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Record Information

Version

5.0

Status

Expected but not Quantified

Creation Date

2012-09-06 15:16:49 UTC

Update Date

2022-03-07 02:51:40 UTC

HMDB ID

HMDB0014563

Secondary Accession Numbers

HMDB14563

Metabolite Identification

Common Name

Miglustat

Description

Miglustat is a drug used to treat Gaucher disease. It inhibits the enzyme glucosylceramide synthase, an essential enzyme for the synthesis of most glycosphingolipids. It is only used for patients who cannot be treated with enzyme replacement therapy with imiglucerase. It is marketed under the trade name Zavesca. Miglustat is now the first and only approved therapy for patients with Niemann-Pick disease type C (NP-C). It has recently been approved for treatment of progressive neurological symptoms in adult and pediatric patients in the European Union, Brazil, and South Korea.

Structure

MOL 3D MOL SDF 3D SDF PDB 3D PDB SMILES InChI

HMDB0014563
     RDKit          3D
Miglustat
 36 36  0  0  0  0  0  0  0  0999 V2000
   -3.6246    0.7044    0.7258 C   0  0  0  0  0  0  0  0  0  0  0  0
   -2.8952   -0.6133    0.7928 C   0  0  0  0  0  0  0  0  0  0  0  0
   -2.2660   -1.0120   -0.5417 C   0  0  0  0  0  0  0  0  0  0  0  0
   -1.2791   -0.0371   -1.0272 C   0  0  0  0  0  0  0  0  0  0  0  0
   -0.1259    0.2158   -0.1853 N   0  0  0  0  0  0  0  0  0  0  0  0
    0.4229    1.4689   -0.8051 C   0  0  0  0  0  0  0  0  0  0  0  0
    1.7181    1.8910   -0.2470 C   0  0  2  0  0  0  0  0  0  0  0  0
    2.6975    2.1186   -1.2420 O   0  0  0  0  0  0  0  0  0  0  0  0
    2.3258    1.0335    0.8093 C   0  0  1  0  0  0  0  0  0  0  0  0
    1.9745    1.5194    2.0644 O   0  0  0  0  0  0  0  0  0  0  0  0
    2.0314   -0.4238    0.6502 C   0  0  2  0  0  0  0  0  0  0  0  0
    3.1841   -1.1718    0.3496 O   0  0  0  0  0  0  0  0  0  0  0  0
    0.9538   -0.7152   -0.3908 C   0  0  1  0  0  0  0  0  0  0  0  0
    0.6727   -2.1594   -0.2807 C   0  0  0  0  0  0  0  0  0  0  0  0
    0.2023   -2.4888    0.9941 O   0  0  0  0  0  0  0  0  0  0  0  0
   -3.2168    1.4328    0.0228 H   0  0  0  0  0  0  0  0  0  0  0  0
   -4.7061    0.5427    0.4956 H   0  0  0  0  0  0  0  0  0  0  0  0
   -3.6326    1.1213    1.7796 H   0  0  0  0  0  0  0  0  0  0  0  0
   -2.1435   -0.5670    1.6086 H   0  0  0  0  0  0  0  0  0  0  0  0
   -3.6395   -1.3721    1.1125 H   0  0  0  0  0  0  0  0  0  0  0  0
   -2.0173   -2.0544   -0.4668 H   0  0  0  0  0  0  0  0  0  0  0  0
   -3.1471   -0.9575   -1.2678 H   0  0  0  0  0  0  0  0  0  0  0  0
   -1.7936    0.9143   -1.2514 H   0  0  0  0  0  0  0  0  0  0  0  0
   -0.8889   -0.3467   -2.0563 H   0  0  0  0  0  0  0  0  0  0  0  0
   -0.3826    2.2219   -0.7305 H   0  0  0  0  0  0  0  0  0  0  0  0
    0.5473    1.2057   -1.8780 H   0  0  0  0  0  0  0  0  0  0  0  0
    1.5826    2.9194    0.2096 H   0  0  0  0  0  0  0  0  0  0  0  0
    2.3584    1.9805   -2.1423 H   0  0  0  0  0  0  0  0  0  0  0  0
    3.4485    1.1385    0.7329 H   0  0  0  0  0  0  0  0  0  0  0  0
    2.6975    2.1288    2.3572 H   0  0  0  0  0  0  0  0  0  0  0  0
    1.6843   -0.7971    1.6344 H   0  0  0  0  0  0  0  0  0  0  0  0
    3.8862   -0.5737   -0.0003 H   0  0  0  0  0  0  0  0  0  0  0  0
    1.4151   -0.5256   -1.3881 H   0  0  0  0  0  0  0  0  0  0  0  0
    0.1371   -2.6141   -1.1108 H   0  0  0  0  0  0  0  0  0  0  0  0
    1.6965   -2.6687   -0.3176 H   0  0  0  0  0  0  0  0  0  0  0  0
    0.1222   -3.4595    1.1468 H   0  0  0  0  0  0  0  0  0  0  0  0
  1  2  1  0
  2  3  1  0
  3  4  1  0
  4  5  1  0
  5  6  1  0
  6  7  1  0
  7  8  1  0
  7  9  1  0
  9 10  1  0
  9 11  1  0
 11 12  1  0
 11 13  1  0
 13 14  1  0
 14 15  1  0
 13  5  1  0
  1 16  1  0
  1 17  1  0
  1 18  1  0
  2 19  1  0
  2 20  1  0
  3 21  1  0
  3 22  1  0
  4 23  1  0
  4 24  1  0
  6 25  1  0
  6 26  1  0
  7 27  1  1
  8 28  1  0
  9 29  1  1
 10 30  1  0
 11 31  1  1
 12 32  1  0
 13 33  1  6
 14 34  1  0
 14 35  1  0
 15 36  1  0
M  END

