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migalastat 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | BioActivity |

Stem definition	Drug id	CAS RN
enzyme inhibitors	5110	108147-54-2

Description:

Molecule	Description
Molfile Inchi Smiles Synonyms: migalastat HCl GR181413A 1-Deoxygalactonojirimycin hydrochloride 1-Deoxygalactonojirimycin migalastat migalastat hydrochloride galafold	Migalastat is a pharmacological chaperone that is designed to selectively and reversibly bind with high affinity to the active sites of certain mutant forms of alpha-Gal A, the genotypes of which are referred to as amenable mutations. Migalastat binding stabilizes these mutant forms of alpha-Gal A in the endoplasmic reticulum and facilitates their proper trafficking to lysosomes where dissociation of migalastat restores alpha-Gal A activity, leading to the catabolism of GL-3 and related substrates. Molecular weight: 163.17 Formula: C6H13NO4 CLOGP: -0.40 LIPINSKI: 0 HAC: 5 HDO: 5 TPSA: 92.95 ALOGS: 0.50 ROTB: 1 Status: ONP Legend: OFP - off patent OFM - off market ONP - on patent

Molecule

Description

Molfile Inchi Smiles

Synonyms:

migalastat HCl
GR181413A
1-Deoxygalactonojirimycin hydrochloride
1-Deoxygalactonojirimycin
migalastat
migalastat hydrochloride
galafold

Migalastat is a pharmacological chaperone that is designed to selectively and reversibly bind with high affinity to the active sites of certain mutant forms of alpha-Gal A, the genotypes of which are referred to as amenable mutations. Migalastat binding stabilizes these mutant forms of alpha-Gal A in the endoplasmic reticulum and facilitates their proper trafficking to lysosomes where dissociation of migalastat restores alpha-Gal A activity, leading to the catabolism of GL-3 and related substrates.

Molecular weight: 163.17
Formula: C6H13NO4
CLOGP: -0.40
LIPINSKI: 0
HAC: 5
HDO: 5
TPSA: 92.95
ALOGS: 0.50
ROTB: 1

Status: ONP

Legend:
OFP - off patent
OFM - off market
ONP - on patent

Drug dosage:

Dose	Unit	Route
61.50	mg	O

ADMET properties:

Property	Value	Reference
S (Water solubility)	200 mg/mL	Bocci G, Oprea TI, Benet LZ
BDDCS (Biopharmaceutical Drug Disposition Classification System)	3	Bocci G, Oprea TI, Benet LZ
CL (Clearance)	2.22 mL/min/kg	Kawashima H, Watanabe R, Esaki T, Kuroda M, Nagao C, Natsume-Kitatani Y, Ohashi R, Komura H, Mizuguchi K
BA (Bioavailability)	0.75 %	Kawashima H, Watanabe R, Esaki T, Kuroda M, Nagao C, Natsume-Kitatani Y, Ohashi R, Komura H, Mizuguchi K

Approvals:

Date	Agency	Company
May 26, 2016	EMA	Amicus Therapeutics UK Ltd
Aug. 8, 2018	FDA	AMICUS THERAPS US
March 23, 2018	PMDA	Amicus Therapeutics Inc

FDA Adverse Event Reporting System (Female)

None

FDA Adverse Event Reporting System (Male)

None

FDA Adverse Event Reporting System (Geriatric)

None

FDA Adverse Event Reporting System (Pediatric)

None

Pharmacologic Action:

Source	Code	Description
ATC	A16AX14	ALIMENTARY TRACT AND METABOLISM OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS Various alimentary tract and metabolism products

Drug Use | Suggest Off label Use Form| |View source of the data|

Disease	Relation	SNOMED_ID	DOID
Fabry's disease	indication	16652001	DOID:14499

🐶 Veterinary Drug Use

None

🐶 Veterinary products

None

Acid dissociation constants calculated using MoKa v3.0.0

Dissociation level	Dissociation constant	Type (acidic/basic)
pKa1	12.16	acidic
pKa2	8.5	Basic

Orange Book patent data (new drug applications)

