 

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Zavesca
® miglustat  
Miglustat acts as a competitive and reversible inhibitor of
glucosylceramide synthase, 
the enzyme responsible for the first and committed step in the
synthesis of most 
glycosphingolipids.  The goal of treatment with miglustat is to
reduce the rate