419
  Mrv0541 02231214402D          

 15 15  0  0  1  0            999 V2000
    4.9509   -1.5222    0.0000 O   0  0  0  0  0  0  0  0  0  0  0  0
    3.5220   -2.3472    0.0000 O   0  0  0  0  0  0  0  0  0  0  0  0
    2.0930   -1.5222    0.0000 O   0  0  0  0  0  0  0  0  0  0  0  0
    4.9509    0.9529    0.0000 O   0  0  0  0  0  0  0  0  0  0  0  0
    3.5220    0.1279    0.0000 N   0  0  0  0  0  0  0  0  0  0  0  0
    4.2365   -0.2846    0.0000 C   0  0  1  0  0  0  0  0  0  0  0  0
    4.2365   -1.1096    0.0000 C   0  0  2  0  0  0  0  0  0  0  0  0
    3.5220   -1.5222    0.0000 C   0  0  1  0  0  0  0  0  0  0  0  0
    2.8075   -1.1096    0.0000 C   0  0  2  0  0  0  0  0  0  0  0  0
    2.8075   -0.2846    0.0000 C   0  0  0  0  0  0  0  0  0  0  0  0
    3.5220    0.9529    0.0000 C   0  0  0  0  0  0  0  0  0  0  0  0
    4.9509    0.1279    0.0000 C   0  0  0  0  0  0  0  0  0  0  0  0
    2.8075    1.3654    0.0000 C   0  0  0  0  0  0  0  0  0  0  0  0
    2.8075    2.1904    0.0000 C   0  0  0  0  0  0  0  0  0  0  0  0
    2.0930    2.6029    0.0000 C   0  0  0  0  0  0  0  0  0  0  0  0
  7  1  1  1  0  0  0
  8  2  1  6  0  0  0
  9  3  1  1  0  0  0
  4 12  1  0  0  0  0
  5  6  1  0  0  0  0
  5 10  1  0  0  0  0
  5 11  1  0  0  0  0
  6  7  1  0  0  0  0
  6 12  1  6  0  0  0
  7  8  1  0  0  0  0
  8  9  1  0  0  0  0
  9 10  1  0  0  0  0
 11 13  1  0  0  0  0
 13 14  1  0  0  0  0
 14 15  1  0  0  0  0
M  END
> <DATABASE_ID>
HMDB0014563

> <DATABASE_NAME>
hmdb

> <SMILES>
CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO

> <INCHI_IDENTIFIER>
InChI=1S/C10H21NO4/c1-2-3-4-11-5-8(13)10(15)9(14)7(11)6-12/h7-10,12-15H,2-6H2,1H3/t7-,8+,9-,10-/m1/s1

