MIGALASTAT HYDROCHLORIDE

Details

Stereochemistry	ABSOLUTE
Molecular Formula	C6H13NO4.ClH
Molecular Weight	199.633
Optical Activity	( + )
Defined Stereocenters	4 / 4
E/Z Centers	0
Charge	0

SHOW SMILES / InChI

Structure Search

SMILES

Cl.OC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O

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	36.461
Charge	0
Count

Stereochemistry	ACHIRAL
Additional Stereochemistry	No
Defined Stereocenters	0 / 0
E/Z Centers	0
Optical Activity	NONE

Molecular Formula	C6H13NO4
Molecular Weight	163.1717
Charge	0
Count

Stereochemistry	ABSOLUTE
Additional Stereochemistry	No
Defined Stereocenters	4 / 4
E/Z Centers	0
Optical Activity	UNSPECIFIED

Description
Sources: https://www.ncbi.nlm.nih.gov/pubmed/27351440 | http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Summary_for_the_public/human/004059/WC500208437.pdf

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.

Originator

Approval Year

Targets

Primary Target	Pharmacology	Condition	Potency
Alpha-galactosidase A 2 Target ID: CHEMBL2524 Sources: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/004059/WC500208434.pdf	Chaperone 14		0.04 µM [IC50]

Conditions

Condition	Modality	Targets	Highest Phase	Product
Fabry's disease 6 Sources: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/004059/WC500208434.pdf	Primary		Approved 8583	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 2016

C_max

Value	Dose	Analyte	Population
2029 ng/mL OTHER GOV https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	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
1561 ng/mL OTHER GOV https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	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
1323 ng/mL OTHER GOV https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	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

AUC

Value	Dose	Analyte	Population
8941.6 ng*h/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT00304512	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:
13217.2 ng*h/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT00304512	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:
2628.9 ng*h/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT00304512	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:
10637.9 ng*h/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT00304512	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:
8581.9 ng*h/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT00304512	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:
14850.6 ng*h/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT00304512	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:
9970.3 ng*h/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT00304512	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:
3191.6 ng*h/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT00304512	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:
2300.3 ng*h/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT00304512	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:
10638 ng × h/mL OTHER GOV https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	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
9805 ng × h/mL OTHER GOV https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	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
6132 ng × h/mL OTHER GOV https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	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

T_1/2

Value	Dose	Co-administered	Analyte	Population
3.9 h OTHER GOV https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	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
4.9 h OTHER GOV https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	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

F_unbound

Value	Dose	Co-administered	Analyte	Population
100% OTHER GOV https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	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

Dose	Population	Adverse 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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	unhealthy, >16 years Health Status: unhealthy Age Group: >16 years Sex: M+F Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf
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: https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/208623lbl.pdf	unhealthy, range: 16-72 years Health Status: unhealthy Age Group: range: 16-72 years Sex: M+F Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/208623lbl.pdf	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: https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/208623lbl.pdf
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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	healthy Health Status: healthy Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf
2000 mg single, oral Highest studied dose Dose: 2000 mg Route: oral Route: single Dose: 2000 mg Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	healthy Health Status: healthy Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf
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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	unhealthy Health Status: unhealthy Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf
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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	unhealthy Health Status: unhealthy Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf
450 mg single, oral Highest studied dose Dose: 450 mg Route: oral Route: single Dose: 450 mg Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	unhealthy Health Status: unhealthy Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf
10 mg/kg single, intravenous Highest studied dose Dose: 10 mg/kg Route: intravenous Route: single Dose: 10 mg/kg Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	healthy Health Status: healthy Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf	Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf

AEs

Overview

CYP3A4	CYP2C9	CYP2D6	hERG
inducer 1087	inhibitor 2438	inhibitor 2507	inhibitor 2217

OverviewOther

Other Inhibitor	Other Substrate	Other Inducer
CYP1A2 2153 CYP2A6 879 CYP2B6 926 CYP2C8 1057 CYP2C19 2057 CYP2E1 1060 CYP3A4/5 296 BCRP 910 P-gp 1741 BSEP 550 OATP1B1 1079 OATP1B3 961 OAT1 725 OAT3 747 OCT1 620 OCT2 779 MATE1 415 MATE2 267	BCRP 697 P-gp 2052 MATE1 182 MATE2 98 OAT1 395 OCT2 434 OAT3 391	CYP1A2 832 CYP2B6 669

Drug as perpetrator

Target	Modality	Activity
BCRP 985	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=353 Page: 353.0	no [IC50 >500 uM]
BSEP 554	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=353 Page: 353.0	no [IC50 >500 uM]
CYP2A6 911	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=336 Page: 332;336	no [IC50 >500 uM]
CYP2C8 1117	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=336 Page: 332;336	no [IC50 >500 uM]
MATE1 416	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=353 Page: 353.0	no [IC50 >500 uM]
MATE2 267	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=353 Page: 353.0	no [IC50 >500 uM]
OAT1 730	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=353 Page: 353.0	no [IC50 >500 uM]
OAT3 749	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=353 Page: 353.0	no [IC50 >500 uM]
OATP1B1 1095	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=353 Page: 353.0	no [IC50 >500 uM]
OATP1B3 970	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=353 Page: 353.0	no [IC50 >500 uM]
OCT1 656	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=353 Page: 353.0	no [IC50 >500 uM]
OCT2 782	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=353 Page: 353.0	no [IC50 >500 uM]
P-gp 1932	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=353 Page: 353.0	no [IC50 >500 uM]
CYP1A2 2333	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=336 Page: 332;336	no [Inhibition 500 uM]
CYP2B6 1160	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=336 Page: 332;336	no [Inhibition 500 uM]
CYP2C19 2141	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=336 Page: 332;336	no [Inhibition 500 uM]
CYP2C9 2497	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=336 Page: 332;336	no [Inhibition 500 uM]
CYP2D6 2546	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=336 Page: 332;336	no [Inhibition 500 uM]
CYP2E1 1146	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=336 Page: 332;336	no [Inhibition 500 uM]
CYP3A4/5 357	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=336 Page: 332;336	no [Inhibition 500 uM]
CYP1A2 2333	inducer 1966 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=338 Page: 338.0	no
CYP2B6 1160	inducer 1966 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=341 Page: 341.0	no
CYP3A4 2633	inducer 1966 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=338 Page: 338.0	no

