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Details

Stereochemistry ABSOLUTE
Molecular Formula C6H13NO4.ClH
Molecular Weight 199.6328
Optical Activity UNSPECIFIED
Defined Stereocenters 4 / 4
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of MIGALASTAT HYDROCHLORIDE

SMILES

C1[C@@]([H])([C@]([H])([C@]([H])([C@@]([H])(CO)N1)O)O)O.Cl

InChI

InChIKey=ZJIHMALTJRDNQI-OLALXQGDSA-N
InChI=1S/C6H13NO4.ClH/c8-2-3-5(10)6(11)4(9)1-7-3;/h3-11H,1-2H2;1H/t3-,4+,5+,6-;/m1./s1

HIDE SMILES / InChI

Molecular Formula ClH
Molecular Weight 36.4609
Charge 0
Count
Stereochemistry ACHIRAL
Additional Stereochemistry No
Defined Stereocenters 0 / 0
E/Z Centers 0
Optical Activity NONE

Molecular Formula C6H13NO4
Molecular Weight 163.172
Charge 0
Count
Stereochemistry ABSOLUTE
Additional Stereochemistry No
Defined Stereocenters 4 / 4
E/Z Centers 0
Optical Activity UNSPECIFIED

Migalastat (Galafold)-a small molecule drug developed by Amicus Therapeutics that restores the activity of specific mutant forms of α-galactosidase-has been approved for the treatment of Fabry disease in the EU in patients with amenable mutations. Migalastat attaches to certain unstable forms of alpha-galactosidase A, stabilising the enzyme. This allows the enzyme to be transported into areas of the cell where it can break down GL-3. Under the trade name Galafold (formerly known as Amigal), Migalastat is used to treat patients aged 16 years or over with Fabry disease. Because the number of patients with Fabry disease is low, the disease is considered ‘rare’, and the US Food and Drug Administration (FDA) assigned Galafold orphan drug status in 2004, and the European Committee for Medicinal Products for Human Use (CHMP) followed in 2006.

Approval Year

Targets

Targets

Primary TargetPharmacologyConditionPotency
0.04 µM [IC50]
Conditions

Conditions

ConditionModalityTargetsHighest PhaseProduct
Primary
Galafold

Approved Use

Galafold is indicated for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who have an amenable mutation

Launch Date

1.46456642E12
Cmax

Cmax

ValueDoseCo-administeredAnalytePopulation
2029 ng/mL
150 mg 1 times / 2 days steady-state, oral
dose: 150 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
MIGALASTAT plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: UNKNOWN
food status: FASTED
1323 ng/mL
150 mg single, oral
dose: 150 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
MIGALASTAT plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: UNKNOWN
food status: HIGH-FAT
1561 ng/mL
150 mg single, oral
dose: 150 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
MIGALASTAT plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: UNKNOWN
food status: FASTED
AUC

