Miglustat

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Medication

title: "Miglustat" type: doc version: 1 created: 2026-02-28 author: "Wikipedia contributors" status: active scope: public tags: ["iminosugars", "orphan-drugs", "piperidines", "transferase-inhibitors"] description: "Medication" topic_path: "general/iminosugars" source: "https://en.wikipedia.org/wiki/Miglustat" license: "CC BY-SA 4.0" wikipedia_page_id: 0 wikipedia_revision_id: 0

::summary Medication ::

::data[format=table title="Infobox drug"]

Field	Value
verifiedrevid	462252392
type
image	Miglustat.svg
image_class	skin-invert-image
width	250
caption
tradename	Zavesca, Brazaves, Opfolda
Drugs.com
MedlinePlus	a604015
licence_CA
licence_EU	yes
DailyMedID	Miglustat
licence_US	Miglustat
pregnancy_AU	D
pregnancy_AU_comment
routes_of_administration	By mouth
ATC_prefix	A16
ATC_suffix	AX06
ATC_supplemental
legal_AU	S4
legal_AU_comment
legal_BR
legal_CA
legal_DE
legal_NZ
legal_UK	POM
legal_UK_comment
legal_US	Rx-only
legal_US_comment
legal_EU	Rx-only
legal_EU_comment
legal_UN
legal_status
	bioavailability
protein_bound	Nil
metabolism	Nil
elimination_half-life	6–7 hours
excretion	Kidney, unchanged
	CAS_number_Ref
CAS_number	72599-27-0
PubChem	51634
IUPHAR_ligand	4841
DrugBank_Ref
DrugBank	DB00419
ChemSpiderID_Ref
ChemSpiderID	46764
UNII_Ref
UNII	ADN3S497AZ
KEGG	D05032
ChEBI_Ref
ChEBI	50381
ChEMBL_Ref
ChEMBL	1029
synonyms	OGT 918, 1,5-(butylimino)-1,5-dideoxy-D-glucitol, N-butyl-deoxynojirimycin (NB-DNJ)
	IUPAC_name
C	10
H	21
N	1
O	4
SMILES	OC[C@H]1N(CCCC)CC@H C@@H[C@@H]1O
StdInChI_Ref
StdInChI	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
StdInChIKey_Ref
StdInChIKey	UQRORFVVSGFNRO-UTINFBMNSA-N
::

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

It was approved for medical use in the European Union in November 2002, and for medical use in the United States in July 2003.

Medical uses

Miglustat is indicated to treat adults with mild to moderate type I Gaucher disease for whom enzyme replacement therapy is unsuitable.

In the European Union, miglustat (Opfolda), in combination with cipaglucosidase alfa, is a long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid α‑glucosidase [GAA] deficiency).

Contraindications

Miglustat is contraindicated for people with neurological conditions, kidney problems, women who are pregnant, and men and women planning to conceive a child.

Adverse effects

Serious side effects include pain, burning, numbness or tingling in the hands, arms, legs, or feet; shaking hands that cannot be controlled; changes in vision; and easy bruising or bleeding. Common side effects include gastrointestinal effects (including diarrhea, stomach pain or bloating, gas, loss of appetite, weight loss, upset stomach, vomiting, constipation), dry mouth, muscular effects (including weakness, muscle cramps, especially in the legs, feeling of heaviness in the arms or legs, unsteadiness when walking), back pain, dizziness, nervousness, headache, memory problems, and difficult or irregular menstruation (period).

Mechanism of action

Type I Gaucher's disease is an autosomal recessive disorder; parents are generally healthy carriers with one functional and one mutated (nonfunctioning) copy of the Gaucher disease gene, GBA. People with type I Gaucher have a defect in the enzyme called glucocerebrosidase (also known as acid β-glucosidase). Glucocerebrosidase is an enzyme, and its function is to convert glucocerebroside (also known as glucosylceramide) into ceramide and glucose. When this enzyme doesn't work, glucocerebroside accumulates, which in turn causes liver and spleen enlargement, changes in the bone marrow and blood, and bone disease. Miglustat functions as a competitive and reversible inhibitor of the enzyme glucosylceramide synthase, the initial enzyme in a series of reactions which results in the synthesis of most glycosphingolipids.

Earlier treatments on the market (imiglucerase (approved in 1995), velaglucerase (approved in 2010), taliglucerase alfa (Elelyso) (approved in 2012)) are enzyme replacement therapy—they are functioning versions of the enzyme that doesn't work. Miglustat, on the other hand, prevents the formation of the substance that builds up when the enzyme doesn't work; this is called substrate reduction therapy.

Chemistry

Miglustat is an iminosugar, a synthetic analogue of D-glucose and a white to off-white crystalline solid that has a bitter taste.

Society and culture

Legal status

Miglustat has been approved in the EU, Canada, and Japan for treating progressive neurological complications in people with Niemann–Pick disease, type C (NPC).