> <INCHI_KEY>
UQRORFVVSGFNRO-UTINFBMNSA-N

> <FORMULA>
C10H21NO4

> <MOLECULAR_WEIGHT>
219.278

> <EXACT_MASS>
219.147058165

> <JCHEM_ACCEPTOR_COUNT>
5

> <JCHEM_AVERAGE_POLARIZABILITY>
23.98501992158001

> <JCHEM_BIOAVAILABILITY>
1

> <JCHEM_DONOR_COUNT>
4

> <JCHEM_FORMAL_CHARGE>
0

> <JCHEM_GHOSE_FILTER>
0

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

> <ALOGPS_LOGP>
-1.14

> <JCHEM_LOGP>
-1.178796231

> <ALOGPS_LOGS>
0.18

> <JCHEM_MDDR_LIKE_RULE>
0

> <JCHEM_NUMBER_OF_RINGS>
1

> <JCHEM_PHYSIOLOGICAL_CHARGE>
1

> <JCHEM_PKA>
14.032374880430773

> <JCHEM_PKA_STRONGEST_ACIDIC>
12.902158855063018

> <JCHEM_PKA_STRONGEST_BASIC>
8.49299815256285

> <JCHEM_POLAR_SURFACE_AREA>
84.16

> <JCHEM_REFRACTIVITY>
55.7427

> <JCHEM_ROTATABLE_BOND_COUNT>
4

> <JCHEM_RULE_OF_FIVE>
1

> <ALOGPS_SOLUBILITY>
3.31e+02 g/l

> <JCHEM_TRADITIONAL_IUPAC>
miglustat

> <JCHEM_VEBER_RULE>
0

$$$$

HMDB0014563
     RDKit          3D
Miglustat
 36 36  0  0  0  0  0  0  0  0999 V2000
   -3.6246    0.7044    0.7258 C   0  0  0  0  0  0  0  0  0  0  0  0
   -2.8952   -0.6133    0.7928 C   0  0  0  0  0  0  0  0  0  0  0  0
   -2.2660   -1.0120   -0.5417 C   0  0  0  0  0  0  0  0  0  0  0  0
   -1.2791   -0.0371   -1.0272 C   0  0  0  0  0  0  0  0  0  0  0  0
   -0.1259    0.2158   -0.1853 N   0  0  0  0  0  0  0  0  0  0  0  0
    0.4229    1.4689   -0.8051 C   0  0  0  0  0  0  0  0  0  0  0  0
    1.7181    1.8910   -0.2470 C   0  0  2  0  0  0  0  0  0  0  0  0
    2.6975    2.1186   -1.2420 O   0  0  0  0  0  0  0  0  0  0  0  0
    2.3258    1.0335    0.8093 C   0  0  1  0  0  0  0  0  0  0  0  0
    1.9745    1.5194    2.0644 O   0  0  0  0  0  0  0  0  0  0  0  0
    2.0314   -0.4238    0.6502 C   0  0  2  0  0  0  0  0  0  0  0  0
    3.1841   -1.1718    0.3496 O   0  0  0  0  0  0  0  0  0  0  0  0
    0.9538   -0.7152   -0.3908 C   0  0  1  0  0  0  0  0  0  0  0  0
    0.6727   -2.1594   -0.2807 C   0  0  0  0  0  0  0  0  0  0  0  0
    0.2023   -2.4888    0.9941 O   0  0  0  0  0  0  0  0  0  0  0  0
   -3.2168    1.4328    0.0228 H   0  0  0  0  0  0  0  0  0  0  0  0
   -4.7061    0.5427    0.4956 H   0  0  0  0  0  0  0  0  0  0  0  0
   -3.6326    1.1213    1.7796 H   0  0  0  0  0  0  0  0  0  0  0  0
   -2.1435   -0.5670    1.6086 H   0  0  0  0  0  0  0  0  0  0  0  0
   -3.6395   -1.3721    1.1125 H   0  0  0  0  0  0  0  0  0  0  0  0
   -2.0173   -2.0544   -0.4668 H   0  0  0  0  0  0  0  0  0  0  0  0
   -3.1471   -0.9575   -1.2678 H   0  0  0  0  0  0  0  0  0  0  0  0
   -1.7936    0.9143   -1.2514 H   0  0  0  0  0  0  0  0  0  0  0  0
   -0.8889   -0.3467   -2.0563 H   0  0  0  0  0  0  0  0  0  0  0  0
   -0.3826    2.2219   -0.7305 H   0  0  0  0  0  0  0  0  0  0  0  0
    0.5473    1.2057   -1.8780 H   0  0  0  0  0  0  0  0  0  0  0  0
    1.5826    2.9194    0.2096 H   0  0  0  0  0  0  0  0  0  0  0  0
    2.3584    1.9805   -2.1423 H   0  0  0  0  0  0  0  0  0  0  0  0
    3.4485    1.1385    0.7329 H   0  0  0  0  0  0  0  0  0  0  0  0
    2.6975    2.1288    2.3572 H   0  0  0  0  0  0  0  0  0  0  0  0
    1.6843   -0.7971    1.6344 H   0  0  0  0  0  0  0  0  0  0  0  0
    3.8862   -0.5737   -0.0003 H   0  0  0  0  0  0  0  0  0  0  0  0
    1.4151   -0.5256   -1.3881 H   0  0  0  0  0  0  0  0  0  0  0  0
    0.1371   -2.6141   -1.1108 H   0  0  0  0  0  0  0  0  0  0  0  0
    1.6965   -2.6687   -0.3176 H   0  0  0  0  0  0  0  0  0  0  0  0
    0.1222   -3.4595    1.1468 H   0  0  0  0  0  0  0  0  0  0  0  0
  1  2  1  0
  2  3  1  0
  3  4  1  0
  4  5  1  0
  5  6  1  0
  6  7  1  0
  7  8  1  0
  7  9  1  0
  9 10  1  0
  9 11  1  0
 11 12  1  0
 11 13  1  0
 13 14  1  0
 14 15  1  0
 13  5  1  0
  1 16  1  0
  1 17  1  0
  1 18  1  0
  2 19  1  0
  2 20  1  0
  3 21  1  0
  3 22  1  0
  4 23  1  0
  4 24  1  0
  6 25  1  0
  6 26  1  0
  7 27  1  1
  8 28  1  0
  9 29  1  1
 10 30  1  0
 11 31  1  1
 12 32  1  0
 13 33  1  6
 14 34  1  0
 14 35  1  0
 15 36  1  0
M  END

HEADER    PROTEIN                                 23-FEB-12   NONE
TITLE     NULL                                                        
COMPND    MOLECULE: 419                                               
SOURCE    NULL                                                        
KEYWDS    NULL                                                        
EXPDTA    NULL                                                        
AUTHOR    Marvin                                                      
REVDAT   1   23-FEB-12         0                                  
HETATM    1  O   UNK     0       9.242  -2.841   0.000  0.00  0.00           O+0
HETATM    2  O   UNK     0       6.574  -4.381   0.000  0.00  0.00           O+0
HETATM    3  O   UNK     0       3.907  -2.841   0.000  0.00  0.00           O+0
HETATM    4  O   UNK     0       9.242   1.779   0.000  0.00  0.00           O+0
HETATM    5  N   UNK     0       6.574   0.239   0.000  0.00  0.00           N+0
HETATM    6  C   UNK     0       7.908  -0.531   0.000  0.00  0.00           C+0
HETATM    7  C   UNK     0       7.908  -2.071   0.000  0.00  0.00           C+0
HETATM    8  C   UNK     0       6.574  -2.841   0.000  0.00  0.00           C+0
HETATM    9  C   UNK     0       5.241  -2.071   0.000  0.00  0.00           C+0
HETATM   10  C   UNK     0       5.241  -0.531   0.000  0.00  0.00           C+0
HETATM   11  C   UNK     0       6.574   1.779   0.000  0.00  0.00           C+0
HETATM   12  C   UNK     0       9.242   0.239   0.000  0.00  0.00           C+0
HETATM   13  C   UNK     0       5.241   2.549   0.000  0.00  0.00           C+0
HETATM   14  C   UNK     0       5.241   4.089   0.000  0.00  0.00           C+0
HETATM   15  C   UNK     0       3.907   4.859   0.000  0.00  0.00           C+0
CONECT    1    7                                                            
CONECT    2    8                                                            
CONECT    3    9                                                            
CONECT    4   12                                                            
CONECT    5    6   10   11                                                  
CONECT    6    5    7   12                                                  
CONECT    7    1    6    8                                                  
CONECT    8    2    7    9                                                  
CONECT    9    3    8   10                                                  
CONECT   10    5    9                                                       
CONECT   11    5   13                                                       
CONECT   12    4    6                                                       
CONECT   13   11   14                                                       
CONECT   14   13   15                                                       
CONECT   15   14                                                            
MASTER        0    0    0    0    0    0    0    0   15    0   30    0
END