Drug as victim

Target	Modality	Activity
BCRP 697	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=345 Page: 345.0	no
MATE1 182	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=345 Page: 345.0	no
MATE2 98	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=345 Page: 345.0	no
OAT1 395	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=345 Page: 345.0	no
OAT3 391	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=345 Page: 345.0	no
OCT2 434	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=345 Page: 345.0	no
P-gp 2052	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=345 Page: 345.0	no

Tox targets

Target	Modality	Activity	Metabolite	Clinical evidence
hERG 2263	inhibitor 2237 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf#page=41 Page: 41.0

Sourcing

Vendor/Aggregator	ID	URL
001 Chemical	DY34270
001 Chemical	DY34271
1PlusChem LLC	1P01CBVD
AA Blocks	AA0039C7	http://www.aablocks.com/goods/Goods/detail?id=AA0039C7
ABI Chem	AC1L41WN
AbovChem LLC	HY-14929A
Achemtek	0102-013497	http://www.achemtek.com/108147-54-2
AK Scientific	8524AJ
Alfa Chemistry	108147-54-2
Alfa Chemistry	ACM108147542
Angene	AGN-PC-0O65G0	http://www.angenechemical.com/productshow/AGN-PC-0O65G0.html
Apollo Scientific	BICL4269
Apollo Scientific	OR2365T
BioSynth	W-203722
BLD Pharm	BD293196	http://www.bldpharm.com/products/108147-54-2.html
BOC Sciences	108147-54-2
Cayman Chemical	17179
Chem Scene	CS-6380
Chemhere	Achem011131
Chemieliva Pharmaceutical Co., Ltd	PBCM1067923
ChemShuttle	171867
Chemspace	CSC015381425
Chemspace	CSC015685547
Chemspace	CSC015915741
Chemspace	CSC015923687
ChemTik	CTK4A5883
Compound Cloud	11644097
Compound Cloud	176077
eMolecules	28294266
eMolecules	300656804
eMolecules	43444744
eMolecules	76740242
Enamine	BBV-39696456
Enamine	BBV-77537908
Enamine	BBV-77614131
Enamine	BBV-77727648
Lab Network	LN01314171
mcule	MCULE-3687609653
mcule	MCULE-3759351391
MedChem Express	HY-14929A
Molcore	MC34270
Molcore	MC34271
MolPort	MolPort-039-016-078
Norris Pharm	NSZB-A005411
Parchem	29429	http://www.parchem.com/chemical-supplier-distributor/Migalastat-019429.aspx
Sigma Aldrich	D9641
Sigma Aldrich	D9641\|SIGMA
Smolecule	S535437	http://www.smolecule.com/products/s535437
SynQuest	4H56-5-00
SynQuest	87535
THE BioTek	bt-276368	https://www.thebiotek.com/product/inhibitors/bt-276368
THE BioTek	bt-315116	https://www.thebiotek.com/product/others/bt-315116
Toronto Research Chemicals	D231750
Toronto Research Chemicals	D236500
ZINC	ZINC000001636704	http://zinc15.docking.org/substances/ZINC000001636704

PubMed

Title	Date	PubMed
α-Galactosidase aggregation is a determinant of pharmacological chaperone efficacy on Fabry disease mutants.	2012-08-17	22773828
[Enzyme replacement therapy of lysosomal storage diseases].	2010-12	21211680

Patents

Sample Use Guides

In Vivo Use Guide

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

In Vitro Use Guide

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 `Mon Mar 31 18:05:31 GMT 2025` Edited by `admin` on `Mon Mar 31 18:05:31 GMT 2025`
Record UNII	CLY7M0XD20
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

GALAFOLD

Preferred Name

English

MIGALASTAT HYDROCHLORIDE

Official Name

English

3,4,5-Piperidinetriol, 2-(hydroxymethyl)-, hydrochloride (1:1), (2R,3S,4R,5S)-

Systematic Name

English

MIGALASTAT HYDROCHLORIDE [JAN]

Common Name

English

AT-1001 (MIGALASTAT HYDROCHLORIDE)

Code

English

Migalastat hydrochloride [WHO-DD]

Common Name

English

(+)-MIGALASTAT HYDROCHLORIDE

Common Name

English

MIGALASTAT HCL

Common Name

English

MIGALASTAT HYDROCHLORIDE [MI]

Common Name

English

MIGALASTAT HYDROCHLORIDE [USAN]

Common Name

English

1-Deoxygalactonojirimycin hydrochloride

Common Name

English

MIGALASTAT HYDROCHLORIDE [ORANGE BOOK]

Common Name

English

Classification Tree Code System Code

FDA ORPHAN DRUG

180003