Migalastat information

Migalastat
Clinical data
Pronunciation	mi GAL a stat
Trade names	Galafold
Other names	DDIG, AT1001, 1-deoxygalactonojirimycin
AHFS/Drugs.com	Monograph
Pregnancy; category	AU: B3; ;
Routes of; administration	By mouth
ATC code	A16AX14 (WHO) ;
Legal status
Legal status	AU: S4 (Prescription only); CA: ℞-only; US: ℞-only; EU: Rx-only; In general: ℞ (Prescription only);
Pharmacokinetic data
Bioavailability	75%
Protein binding	None
Metabolites	O-glucuronides (<15%)
Elimination half-life	3–5 hours (single dose)
Excretion	Urine (77%), feces (20%)
Identifiers
	IUPAC name (2R,3S,4R,5S)-2-(Hydroxymethyl)-3,4,5-piperidinetriol;
CAS Number	108147-54-2 ; HCl: 75172-81-5 ;
PubChem CID	176077;
DrugBank	DB05018;
ChemSpider	153388;
UNII	C4XNY919FW; HCl: CLY7M0XD20 ;
KEGG	D10359; HCl: D05031;
ChEMBL	ChEMBL110458;
Chemical and physical data
Formula	C6H13NO4
Molar mass	163.173 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES C1[C@@H]([C@H]([C@H]([C@H](N1)CO)O)O)O;
	InChI InChI=1S/C6H13NO4/c8-2-3-5(10)6(11)4(9)1-7-3/h3-11H,1-2H2/t3-,4+,5+,6-/m1/s1; Key:LXBIFEVIBLOUGU-DPYQTVNSSA-N;

Migalastat, sold under the brand name Galafold, is a medication for the treatment of Fabry disease, a rare genetic disorder. It was developed by Amicus Therapeutics. The US Food and Drug Administration (FDA) granted it orphan drug status in 2004,^[5] and the European Commission followed in 2006.^[6] The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) granted the drug a marketing approval under the name Galafold in May 2016.^[7]^[8]^[9]

The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.^[10]

^ "Galafold (Amicus Therapeutics Pty Ltd)". tga.gov.au. Retrieved 29 March 2023.
^ "Prescription medicines: registration of new chemical entities in Australia, 2017". Therapeutic Goods Administration (TGA). 21 June 2022. Retrieved 9 April 2023.
^ "Prescription medicines and biologicals: TGA annual summary 2017". Therapeutic Goods Administration (TGA). 21 June 2022. Retrieved 31 March 2024.
^ "Genetic disorders". Health Canada. 9 May 2018. Retrieved 13 April 2024.
^ "Migalastat Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). Retrieved 16 September 2020.
^ "EU/3/06/368". European Medicines Agency (EMA). 17 September 2018. Retrieved 16 September 2020.
^ Cite error: The named reference Newswire was invoked but never defined (see the help page).
^ Cite error: The named reference EPAR was invoked but never defined (see the help page).
^ "Galafold EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 16 September 2020.
^ New Drug Therapy Approvals 2018 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2019. Retrieved 16 September 2020.

[1] "Galafold (Amicus Therapeutics Pty Ltd)". tga.gov.au. Retrieved 29 March 2023.

[2] "Prescription medicines: registration of new chemical entities in Australia, 2017". Therapeutic Goods Administration (TGA). 21 June 2022. Retrieved 9 April 2023.

[3] "Prescription medicines and biologicals: TGA annual summary 2017". Therapeutic Goods Administration (TGA). 21 June 2022. Retrieved 31 March 2024.

[4] "Genetic disorders". Health Canada. 9 May 2018. Retrieved 13 April 2024.

[Orphans-5] "Migalastat Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). Retrieved 16 September 2020.

[6] "EU/3/06/368". European Medicines Agency (EMA). 17 September 2018. Retrieved 16 September 2020.

[Newswire-7] Cite error: The named reference Newswire was invoked but never defined (see the help page).

[EPAR-8] Cite error: The named reference EPAR was invoked but never defined (see the help page).

[Galafold_EPAR-9] "Galafold EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 16 September 2020.

[10] New Drug Therapy Approvals 2018 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2019. Retrieved 16 September 2020.

Migalastat information

and 7 Related for: Migalastat information

Migalastat

Fabry disease

C6H13NO4

Nojirimycin

Globotriaosylceramide

ATC code A16

Pharmacological chaperone