COMPND    HMDB0014563
HETATM    1  C1  UNL     1      -3.625   0.704   0.726  1.00  0.00           C  
HETATM    2  C2  UNL     1      -2.895  -0.613   0.793  1.00  0.00           C  
HETATM    3  C3  UNL     1      -2.266  -1.012  -0.542  1.00  0.00           C  
HETATM    4  C4  UNL     1      -1.279  -0.037  -1.027  1.00  0.00           C  
HETATM    5  N1  UNL     1      -0.126   0.216  -0.185  1.00  0.00           N  
HETATM    6  C5  UNL     1       0.423   1.469  -0.805  1.00  0.00           C  
HETATM    7  C6  UNL     1       1.718   1.891  -0.247  1.00  0.00           C  
HETATM    8  O1  UNL     1       2.698   2.119  -1.242  1.00  0.00           O  
HETATM    9  C7  UNL     1       2.326   1.033   0.809  1.00  0.00           C  
HETATM   10  O2  UNL     1       1.975   1.519   2.064  1.00  0.00           O  
HETATM   11  C8  UNL     1       2.031  -0.424   0.650  1.00  0.00           C  
HETATM   12  O3  UNL     1       3.184  -1.172   0.350  1.00  0.00           O  
HETATM   13  C9  UNL     1       0.954  -0.715  -0.391  1.00  0.00           C  
HETATM   14  C10 UNL     1       0.673  -2.159  -0.281  1.00  0.00           C  
HETATM   15  O4  UNL     1       0.202  -2.489   0.994  1.00  0.00           O  
HETATM   16  H1  UNL     1      -3.217   1.433   0.023  1.00  0.00           H  
HETATM   17  H2  UNL     1      -4.706   0.543   0.496  1.00  0.00           H  
HETATM   18  H3  UNL     1      -3.633   1.121   1.780  1.00  0.00           H  
HETATM   19  H4  UNL     1      -2.144  -0.567   1.609  1.00  0.00           H  
HETATM   20  H5  UNL     1      -3.639  -1.372   1.113  1.00  0.00           H  
HETATM   21  H6  UNL     1      -2.017  -2.054  -0.467  1.00  0.00           H  
HETATM   22  H7  UNL     1      -3.147  -0.957  -1.268  1.00  0.00           H  
HETATM   23  H8  UNL     1      -1.794   0.914  -1.251  1.00  0.00           H  
HETATM   24  H9  UNL     1      -0.889  -0.347  -2.056  1.00  0.00           H  
HETATM   25  H10 UNL     1      -0.383   2.222  -0.730  1.00  0.00           H  
HETATM   26  H11 UNL     1       0.547   1.206  -1.878  1.00  0.00           H  
HETATM   27  H12 UNL     1       1.583   2.919   0.210  1.00  0.00           H  
HETATM   28  H13 UNL     1       2.358   1.981  -2.142  1.00  0.00           H  
HETATM   29  H14 UNL     1       3.448   1.138   0.733  1.00  0.00           H  
HETATM   30  H15 UNL     1       2.698   2.129   2.357  1.00  0.00           H  
HETATM   31  H16 UNL     1       1.684  -0.797   1.634  1.00  0.00           H  
HETATM   32  H17 UNL     1       3.886  -0.574  -0.000  1.00  0.00           H  
HETATM   33  H18 UNL     1       1.415  -0.526  -1.388  1.00  0.00           H  
HETATM   34  H19 UNL     1       0.137  -2.614  -1.111  1.00  0.00           H  
HETATM   35  H20 UNL     1       1.696  -2.669  -0.318  1.00  0.00           H  
HETATM   36  H21 UNL     1       0.122  -3.460   1.147  1.00  0.00           H  
CONECT    1    2   16   17   18
CONECT    2    3   19   20
CONECT    3    4   21   22
CONECT    4    5   23   24
CONECT    5    6   13
CONECT    6    7   25   26
CONECT    7    8    9   27
CONECT    8   28
CONECT    9   10   11   29
CONECT   10   30
CONECT   11   12   13   31
CONECT   12   32
CONECT   13   14   33
CONECT   14   15   34   35
CONECT   15   36
END

HMDB0014563
     RDKit          3D
Miglustat
 36 36  0  0  0  0  0  0  0  0999 V2000
   -3.6246    0.7044    0.7258 C   0  0  0  0  0  0  0  0  0  0  0  0
   -2.8952   -0.6133    0.7928 C   0  0  0  0  0  0  0  0  0  0  0  0
   -2.2660   -1.0120   -0.5417 C   0  0  0  0  0  0  0  0  0  0  0  0
   -1.2791   -0.0371   -1.0272 C   0  0  0  0  0  0  0  0  0  0  0  0
   -0.1259    0.2158   -0.1853 N   0  0  0  0  0  0  0  0  0  0  0  0
    0.4229    1.4689   -0.8051 C   0  0  0  0  0  0  0  0  0  0  0  0
    1.7181    1.8910   -0.2470 C   0  0  2  0  0  0  0  0  0  0  0  0
    2.6975    2.1186   -1.2420 O   0  0  0  0  0  0  0  0  0  0  0  0
    2.3258    1.0335    0.8093 C   0  0  1  0  0  0  0  0  0  0  0  0
    1.9745    1.5194    2.0644 O   0  0  0  0  0  0  0  0  0  0  0  0
    2.0314   -0.4238    0.6502 C   0  0  2  0  0  0  0  0  0  0  0  0
    3.1841   -1.1718    0.3496 O   0  0  0  0  0  0  0  0  0  0  0  0
    0.9538   -0.7152   -0.3908 C   0  0  1  0  0  0  0  0  0  0  0  0
    0.6727   -2.1594   -0.2807 C   0  0  0  0  0  0  0  0  0  0  0  0
    0.2023   -2.4888    0.9941 O   0  0  0  0  0  0  0  0  0  0  0  0
   -3.2168    1.4328    0.0228 H   0  0  0  0  0  0  0  0  0  0  0  0
   -4.7061    0.5427    0.4956 H   0  0  0  0  0  0  0  0  0  0  0  0
   -3.6326    1.1213    1.7796 H   0  0  0  0  0  0  0  0  0  0  0  0
   -2.1435   -0.5670    1.6086 H   0  0  0  0  0  0  0  0  0  0  0  0
   -3.6395   -1.3721    1.1125 H   0  0  0  0  0  0  0  0  0  0  0  0
   -2.0173   -2.0544   -0.4668 H   0  0  0  0  0  0  0  0  0  0  0  0
   -3.1471   -0.9575   -1.2678 H   0  0  0  0  0  0  0  0  0  0  0  0
   -1.7936    0.9143   -1.2514 H   0  0  0  0  0  0  0  0  0  0  0  0
   -0.8889   -0.3467   -2.0563 H   0  0  0  0  0  0  0  0  0  0  0  0
   -0.3826    2.2219   -0.7305 H   0  0  0  0  0  0  0  0  0  0  0  0
    0.5473    1.2057   -1.8780 H   0  0  0  0  0  0  0  0  0  0  0  0
    1.5826    2.9194    0.2096 H   0  0  0  0  0  0  0  0  0  0  0  0
    2.3584    1.9805   -2.1423 H   0  0  0  0  0  0  0  0  0  0  0  0
    3.4485    1.1385    0.7329 H   0  0  0  0  0  0  0  0  0  0  0  0
    2.6975    2.1288    2.3572 H   0  0  0  0  0  0  0  0  0  0  0  0
    1.6843   -0.7971    1.6344 H   0  0  0  0  0  0  0  0  0  0  0  0
    3.8862   -0.5737   -0.0003 H   0  0  0  0  0  0  0  0  0  0  0  0
    1.4151   -0.5256   -1.3881 H   0  0  0  0  0  0  0  0  0  0  0  0
    0.1371   -2.6141   -1.1108 H   0  0  0  0  0  0  0  0  0  0  0  0
    1.6965   -2.6687   -0.3176 H   0  0  0  0  0  0  0  0  0  0  0  0
    0.1222   -3.4595    1.1468 H   0  0  0  0  0  0  0  0  0  0  0  0
  1  2  1  0
  2  3  1  0
  3  4  1  0
  4  5  1  0
  5  6  1  0
  6  7  1  0
  7  8  1  0
  7  9  1  0
  9 10  1  0
  9 11  1  0
 11 12  1  0
 11 13  1  0
 13 14  1  0
 14 15  1  0
 13  5  1  0
  1 16  1  0
  1 17  1  0
  1 18  1  0
  2 19  1  0
  2 20  1  0
  3 21  1  0
  3 22  1  0
  4 23  1  0
  4 24  1  0
  6 25  1  0
  6 26  1  0
  7 27  1  1
  8 28  1  0
  9 29  1  1
 10 30  1  0
 11 31  1  1
 12 32  1  0
 13 33  1  6
 14 34  1  0
 14 35  1  0
 15 36  1  0
M  END