AUC

ValueDoseCo-administeredAnalytePopulation
10638 ng × h/mL
150 mg 1 times / 2 days steady-state, oral
dose: 150 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
MIGALASTAT plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: UNKNOWN
food status: FASTED
6132 ng × h/mL
150 mg single, oral
dose: 150 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
MIGALASTAT plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: UNKNOWN
food status: HIGH-FAT
9805 ng × h/mL
150 mg single, oral
dose: 150 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
MIGALASTAT plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: UNKNOWN
food status: FASTED
10637.9 ng*h/mL
150 mg 1 times / 2 days steady, oral
dose: 150 mg
route of administration: oral
experiment type: steady
co-administered:
MIGALASTAT plasma
Homo sapiens
population: unhealthy
age:
sex: F
food status:
13217.2 ng*h/mL
250 mg single, oral
dose: 250 mg
route of administration: oral
experiment type: single
co-administered:
MIGALASTAT plasma
Homo sapiens
population: unhealthy
age:
sex: F
food status:
14850.6 ng*h/mL
250 mg 1 times / 2 days steady, oral
dose: 250 mg
route of administration: oral
experiment type: steady
co-administered:
MIGALASTAT plasma
Homo sapiens
population: unhealthy
age:
sex: F
food status:
2300.3 ng*h/mL
50 mg 1 times / 2 days steady, oral
dose: 50 mg
route of administration: oral
experiment type: steady
co-administered:
MIGALASTAT plasma
Homo sapiens
population: unhealthy
age:
sex: F
food status:
2628.9 ng*h/mL
50 mg single, oral
dose: 50 mg
route of administration: oral
experiment type: single
co-administered:
MIGALASTAT plasma
Homo sapiens
population: unhealthy
age:
sex: F
food status:
3191.6 ng*h/mL
50 mg 1 times / 2 days steady, oral
dose: 50 mg
route of administration: oral
experiment type: steady
co-administered:
MIGALASTAT plasma
Homo sapiens
population: unhealthy
age:
sex: F
food status:
8581.9 ng*h/mL
150 mg 1 times / 2 days steady, oral
dose: 150 mg
route of administration: oral
experiment type: steady
co-administered:
MIGALASTAT plasma
Homo sapiens
population: unhealthy
age:
sex: F
food status:
8941.6 ng*h/mL
150 mg single, oral
dose: 150 mg
route of administration: oral
experiment type: single
co-administered:
MIGALASTAT plasma
Homo sapiens
population: unhealthy
age:
sex: F
food status:
9970.3 ng*h/mL
250 mg 1 times / 2 days steady, oral
dose: 250 mg
route of administration: oral
experiment type: steady
co-administered:
MIGALASTAT plasma
Homo sapiens
population: unhealthy
age:
sex: F
food status:
T1/2

T1/2

ValueDoseCo-administeredAnalytePopulation
4.9 h
150 mg single, oral
dose: 150 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
MIGALASTAT plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: UNKNOWN
food status: HIGH-FAT
3.9 h
150 mg single, oral
dose: 150 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
MIGALASTAT plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: UNKNOWN
food status: FASTED
Funbound

Funbound

ValueDoseCo-administeredAnalytePopulation
100%
150 mg 1 times / 2 days steady-state, oral
dose: 150 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
MIGALASTAT plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: UNKNOWN
food status: FASTED
Doses

Doses

DosePopulationAdverse events​
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Other AEs: Myalgia, Oropharyngeal pain...
Other AEs:
Myalgia (8.82%)
Oropharyngeal pain (8.82%)
Hematuria (8.82%)
Insomnia (8.82%)
Dizziness (5.88%)
Hypoaesthesia (5.88%)
Asthenia (5.88%)
Chest pain (5.88%)
Weight increased (5.88%)
Torticollis (5.88%)
Atrial fibrillation (5.88%)
Myalgia (8.82%)
Oropharyngeal pain (8.82%)
Hematuria (8.82%)
Insomnia (8.82%)
Dizziness (5.88%)
Hypoaesthesia (5.88%)
Asthenia (5.88%)
Chest pain (5.88%)
Weight increased (5.88%)
Torticollis (5.88%)
Atrial fibrillation (5.88%)
Sources:
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Other AEs: Headache, Nasopharyngitis...
Other AEs:
Headache (35%)
Nasopharyngitis (18%)
Urinary tract infection (15%)
Cystitis (15%)
Kidney infection (15%)
Nausea (12%)
Pyrexia (12%)
Abdominal pain (9%)
Back pain (9%)
Cough (9%)
Diarrhea (9%)
Epistaxis (9%)
Headache (35%)
Nasopharyngitis (18%)
Urinary tract infection (15%)
Cystitis (15%)
Kidney infection (15%)
Nausea (12%)
Pyrexia (12%)
Abdominal pain (9%)
Back pain (9%)
Cough (9%)
Diarrhea (9%)
Epistaxis (9%)
Sources:
150 mg 2 times / day multiple, oral
Highest studied dose
Dose: 150 mg, 2 times / day
Route: oral
Route: multiple
Dose: 150 mg, 2 times / day
Sources:
healthy
2000 mg single, oral
Highest studied dose
healthy
250 mg 1 times / 2 days multiple, oral
Highest studied dose
Dose: 250 mg, 1 times / 2 days
Route: oral
Route: multiple
Dose: 250 mg, 1 times / 2 days
Sources:
unhealthy
250 mg 2 times / day multiple, oral
Highest studied dose
Dose: 250 mg, 2 times / day
Route: oral
Route: multiple
Dose: 250 mg, 2 times / day
Sources:
unhealthy
450 mg single, oral
Highest studied dose
unhealthy
10 mg/kg single, intravenous
Highest studied dose
Dose: 10 mg/kg
Route: intravenous
Route: single
Dose: 10 mg/kg
Sources:
healthy
AEs

