KEGG   DRUG: Migalastat hydrochloride
Entry
D05031                      Drug                                   
Name
Migalastat hydrochloride (JAN/USAN);
Galafold (TN)
Product
GALAFOLD (Amicus Therapeutics US)
Formula
C6H13NO4. HCl
Exact mass
199.0611
Mol weight
199.63
Structure
Simcomp
Remark
Therapeutic category: 3999
ATC code: A16AX14
Chemical structure group: DG02691
Product (DG02691): D05031<JP/US>
Efficacy
Lysosomal storage disease treatment, Pharmacological chaperone
  Disease
Fabry disease (GLA variant) [DS:H00125]
Comment
Pharmacological chaperone
Target
GLA* [HSA_VAR:2717v2] [HSA:2717] [KO:K01189]
  Pathway
hsa00600  Sphingolipid metabolism
hsa04142  Lysosome
  Network
nt06014  Sphingolipid degradation
Interaction
Brite
Anatomical Therapeutic Chemical (ATC) classification [BR:br08303]
 A ALIMENTARY TRACT AND METABOLISM
  A16 OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
   A16A OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
    A16AX Various alimentary tract and metabolism products
     A16AX14 Migalastat
      D05031  Migalastat hydrochloride (JAN/USAN) <JP/US>
USP drug classification [BR:br08302]
 Genetic, Enzyme, or Protein Disorder: Replacement, Modifiers, Treatment
  Migalastat
   D05031  Migalastat hydrochloride (JAN/USAN)
Therapeutic category of drugs in Japan [BR:br08301]
 3  Agents affecting metabolism
  39  Other agents affecting metabolism
   399  Miscellaneous
    3999  Others
     D05031  Migalastat hydrochloride (JAN/USAN)
Target-based classification of drugs [BR:br08310]
 Enzymes
  Hydrolases (EC3)
   Glycosidases
    GLA* [HSA_VAR:2717v2]
     D05031  Migalastat hydrochloride (JAN/USAN) <JP/US>
New drug approvals in the USA [br08319.html]
 New molecular entities and new therapeutic biological products
  D05031
New drug approvals in Europe [br08329.html]
 European public assessment reports (EPAR) authorised medicine
  D05031
New drug approvals in Japan [br08318.html]
 Drugs with new active ingredients
  D05031
New drug approvals in the USA, Europe and Japan [br08328.html]
 Approval dates by FDA, EMA and PMDA
  D05031
Pharmacogenomic biomarkers [br08341.html]
 Germline mutations in genetic disorder treatments
  D05031
Other DBs
CAS: 75172-81-5
PubChem: 47206758
LigandBox: D05031
LinkDB
KCF data

ATOM        12
            1   C1y C    10.9200  -14.9800
            2   C1y C    10.9200  -16.3800
            3   C1y C    12.1324  -17.0800
            4   C1y C    13.3449  -16.3800
            5   C1x C    13.3449  -14.9800
            6   N1x N    12.1324  -14.2800
            7   C1b C     9.7076  -14.2800
            8   O1a O     9.7076  -17.0800
            9   O1a O    12.1324  -18.4798
            10  O1a O    14.5760  -17.0910
            11  O1a O     8.5121  -14.9704
            12  X   Cl   17.3600  -15.1900
BOND        11
            1     1   2 1
            2     2   3 1
            3     3   4 1
            4     4   5 1
            5     5   6 1
            6     1   6 1
            7     1   7 1 #Up
            8     2   8 1 #Up
            9     3   9 1 #Up
            10    4  10 1 #Down
            11    7  11 1

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