e-Drug3D

889

MIGLUSTAT

INN
MIGLUSTAT

cas 72599-27-0

CLASS

METABOLISM
TYPE 1 GAUCHER DISEASE
GLUCOSYLCERAMIDE SYNTHASE INHIBITOR
SYNTHESIS OF MOST GLYCOSPHINGOLIPIDS
TREATMENT OF NIEMANN-PICK TYPE C DISEASE (DRUG FOR RARE DISEASE IN EUROPE)

ROUTE (list)

ORAL

ATC code

A16AX06

PK parameters

F 97 PERCENT
Tmax 2.25 HOUR
VD 94 LITER
PPB 0 PERCENT
t1/2 6.5 HOUR

Solubility

HIGHLY SOLUBLE IN WATER (> 1000 MILLIGRAM / MILLILITER)

Target

TARGET GLUCOSYLCERAMIDE SYNTHASE

PDB
2V3D (ACID-BETA-GLUCOSIDASE WITH N-BUTYL-DEOXYNOJIRIMYCIN)

Ligand code = NBV (link to the list of PDB complexes)

Download experimental 3D coordinates of NBV with added hydrogens

DrugCentral

Ceramide glucosyltransferase UNIPROT Q16739 UGCG

Download SDF file

(2R,3R,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol

Formula:
C10H21NO4

Calculated chemical properties:

MW 219.28
NB_atoms 15
XLogP -1.45
PSA 87.769
NB_HD 4
NB_HA 5
fsp3 1.00
Lipinski(0=no;1=yes) 1

FDA
NDA 021348 (label) INN (MIGLUSTAT) ZAVESCA (ACTELION) APPROVAL= AP YEAR= 2003 NEWMOLECULARENTITY
NDA 208342 (pdf not found) INN (MIGLUSTAT) MIGLUSTAT (ANI PHARMS) APPROVAL= AP YEAR= 2018
NDA 209821 (pdf not found) INN (MIGLUSTAT) YARGESA (EDENBRIDGE PHARMS) APPROVAL= AP YEAR= 2020
NDA 209325 (pdf not found) INN (MIGLUSTAT) MIGLUSTAT (CHARTWELL RX) APPROVAL= AP YEAR= 2022 DISCONTINUED
NDA 215211 (label) INN (MIGLUSTAT) OPFOLDA (AMICUS THERAP US) APPROVAL= AP YEAR= 2023
NDA 219111 (pdf not found) INN (MIGLUSTAT) MIGLUSTAT (NAVINTA LLC) APPROVAL= AP YEAR= 2025