Structure via PubChem · Public domain (PubChem)
migalastat
Sign in to saveAlso known as 1-deoxygalactonojirimycin
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.
Key facts
- Drug.image
- 1-Deoxygalactonojirimycin.svg
- Drug.image_class
- skin-invert-image
- Drug.width
- 125
- Drug.pronounce
- mi GAL a stat
- Drug.tradename
- Galafold
- Drug.MedlinePlus
- a625101
- Drug.DailyMedID
- Migalastat
- Drug.pregnancy_AU
- B3
- Drug.routes_of_administration
- By mouth
- Drug.class
- Pharmacological chaperone
- Drug.ATC_prefix
- A16
- Drug.ATC_suffix
- AX14
- Drug.legal_AU
- S4
- Drug.legal_CA
- Rx-only
- Drug.legal_US
- Rx-only
- Drug.legal_EU
- Rx-only
- Drug.legal_status
- JP: Rx-only
- Drug.bioavailability
- 75%
via Wikipedia infobox
Chemical data
- Formula
- C6H13NO4
- Molecular weight
- 163.17 g/mol
- IUPAC name
- (2R,3S,4R,5S)-2-(hydroxymethyl)piperidine-3,4,5-triol
- SMILES
- C1[C@@H]([C@H]([C@H]([C@H](N1)CO)O)O)O
- InChIKey
- LXBIFEVIBLOUGU-DPYQTVNSSA-N
- XLogP
- -2.3
- Polar surface area
- 93 Ų
- H-bond donors
- 5
- H-bond acceptors
- 5
- Formal charge
- 0
via PubChem
Drug data · ChEMBL
- Max clinical phase
- Approved
- Molecule type
- Small molecule
- First approval
- 2016
- Admin. routes
- oral
- Indications
- Fabry disease
via ChEMBL · EBI
Wikidata facts
- Mass
- 163.084
- Has use
- medication
Show 6 more facts
- isomeric SMILES
- C1[C@@H]([C@H]([C@H]([C@H](N1)CO)O)O)O
- canonical SMILES
- C1C(C(C(C(N1)CO)O)O)O
- chemical formula
- C₆H₁₃NO₄
- medical condition treated
- Fabry disease
- stylized name
- migALAstat
- defined daily dose
- 61.5
Sources (9)
via Wikidata · CC0
~7 min read
Encyclopedic overview
11 sectionsContents
- Medical uses
- Adverse effects
- Interactions
- Pharmacology
- Mechanism of action
- Pharmacokinetics
- Chemistry
- History
- Society and culture
- Legal status
- References
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).
Excerpted from Wikipedia’s “migalastat” article, available under the CC BY-SA 4.0 licence.