User:Mr. Ibrahem/Miglustat

From Wikipedia, the free encyclopedia
Mr. Ibrahem/Miglustat
Clinical data
Trade namesZavesca, Brazaves
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
Drug classGlucosylceramide synthase inhibitor[2]
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
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, is a medication used to treat Gaucher disease type 1 (GD1) and Niemann–Pick disease type C (NPC).[5] It is taken by mouth.[2]

Common side effects include weight loss, shaking, diarrhea, increased intestinal gas, and abdominal pain.[5] Other side effects may include peripheral neuropathy and low platelets.[2] Safety in pregnancy is unclear.[2] It is a glucosylceramide synthase inhibitor.[2]

Miglustat was approved for medical use Europe in 2002 and the United States in 2003.[5][2] It is available as a generic medication.[6] In the United Kingdom 4 weeks of medication costs the NHS about £3,400 as of 2021.[6] This amount in the United States costs about 23,000 USD.[7]

References[edit]

  1. ^ a b "Miglustat (Zavesca) Use During Pregnancy". Drugs.com. 4 February 2020. Archived from the original on 28 November 2020. Retrieved 7 August 2020.
  2. ^ a b c d e f g "Miglustat Monograph for Professionals". Drugs.com. Archived from the original on 4 March 2021. Retrieved 18 November 2021.
  3. ^ "Summary for ARTG Entry:122957 Zavesca miglustat 100 mg capsules blister pack". Therapeutic Goods Administration.{{cite web}}: CS1 maint: url-status (link)
  4. ^ "Zavesca (miglustat) 100 mg hard capsules - Summary of Product Characteristics (SmPC)". (emc). Archived from the original on 25 February 2021. Retrieved 7 August 2020.
  5. ^ a b c d e f "Zavesca EPAR". European Medicines Agency (EMA). Archived from the original on 5 August 2020. Retrieved 7 August 2020.
  6. ^ a b BNF 81: March-September 2021. BMJ Group and the Pharmaceutical Press. 2021. p. 1117. ISBN 978-0857114105.
  7. ^ "Miglustat Prices, Coupons & Patient Assistance Programs". Drugs.com. Archived from the original on 18 April 2021. Retrieved 18 November 2021.