Migalastat

Migalastat
Clinical data
Pronunciation	mi GAL a stat
Trade names	Galafold
Other names	DDIG, AT1001, 1-deoxygalactonojirimycin
AHFS/Drugs.com	Monograph
MedlinePlus	a625101
License data	US DailyMed: Migalastat;
Pregnancy; category	AU: B3; ;
Routes of; administration	By mouth
Drug class	Pharmacological chaperone
ATC code	A16AX14 (WHO) ;
Legal status
Legal status	AU: S4 (Prescription only); CA: ℞-only; US: ℞-only; EU: Rx-only; JP: Rx-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; HCl: 11644097;
DrugBank	DB05018; HCl: DBSALT000828;
ChemSpider	153388; HCl: 9818836;
UNII	C4XNY919FW; HCl: CLY7M0XD20;
KEGG	D10359; HCl: D05031;
ChEMBL	ChEMBL110458; HCl: ChEMBL2107355;
CompTox Dashboard (EPA)	DTXSID201022520 ;
Chemical and physical data
Formula	C6H13NO4
Molar mass	163.173 g·mol−1
3D model (JSmol)	Interactive image; HCl: Interactive image;
	SMILES C1[C@@H]([C@H]([C@H]([C@H](N1)CO)O)O)O; ; HCl: Cl.OC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O;
	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; ; HCl: 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; Key:ZJIHMALTJRDNQI-OLALXQGDSA-N;

Migalastat, sold under the brand name Galafold, is a medication used for the treatment of Fabry disease, a rare genetic disorder. Migalastat is an alpha-galactosidase A (alpha-Gal A) pharmacological chaperone. It was developed by Amicus Therapeutics.

The most common adverse drug reactions include headache, nasal and throat irritation (nasopharyngitis), urinary tract infection, nausea, and fever (pyrexia).

Migalastat was authorized for medical use in the European Union in May 2016, and approved for medical use in the United States in August 2018. The US Food and Drug Administration considers it to be a first-in-class medication.