Formulation strength	Trade name	Applicant	Application number	Approval date	Type	Dose form	Route	Patent number	Patent expiration date	Patent use
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	10383864	May 16, 2027	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	10406143	May 16, 2027	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	11241422	May 16, 2027	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	9000011	May 16, 2027	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	9480682	May 16, 2027	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	9987263	May 16, 2027	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	10525045	April 28, 2028	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	10925866	April 28, 2028	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	11033538	April 28, 2028	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	9999618	April 28, 2028	A METHOD OF REDUCING LEFT VENTRICULAR MASS INDEX (LVMI) IN A FABRY PATIENT BY ADMINISTERING MIGALASTAT
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	9999618	April 28, 2028	A METHOD OF REDUCING PODOCYTE GLOBOTRIAOSYLCERAMIDE (GL-3) IN A FABRY PATIENT BY ADMINISTERING MIGALASTAT
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	10813921	Feb. 12, 2029	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	8592362	Feb. 12, 2029	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	9095584	Feb. 12, 2029	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	RE48608	Feb. 12, 2029	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	10076514	March 15, 2037	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	11234972	March 15, 2037	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	10251873	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	10471053	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	10792278	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	10792279	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	10799491	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	10806727	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	10849889	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	10849890	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	10857141	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	10857142	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	10874655	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	10874656	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	10874657	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	11278536	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	11278537	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	11278538	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	11278539	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	11278540	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	11357761	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	11357762	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	11357763	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	11389436	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	11389437	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	11458128	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	11666564	May 30, 2038	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	11357784	Feb. 6, 2039	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	11642334	Feb. 20, 2039	THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	11633388	March 25, 2039	THE TREATMENT OF FABRY PATIENTS

Orange Book exclusivity data (new drug applications)

Formulation strength	Trade name	Applicant	Application number	Approval date	Type	Dose form	Route	Exclusivity date	Description
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	Aug. 10, 2023	NEW CHEMICAL ENTITY
EQ 123MG BASE	GALAFOLD	AMICUS THERAP US	N208623	Aug. 10, 2018	RX	CAPSULE	ORAL	Aug. 10, 2025	INDICATED FOR THE TREATMENT OF ADULTS WITH A CONFIRMED DIAGNOSIS OF FABRY DISEASE AND AN AMENABLE GALACTOSIDASE ALPHA GENE (GLA) VARIANT BASED ON IN VITRO ASSAY DATA

Bioactivity Summary:

Target	Class	Pharos	UniProt	Action	Type	Activity value (-log[M])	Bioact source	MoA source
Alpha-galactosidase A	Enzyme	P06280	AGAL_HUMAN	PHARMACOLOGICAL CHAPERONE	IC50	7.15	CHEMBL	DRUG LABEL
Lysosomal alpha-glucosidase	Enzyme	P10253	LYAG_HUMAN		IC50	5.22	CHEMBL
Non-lysosomal glucosylceramidase	Enzyme	Q9HCG7	GBA2_HUMAN		IC50	4	CHEMBL
Sucrase-isomaltase, intestinal	Enzyme	P14410	SUIS_HUMAN		IC50	6.59	CHEMBL
Glycogen debranching enzyme	Enzyme	P35573	GDE_HUMAN		IC50	5	CHEMBL
Lactase-phlorizin hydrolase	Enzyme	P09848	LPH_HUMAN		IC50	4.30	CHEMBL
Beta-galactosidase	Enzyme	P16278	BGAL_HUMAN		IC50	4.05	CHEMBL
Maltase-glucoamylase, intestinal	Enzyme	O43451	MGA_HUMAN		IC50	5.82	CHEMBL
Lactase-phlorizin hydrolase	Enzyme		LPH_RAT		IC50	4.14	CHEMBL
Alpha-galactosidase C	Enzyme		AGALC_ASPNG		IC50	5.74	CHEMBL
Alpha-galactosidase	Enzyme		AGAL_COFAR		IC50	8.52	CHEMBL
Beta-galactosidase	Enzyme		D3ZUM4_RAT		IC50	4.62	CHEMBL
Beta-galactosidase	Enzyme		BGAL_ECOLX		Ki	4.89	CHEMBL
Beta-galactosidase	Enzyme		BGAL_RHIML		Ki	4.90	CHEMBL

External reference:

ID	Source
C4XNY919FW	UNII
75172-81-5	SECONDARY_CAS_RN
D10359	KEGG_DRUG
4038521	VANDF
C2698220	UMLSCUI
CHEBI:135923	CHEBI
DGJ	PDB_CHEM_ID
CHEMBL110458	ChEMBL_ID
176077	PUBCHEM_CID
DB05018	DRUGBANK_ID
CHEMBL2107355	ChEMBL_ID
8700	INN_ID
C090092	MESH_SUPPLEMENTAL_RECORD_UI
10200	IUPHAR_LIGAND_ID
2054252	RXNORM
261328	MMSL
33035	MMSL
d08640	MMSL
017003	NDDF
017004	NDDF
765340000	SNOMEDCT_US
765341001	SNOMEDCT_US
773543004	SNOMEDCT_US

Pharmaceutical products:

Product	Category	Ingredients	NDC	Form	Quantity	Route	Marketing	Label
Galafold	HUMAN PRESCRIPTION DRUG LABEL	1	71904-100	CAPSULE	123 mg	ORAL	NDA	28 sections
Galafold	HUMAN PRESCRIPTION DRUG LABEL	1	71904-100	CAPSULE	123 mg	ORAL	NDA	28 sections