AEs

AESignificanceDosePopulation
Asthenia 5.88%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Asthenia 5.88%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Atrial fibrillation 5.88%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Atrial fibrillation 5.88%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Chest pain 5.88%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Chest pain 5.88%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Dizziness 5.88%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Dizziness 5.88%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Hypoaesthesia 5.88%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Hypoaesthesia 5.88%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Torticollis 5.88%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Torticollis 5.88%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Weight increased 5.88%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Weight increased 5.88%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Hematuria 8.82%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Hematuria 8.82%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Insomnia 8.82%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Insomnia 8.82%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Myalgia 8.82%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Myalgia 8.82%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Oropharyngeal pain 8.82%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Oropharyngeal pain 8.82%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, >16 years
n = 34
Health Status: unhealthy
Condition: Fabry disease
Age Group: >16 years
Sex: M+F
Population Size: 34
Sources:
Nausea 12%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Nausea 12%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Pyrexia 12%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Pyrexia 12%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Cystitis 15%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Cystitis 15%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Kidney infection 15%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Kidney infection 15%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Urinary tract infection 15%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Urinary tract infection 15%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Nasopharyngitis 18%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Nasopharyngitis 18%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Headache 35%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Headache 35%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Abdominal pain 9%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Abdominal pain 9%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Back pain 9%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Back pain 9%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Cough 9%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Cough 9%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Diarrhea 9%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Diarrhea 9%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Epistaxis 9%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Epistaxis 9%
123 mg 1 times / 2 days steady, oral
Recommended
Dose: 123 mg, 1 times / 2 days
Route: oral
Route: steady
Dose: 123 mg, 1 times / 2 days
Sources:
unhealthy, range: 16-72 years
n = 34
Health Status: unhealthy
Condition: Fabry disease | amenable galactosidase alpha gene (GLA) variant
Age Group: range: 16-72 years
Sex: M+F
Population Size: 34
Sources:
Overview

Overview

Drug as perpetrator​

Drug as perpetrator​

TargetModalityActivityMetaboliteClinical evidence
no [IC50 >500 uM]
no [IC50 >500 uM]
no [IC50 >500 uM]
no [IC50 >500 uM]
no [IC50 >500 uM]
no [IC50 >500 uM]
no [IC50 >500 uM]
no [IC50 >500 uM]
no [IC50 >500 uM]
no [IC50 >500 uM]
no [IC50 >500 uM]
no [IC50 >500 uM]
no [IC50 >500 uM]
no [Inhibition 500 uM]
no [Inhibition 500 uM]
no [Inhibition 500 uM]
no [Inhibition 500 uM]
no [Inhibition 500 uM]
no [Inhibition 500 uM]
no [Inhibition 500 uM]
no
no
no
Drug as victimTox targets

Tox targets

Sourcing

Sourcing

Vendor/AggregatorIDURL
PubMed

PubMed

TitleDatePubMed
[Enzyme replacement therapy of lysosomal storage diseases].
2010 Dec
α-Galactosidase aggregation is a determinant of pharmacological chaperone efficacy on Fabry disease mutants.
2012 Aug 17
Patents