View in JSmol View NMR Assignments View Stereo Labels

Synonyms

Value	Source
BuDNJ	ChEBI
Butyldeoxynojirimycin	ChEBI
Miglustatum	ChEBI
N-(N-Butyl)deoxynojirimycin	ChEBI
N-Butyl deoxynojirimycin	ChEBI
N-Butyl-1-deoxynojirimycin	ChEBI
N-Butylmoranoline	ChEBI
NB-DNJ	ChEBI
SC-48334	ChEBI
Zavesca	ChEBI
Brazaves	Kegg
N-(N-Butyl)deoxy-nojirimycin	HMDB
N-Butyldeoxynojirimycin	HMDB
OGT 918	HMDB

Chemical Formula

C₁₀H₂₁NO₄

Average Molecular Weight

219.278

Monoisotopic Molecular Weight

219.147058165

IUPAC Name

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

Traditional Name

miglustat

CAS Registry Number

72599-27-0

SMILES

CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO

InChI Identifier

InChI=1S/C10H21NO4/c1-2-3-4-11-5-8(13)10(15)9(14)7(11)6-12/h7-10,12-15H,2-6H2,1H3/t7-,8+,9-,10-/m1/s1

InChI Key

UQRORFVVSGFNRO-UTINFBMNSA-N

Chemical Taxonomy

Description

Belongs to the class of organic compounds known as piperidines. Piperidines are compounds containing a piperidine ring, which is a saturated aliphatic six-member ring with one nitrogen atom and five carbon atoms.

Kingdom

Organic compounds

Super Class

Organoheterocyclic compounds

Class

Piperidines

Sub Class

Not Available

Direct Parent

Piperidines

Alternative Parents

Substituents

Piperidine
1,2-aminoalcohol
Secondary alcohol
Tertiary aliphatic amine
Tertiary amine
Polyol
Azacycle
Organic nitrogen compound
Hydrocarbon derivative
Primary alcohol
Organopnictogen compound
Organic oxygen compound
Organooxygen compound
Organonitrogen compound
Amine
Alcohol
Aliphatic heteromonocyclic compound

Molecular Framework

Aliphatic heteromonocyclic compounds

External Descriptors

piperidines (CHEBI:50381 )
tertiary amino compound (CHEBI:50381 )

Ontology

Physiological effect

Not Available

Disposition

Biological location

Non-excretory biofluid

Biofluid or Excreta

Blood (PMID: 21059682)

Excreta

Biofluid or Excreta

Urine (PMID: 21059682)

Cellular substructure

Membrane (HMDB: HMDB0014563)

Source

Exogenous

Exogenous (HMDB: HMDB0014563)

Process

Not Available

Role

Not Available

Physical Properties

State

Solid

Experimental Molecular Properties

Property	Value	Reference
Melting Point	169 - 172 °C	Not Available
Boiling Point	Not Available	Not Available
Water Solubility	331 g/L	Not Available
LogP	-0.6	Not Available

Experimental Chromatographic Properties

Not Available

Predicted Molecular Properties

Property	Value	Source
Water Solubility	331 g/L	ALOGPS
logP	-1.1	ALOGPS
logP	-1.2	ChemAxon
logS	0.18	ALOGPS
pKa (Strongest Acidic)	12.9	ChemAxon
pKa (Strongest Basic)	8.49	ChemAxon
Physiological Charge	1	ChemAxon
Hydrogen Acceptor Count	5	ChemAxon
Hydrogen Donor Count	4	ChemAxon
Polar Surface Area	84.16 Å²	ChemAxon
Rotatable Bond Count	4	ChemAxon
Refractivity	55.74 m³·mol⁻¹	ChemAxon
Polarizability	23.99 Å³	ChemAxon
Number of Rings	1	ChemAxon
Bioavailability	Yes	ChemAxon
Rule of Five	Yes	ChemAxon
Ghose Filter	No	ChemAxon
Veber's Rule	No	ChemAxon
MDDR-like Rule	No	ChemAxon

