Miglustat

Not to be confused with migalastat.

Miglustat
Clinical data
Trade namesZavesca, Brazaves, Opfolda
Other namesOGT 918, 1,5-(butylimino)-1,5-dideoxy-D-glucitol, N-butyl-deoxynojirimycin
AHFS/Drugs.comMonograph
MedlinePlusa604015
License data
Pregnancy
category
Routes of
administration		By mouth
ATC code
Legal status
Legal status
Pharmacokinetic data
Bioavailability97%
Protein bindingNil
MetabolismNil
Elimination half-life6–7 hours
ExcretionKidney, unchanged
Identifiers
  • (2R,3R,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol
CAS Number
PubChem CID
IUPHAR/BPS
DrugBank
ChemSpider
UNII
KEGG
ChEBI
ChEMBL
CompTox Dashboard (EPA)
ECHA InfoCard100.216.074 Edit this at Wikidata
Chemical and physical data
FormulaC10H21NO4
Molar mass219.281 g·mol−1
3D model (JSmol)
  • OC[C@H]1N(CCCC)C[C@H](O)[C@@H](O)[C@@H]1O
  • InChI=1S/C10H21NO4/c1-2-3-4-11-5-8(13)10(15)9(14)7(11)6-12/h7-10,12-15H,2-6H2,1H3/t7-,8+,9-,10-/m1/s1 checkY
  • Key:UQRORFVVSGFNRO-UTINFBMNSA-N checkY
  (verify)

Miglustat, sold under the brand name Zavesca among others, is a medication used to treat type I Gaucher disease[7] and Pompe disease.[10]

It was approved for medical use in the European Union in November 2002,[7][11] and for medical use in the United States in July 2003.[12][13]

  1. ^ "Miglustat (Zavesca) Use During Pregnancy". Drugs.com. 4 February 2020. Archived from the original on 28 November 2020. Retrieved 7 August 2020.
  2. ^ "Summary for ARTG Entry:122957 Zavesca miglustat 100 mg capsules blister pack". Therapeutic Goods Administration.[permanent dead link]
  3. ^ "Zavesca (miglustat) 100 mg hard capsules - Summary of Product Characteristics (SmPC)". (emc). Archived from the original on 25 February 2021. Retrieved 7 August 2020.
  4. ^ "Zavesca- miglustat capsule". DailyMed. 20 April 2023. Retrieved 2 October 2023.
  5. ^ "Yargesa- miglustat capsule". DailyMed. 12 July 2023. Retrieved 2 October 2023.
  6. ^ https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215211s000lbl.pdf [bare URL PDF]
  7. ^ a b c "Zavesca EPAR". European Medicines Agency (EMA). 17 September 2018. Archived from the original on 5 August 2020. Retrieved 7 August 2020.
  8. ^ "Opfolda EPAR". European Medicines Agency (EMA). 7 July 2023. Retrieved 7 July 2023.
  9. ^ "Yargesa EPAR". European Medicines Agency. 11 April 2023. Retrieved 2 October 2023.
  10. ^ Cite error: The named reference Opfolda: Pending EC decision was invoked but never defined (see the help page).
  11. ^ European Medicines Agency. Human Medicines Database. Zavesca (miglustat) Archived 7 January 2016 at the Wayback Machine Page Accessed 1 September 2014.
  12. ^ "Drug Approval Package: Zavesca (Miglustat) NDA #021348". U.S. Food and Drug Administration (FDA). 4 April 2002. Archived from the original on 27 February 2021. Retrieved 9 August 2020.
  13. ^ Actelion Press Release August 2003. Zavesca approved -- first oral treatment option for type 1 Gaucher disease Archived 3 March 2016 at the Wayback Machine