Patents

Sample Use Guides

The recommended dosage regimen in adults and adolescents 16 years and older is 123 mg migalastat (1 capsule) once every other day at the same time of day.
Route of Administration: Oral
Migalastat (1-Deoxygalactonojirimycin) was the most potent inhibitor of alpha-Gal A in Fabry lymphoblasts with an IC50 value of 0.04 uM. Inclusion of 1-deoxygalactonojirimycin at 100 uM in culture medium of Fabry lymphoblasts increased the intracellular alpha-Gal A activity by 14-fold.
Substance Class Chemical
Created
by admin
on Fri Jun 25 22:48:12 UTC 2021
Edited
by admin
on Fri Jun 25 22:48:12 UTC 2021
Record UNII
CLY7M0XD20
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
MIGALASTAT HYDROCHLORIDE
USAN   WHO-DD  
USAN  
Official Name English
MIGALASTAT HYDROCHLORIDE [WHO-DD]
Common Name English
GALAFOLD
Brand Name English
MIGALASTAT HYDROCHLORIDE [JAN]
Common Name English
AT-1001 (MIGALASTAT HYDROCHLORIDE)
Code English
MIGALASTAT HCL
Common Name English
MIGALASTAT HYDROCHLORIDE [MI]
Common Name English
MIGALASTAT HYDROCHLORIDE [USAN]
Common Name English
MIGALASTAT HYDROCHLORIDE [ORANGE BOOK]
Common Name English
Classification Tree Code System Code
FDA ORPHAN DRUG 180003
Created by admin on Fri Jun 25 22:48:12 UTC 2021 , Edited by admin on Fri Jun 25 22:48:12 UTC 2021
EU-Orphan Drug EU/3/06/368
Created by admin on Fri Jun 25 22:48:12 UTC 2021 , Edited by admin on Fri Jun 25 22:48:12 UTC 2021
NCI_THESAURUS C87006
Created by admin on Fri Jun 25 22:48:12 UTC 2021 , Edited by admin on Fri Jun 25 22:48:12 UTC 2021
Code System Code Type Description
MERCK INDEX
M11983
Created by admin on Fri Jun 25 22:48:12 UTC 2021 , Edited by admin on Fri Jun 25 22:48:12 UTC 2021
PRIMARY
DRUG BANK
DBSALT000828
Created by admin on Fri Jun 25 22:48:12 UTC 2021 , Edited by admin on Fri Jun 25 22:48:12 UTC 2021
PRIMARY
ChEMBL
CHEMBL110458
Created by admin on Fri Jun 25 22:48:12 UTC 2021 , Edited by admin on Fri Jun 25 22:48:12 UTC 2021
PRIMARY
NCI_THESAURUS
C78121
Created by admin on Fri Jun 25 22:48:12 UTC 2021 , Edited by admin on Fri Jun 25 22:48:12 UTC 2021
PRIMARY
RXCUI
2054264
Created by admin on Fri Jun 25 22:48:12 UTC 2021 , Edited by admin on Fri Jun 25 22:48:12 UTC 2021
PRIMARY
PUBCHEM
11644097
Created by admin on Fri Jun 25 22:48:12 UTC 2021 , Edited by admin on Fri Jun 25 22:48:12 UTC 2021
PRIMARY
FDA UNII
CLY7M0XD20
Created by admin on Fri Jun 25 22:48:12 UTC 2021 , Edited by admin on Fri Jun 25 22:48:12 UTC 2021
PRIMARY
CAS
75172-81-5
Created by admin on Fri Jun 25 22:48:12 UTC 2021 , Edited by admin on Fri Jun 25 22:48:12 UTC 2021
PRIMARY
EVMPD
SUB177095
Created by admin on Fri Jun 25 22:48:12 UTC 2021 , Edited by admin on Fri Jun 25 22:48:12 UTC 2021
PRIMARY
Related Record Type Details
PARENT -> SALT/SOLVATE
Related Record Type Details
ACTIVE MOIETY