Predicted Chromatographic Properties

Predicted Collision Cross Sections

Predictor	Adduct Type	CCS Value (Å²)	Reference
DarkChem	[M+H]+	152.861	31661259
DarkChem	[M-H]-	147.48	31661259
DeepCCS	[M+H]+	155.738	30932474
DeepCCS	[M-H]-	153.38	30932474
DeepCCS	[M-2H]-	186.468	30932474
DeepCCS	[M+Na]+	161.841	30932474
AllCCS	[M+H]+	152.5	32859911
AllCCS	[M+H-H2O]+	148.7	32859911
AllCCS	[M+NH4]+	155.9	32859911
AllCCS	[M+Na]+	156.9	32859911
AllCCS	[M-H]-	151.4	32859911
AllCCS	[M+Na-2H]-	152.1	32859911
AllCCS	[M+HCOO]-	153.0	32859911

Predicted Retention Times

Underivatized

Chromatographic Method	Retention Time	Reference
Measured using a Waters Acquity ultraperformance liquid chromatography (UPLC) ethylene-bridged hybrid (BEH) C18 column (100 mm × 2.1 mm; 1.7 μmparticle diameter). Predicted by Afia on May 17, 2022. Predicted by Afia on May 17, 2022.	1.28 minutes	32390414
Predicted by Siyang on May 30, 2022	10.1415 minutes	33406817
Predicted by Siyang using ReTip algorithm on June 8, 2022	6.1 minutes	32390414
AjsUoB = Accucore 150 Amide HILIC with 10mM Ammonium Formate, 0.1% Formic Acid	349.0 seconds	40023050
Fem_Long = Waters ACQUITY UPLC HSS T3 C18 with Water:MeOH and 0.1% Formic Acid	503.5 seconds	40023050
Fem_Lipids = Ascentis Express C18 with (60:40 water:ACN):(90:10 IPA:ACN) and 10mM NH4COOH + 0.1% Formic Acid	249.4 seconds	40023050
Life_Old = Waters ACQUITY UPLC BEH C18 with Water:(20:80 acetone:ACN) and 0.1% Formic Acid	55.6 seconds	40023050
Life_New = RP Waters ACQUITY UPLC HSS T3 C18 with Water:(30:70 MeOH:ACN) and 0.1% Formic Acid	171.5 seconds	40023050
RIKEN = Waters ACQUITY UPLC BEH C18 with Water:ACN and 0.1% Formic Acid	61.3 seconds	40023050
Eawag_XBridgeC18 = XBridge C18 3.5u 2.1x50 mm with Water:MeOH and 0.1% Formic Acid	325.6 seconds	40023050
BfG_NTS_RP1 =Agilent Zorbax Eclipse Plus C18 (2.1 mm x 150 mm, 3.5 um) with Water:ACN and 0.1% Formic Acid	264.4 seconds	40023050
HILIC_BDD_2 = Merck SeQuant ZIC-HILIC with ACN(0.1% formic acid):water(16 mM ammonium formate)	798.4 seconds	40023050
UniToyama_Atlantis = RP Waters Atlantis T3 (2.1 x 150 mm, 5 um) with ACN:Water and 0.1% Formic Acid	612.4 seconds	40023050
BDD_C18 = Hypersil Gold 1.9µm C18 with Water:ACN and 0.1% Formic Acid	44.8 seconds	40023050
UFZ_Phenomenex = Kinetex Core-Shell C18 2.6 um, 3.0 x 100 mm, Phenomenex with Water:MeOH and 0.1% Formic Acid	712.1 seconds	40023050
SNU_RIKEN_POS = Waters ACQUITY UPLC BEH C18 with Water:ACN and 0.1% Formic Acid	167.6 seconds	40023050
RPMMFDA = Waters ACQUITY UPLC BEH C18 with Water:ACN and 0.1% Formic Acid	223.8 seconds	40023050
MTBLS87 = Merck SeQuant ZIC-pHILIC column with ACN:Water and :ammonium carbonate	766.3 seconds	40023050
KI_GIAR_zic_HILIC_pH2_7 = Merck SeQuant ZIC-HILIC with ACN:Water and 0.1% FA	519.6 seconds	40023050
Meister zic-pHILIC pH9.3 = Merck SeQuant ZIC-pHILIC column with ACN:Water 5mM NH4Ac pH9.3 and 5mM ammonium acetate in water	325.5 seconds	40023050

Predicted Kovats Retention Indices

Underivatized

Metabolite	SMILES	Kovats RI Value	Column Type	Reference
Miglustat	CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO	3314.8	Standard polar	33892256
Miglustat	CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO	1904.5	Standard non polar	33892256
Miglustat	CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO	2070.0	Semi standard non polar	33892256

Derivatized

Derivative Name / Structure	SMILES	Kovats RI Value	Column Type	Reference
Miglustat,1TMS,isomer #1	CCCCN1C[C@H](O[Si](C)(C)C)[C@@H](O)[C@H](O)[C@H]1CO	1729.1	Semi standard non polar	33892256
Miglustat,1TMS,isomer #2	CCCCN1C[C@H](O)[C@@H](O[Si](C)(C)C)[C@H](O)[C@H]1CO	1713.2	Semi standard non polar	33892256
Miglustat,1TMS,isomer #3	CCCCN1C[C@H](O)[C@@H](O)[C@H](O[Si](C)(C)C)[C@H]1CO	1725.3	Semi standard non polar	33892256
Miglustat,1TMS,isomer #4	CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO[Si](C)(C)C	1760.7	Semi standard non polar	33892256
Miglustat,2TMS,isomer #1	CCCCN1C[C@H](O[Si](C)(C)C)[C@@H](O[Si](C)(C)C)[C@H](O)[C@H]1CO	1763.1	Semi standard non polar	33892256
Miglustat,2TMS,isomer #2	CCCCN1C[C@H](O[Si](C)(C)C)[C@@H](O)[C@H](O[Si](C)(C)C)[C@H]1CO	1781.6	Semi standard non polar	33892256
Miglustat,2TMS,isomer #3	CCCCN1C[C@H](O[Si](C)(C)C)[C@@H](O)[C@H](O)[C@H]1CO[Si](C)(C)C	1803.8	Semi standard non polar	33892256
Miglustat,2TMS,isomer #4	CCCCN1C[C@H](O)[C@@H](O[Si](C)(C)C)[C@H](O[Si](C)(C)C)[C@H]1CO	1792.3	Semi standard non polar	33892256
Miglustat,2TMS,isomer #5	CCCCN1C[C@H](O)[C@@H](O[Si](C)(C)C)[C@H](O)[C@H]1CO[Si](C)(C)C	1817.7	Semi standard non polar	33892256
Miglustat,2TMS,isomer #6	CCCCN1C[C@H](O)[C@@H](O)[C@H](O[Si](C)(C)C)[C@H]1CO[Si](C)(C)C	1813.0	Semi standard non polar	33892256
Miglustat,3TMS,isomer #1	CCCCN1C[C@H](O[Si](C)(C)C)[C@@H](O[Si](C)(C)C)[C@H](O[Si](C)(C)C)[C@H]1CO	1845.4	Semi standard non polar	33892256
Miglustat,3TMS,isomer #2	CCCCN1C[C@H](O[Si](C)(C)C)[C@@H](O[Si](C)(C)C)[C@H](O)[C@H]1CO[Si](C)(C)C	1874.8	Semi standard non polar	33892256
Miglustat,3TMS,isomer #3	CCCCN1C[C@H](O[Si](C)(C)C)[C@@H](O)[C@H](O[Si](C)(C)C)[C@H]1CO[Si](C)(C)C	1901.7	Semi standard non polar	33892256
Miglustat,3TMS,isomer #4	CCCCN1C[C@H](O)[C@@H](O[Si](C)(C)C)[C@H](O[Si](C)(C)C)[C@H]1CO[Si](C)(C)C	1903.2	Semi standard non polar	33892256
Miglustat,4TMS,isomer #1	CCCCN1C[C@H](O[Si](C)(C)C)[C@@H](O[Si](C)(C)C)[C@H](O[Si](C)(C)C)[C@H]1CO[Si](C)(C)C	1948.0	Semi standard non polar	33892256
Miglustat,1TBDMS,isomer #1	CCCCN1C[C@H](O[Si](C)(C)C(C)(C)C)[C@@H](O)[C@H](O)[C@H]1CO	1985.1	Semi standard non polar	33892256
Miglustat,1TBDMS,isomer #2	CCCCN1C[C@H](O)[C@@H](O[Si](C)(C)C(C)(C)C)[C@H](O)[C@H]1CO	1961.9	Semi standard non polar	33892256
Miglustat,1TBDMS,isomer #3	CCCCN1C[C@H](O)[C@@H](O)[C@H](O[Si](C)(C)C(C)(C)C)[C@H]1CO	1976.4	Semi standard non polar	33892256
Miglustat,1TBDMS,isomer #4	CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO[Si](C)(C)C(C)(C)C	2011.8	Semi standard non polar	33892256
Miglustat,2TBDMS,isomer #1	CCCCN1C[C@H](O[Si](C)(C)C(C)(C)C)[C@@H](O[Si](C)(C)C(C)(C)C)[C@H](O)[C@H]1CO	2268.2	Semi standard non polar	33892256
Miglustat,2TBDMS,isomer #2	CCCCN1C[C@H](O[Si](C)(C)C(C)(C)C)[C@@H](O)[C@H](O[Si](C)(C)C(C)(C)C)[C@H]1CO	2277.7	Semi standard non polar	33892256
Miglustat,2TBDMS,isomer #3	CCCCN1C[C@H](O[Si](C)(C)C(C)(C)C)[C@@H](O)[C@H](O)[C@H]1CO[Si](C)(C)C(C)(C)C	2305.8	Semi standard non polar	33892256
Miglustat,2TBDMS,isomer #4	CCCCN1C[C@H](O)[C@@H](O[Si](C)(C)C(C)(C)C)[C@H](O[Si](C)(C)C(C)(C)C)[C@H]1CO	2277.8	Semi standard non polar	33892256
Miglustat,2TBDMS,isomer #5	CCCCN1C[C@H](O)[C@@H](O[Si](C)(C)C(C)(C)C)[C@H](O)[C@H]1CO[Si](C)(C)C(C)(C)C	2307.0	Semi standard non polar	33892256
Miglustat,2TBDMS,isomer #6	CCCCN1C[C@H](O)[C@@H](O)[C@H](O[Si](C)(C)C(C)(C)C)[C@H]1CO[Si](C)(C)C(C)(C)C	2295.3	Semi standard non polar	33892256
Miglustat,3TBDMS,isomer #1	CCCCN1C[C@H](O[Si](C)(C)C(C)(C)C)[C@@H](O[Si](C)(C)C(C)(C)C)[C@H](O[Si](C)(C)C(C)(C)C)[C@H]1CO	2553.4	Semi standard non polar	33892256
Miglustat,3TBDMS,isomer #2	CCCCN1C[C@H](O[Si](C)(C)C(C)(C)C)[C@@H](O[Si](C)(C)C(C)(C)C)[C@H](O)[C@H]1CO[Si](C)(C)C(C)(C)C	2582.1	Semi standard non polar	33892256
Miglustat,3TBDMS,isomer #3	CCCCN1C[C@H](O[Si](C)(C)C(C)(C)C)[C@@H](O)[C@H](O[Si](C)(C)C(C)(C)C)[C@H]1CO[Si](C)(C)C(C)(C)C	2601.5	Semi standard non polar	33892256
Miglustat,3TBDMS,isomer #4	CCCCN1C[C@H](O)[C@@H](O[Si](C)(C)C(C)(C)C)[C@H](O[Si](C)(C)C(C)(C)C)[C@H]1CO[Si](C)(C)C(C)(C)C	2595.6	Semi standard non polar	33892256
Miglustat,4TBDMS,isomer #1	CCCCN1C[C@H](O[Si](C)(C)C(C)(C)C)[C@@H](O[Si](C)(C)C(C)(C)C)[C@H](O[Si](C)(C)C(C)(C)C)[C@H]1CO[Si](C)(C)C(C)(C)C	2835.9	Semi standard non polar	33892256

Spectra

Biological Properties

Cellular Locations

Membrane

Biospecimen Locations

Blood
Urine

Tissue Locations

Not Available

Pathways

Not Available

Name	SMPDB/PathBank	KEGG

Normal Concentrations

Biospecimen	Status	Value	Age	Sex	Condition	Reference	Details
Blood	Expected but not Quantified	Not Quantified	Not Available	Not Available	Taking drug identified by DrugBank entry DB00419	21059682	details
Urine	Expected but not Quantified	Not Quantified	Not Available	Not Available	Taking drug identified by DrugBank entry DB00419	21059682	details

Abnormal Concentrations

Not Available

Associated Disorders and Diseases

Disease References

None

Associated OMIM IDs

None

External Links

DrugBank ID

DB00419

Phenol Explorer Compound ID

Not Available

FooDB ID

Not Available

KNApSAcK ID

Not Available

Chemspider ID

46764

KEGG Compound ID

Not Available

BioCyc ID

Not Available

BiGG ID

Not Available

Wikipedia Link

Miglustat

METLIN ID

Not Available

PubChem Compound

51634

PDB ID

Not Available

ChEBI ID

50381

Food Biomarker Ontology

Not Available

VMH ID

Not Available

MarkerDB ID

Not Available

Good Scents ID

Not Available

References

Synthesis Reference

Not Available

Material Safety Data Sheet (MSDS)

Not Available

General References

Moyses C: Substrate reduction therapy: clinical evaluation in type 1 Gaucher disease. Philos Trans R Soc Lond B Biol Sci. 2003 May 29;358(1433):955-60. [PubMed:12803929 ]
Weinreb NJ, Barranger JA, Charrow J, Grabowski GA, Mankin HJ, Mistry P: Guidance on the use of miglustat for treating patients with type 1 Gaucher disease. Am J Hematol. 2005 Nov;80(3):223-9. [PubMed:16247743 ]
Wraith JE, Imrie J: New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat. Ther Clin Risk Manag. 2009;5:877-87. Epub 2009 Nov 18. [PubMed:19956552 ]
McCormack PL, Goa KL: Miglustat. Drugs. 2003;63(22):2427-34; discussion 2435-6. [PubMed:14609352 ]
Patterson MC, Vecchio D, Prady H, Abel L, Wraith JE: Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study. Lancet Neurol. 2007 Sep;6(9):765-72. [PubMed:17689147 ]
van Giersbergen PL, Dingemanse J: Influence of food intake on the pharmacokinetics of miglustat, an inhibitor of glucosylceramide synthase. J Clin Pharmacol. 2007 Oct;47(10):1277-82. Epub 2007 Aug 24. [PubMed:17720777 ]

Enzymes

Enzyme Details

1. Ceramide glucosyltransferase

General function:: Cell wall/membrane/envelope biogenesis
Specific function:: Catalyzes the first glycosylation step in glycosphingolipid biosynthesis, the transfer of glucose to ceramide. May also serve as a "flippase".
Gene Name:: UGCG
Uniprot ID:: Q16739
Molecular weight:: 44853.255

References

Moyses C: Substrate reduction therapy: clinical evaluation in type 1 Gaucher disease. Philos Trans R Soc Lond B Biol Sci. 2003 May 29;358(1433):955-60. [PubMed:12803929 ]
Weinreb NJ, Barranger JA, Charrow J, Grabowski GA, Mankin HJ, Mistry P: Guidance on the use of miglustat for treating patients with type 1 Gaucher disease. Am J Hematol. 2005 Nov;80(3):223-9. [PubMed:16247743 ]
Chien YH, Lee NC, Tsai LK, Huang AC, Peng SF, Chen SJ, Hwu WL: Treatment of Niemann-Pick disease type C in two children with miglustat: initial responses and maintenance of effects over 1 year. J Inherit Metab Dis. 2007 Oct;30(5):826. Epub 2007 Jun 21. [PubMed:17603755 ]
Treiber A, Morand O, Clozel M: The pharmacokinetics and tissue distribution of the glucosylceramide synthase inhibitor miglustat in the rat. Xenobiotica. 2007 Mar;37(3):298-314. [PubMed:17624027 ]
Ficicioglu C: Review of miglustat for clinical management in Gaucher disease type 1. Ther Clin Risk Manag. 2008 Apr;4(2):425-31. [PubMed:18728838 ]
Wraith JE, Imrie J: New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat. Ther Clin Risk Manag. 2009;5:877-87. Epub 2009 Nov 18. [PubMed:19956552 ]
Pastores GM: Miglustat: substrate reduction therapy for lysosomal storage disorders associated with primary central nervous system involvement. Recent Pat CNS Drug Discov. 2006 Jan;1(1):77-82. [PubMed:18221193 ]
McCormack PL, Goa KL: Miglustat. Drugs. 2003;63(22):2427-34; discussion 2435-6. [PubMed:14609352 ]
Giraldo P, Latre P, Alfonso P, Acedo A, Alonso D, Barez A, Corrales A, Franco R, Roldan V, Serrano S, Pocovi M: Short-term effect of miglustat in every day clinical use in treatment-naive or previously treated patients with type 1 Gaucher's disease. Haematologica. 2006 May;91(5):703-6. Epub 2006 Apr 19. [PubMed:16627252 ]
Chen X, Ji ZL, Chen YZ: TTD: Therapeutic Target Database. Nucleic Acids Res. 2002 Jan 1;30(1):412-5. [PubMed:11752352 ]

Showing metabocard for Miglustat (HMDB0014563)

MOL for HMDB0014563 (Miglustat)

3D MOL for HMDB0014563 (Miglustat)

3D SDF for HMDB0014563 (Miglustat)

3D-SDF for HMDB0014563 (Miglustat)

PDB for HMDB0014563 (Miglustat)

3D PDB for HMDB0014563 (Miglustat)

SMILES for HMDB0014563 (Miglustat)

INCHI for HMDB0014563 (Miglustat)

Structure for HMDB0014563 (Miglustat)

3D Structure for HMDB0014563 (Miglustat)

Predicted Collision Cross Sections

Predicted Retention Times

Underivatized

Predicted Kovats Retention Indices

Underivatized

Derivatized

Enzymes

References