On 26 April 2023, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Opfolda, intended for the treatment of glycogen storage disease type II (Pompe disease) in combination with cipaglucosidase alfa. The applicant for this medicinal product is Amicus Therapeutics Europe Limited. Opfolda is a hybrid medicine of Zavesca which has been authorized in the EU since 2002. Opfolda contains the same active substance as Zavesca but in a lower strength. It is also authorized for a different indication and can only be used in combination with cipaglucosidase alfa. Miglustat (Opfolda) was approved for medical use in the European Union in June 2023.

Research

In July 2004, Actelion started a clinical trial of miglustat to treat Tay–Sachs disease, particularly late-onset Tay–Sachs with an estimated enrollment of 10 subjects; the trial ended August 2007.

In November 2007, Actelion initiated a clinical trial with miglustat in people with cystic fibrosis (CF) who have the ΔF508 in both copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene; the study ended in March 2008. The cystic fibrosis trial showed no effect.

N-butyldeoxynojirimycin interferes with the secretion of hepatitis B virus and reduces the infectivity of HIV virions, in the latter case by preventing proper folding of the gp160 precursor glycoprotein to cause a conformational defect in mature gp120, which interferes with the process of fusion with host membranes.

References

(4 February 2020). "Miglustat (Zavesca) Use During Pregnancy".
(July 2023). "Summary for ARTG Entry:122957 Zavesca miglustat 100 mg capsules blister pack".
"Zavesca (miglustat) 100 mg hard capsules - Summary of Product Characteristics (SmPC)".
(20 April 2023). "Zavesca- miglustat capsule".
(12 July 2023). "Yargesa- miglustat capsule".
(September 2023). "Opfolda (miglustat) capsules prescribing information".
(17 September 2018). "Zavesca EPAR".
(7 July 2023). "Opfolda EPAR".
(11 April 2023). "Yargesa EPAR".
link. (7 January 2016 Page Accessed 1 September 2014.)
(4 April 2002). "Drug Approval Package: Zavesca (Miglustat) NDA #021348".
Actelion Press Release August 2003. [https://www.drugs.com/news/zavesca-approved-first-oral-option-type-1-gaucher-3351.html Zavesca approved -- first oral treatment option for type 1 Gaucher disease] {{Webarchive. link. (3 March 2016)
(2003). "The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) Gaucher disease: a position statement". Journal of Inherited Metabolic Disease.
American Society of Health-System Pharmacists, Inc. for the Public Library of Medicine. [https://www.medlineplus.gov/druginfo/meds/a604015.html Miglustat on MedlinePlus] Accessed 1 September 2014
(2012). "Gaucher disease and other storage disorders". Hematology. American Society of Hematology. Education Program.
(July 1999). "FDA Clinical Investigator Site Inspections: The Sponsor's Role". Drug Information Journal.
(2012). "Imiglucerase in the treatment of Gaucher disease: a history and perspective". Drug Design, Development and Therapy.
"Shire Announces FDA Approval Of VPRIV(TM) (velaglucerase Alfa For Injection) For The Treatment Of Type I Gaucher Disease". Medicalnewstoday.com.
(1 May 2012). "U.S. FDA approves Pfizer/Protalix drug for Gaucher". [[Chicago Tribune]].
Actelion. [https://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/EndocrinologicandMetabolicDrugsAdvisoryCommittee/UCM196758.pdf FDA Advisory Briefing Book for Miglustat (Ogt 918, Zavesca) in Niemann-Pick Type C Disease NDA 021-348/S-007] {{Webarchive. link. (9 May 2017 Prepared for the Endocrinologic and Metabolic Drugs Advisory Committee meeting, 1 December 2009)
(December 2011). "Therapeutic strategies for Gaucher disease: miglustat (NB-DNJ) as a pharmacological chaperone for glucocerebrosidase and the different thermostability of velaglucerase alfa and imiglucerase". Molecular Pharmaceutics.
European Medicines Agency 1 April 2003 [http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Scientific_Discussion/human/000435/WC500046721.pdf Scientific discussion related to approval of Zavesca] {{Webarchive. link. (24 September 2015 .)
link. (4 March 2016)
Staff, The Pharma Letter. 4 April 2012. [http://www.thepharmaletter.com/article/actelion-drops-setipiprant-gets-miglustat-approval-in-japan Actelion drops setipiprant, gets miglustat approval in Japan] {{Webarchive. link. (31 October 2018)
Kevin Grogan for PharmaTimes. 10 March 2010. [http://www.pharmatimes.com/article/10-03-10/FDA_rejects_Actelion_s_Zavesca_for_rare_NP-C_disease.aspx FDA rejects Actelion's Zavesca for rare NP-C disease] {{webarchive. link. (3 September 2014)
Actelion Press Release. 23 March 2010 [http://www.marketwired.com/press-release/Zavesca-Miglustat-First-Treatment-Available-Canada-Rare-Progressive-Niemann-Pick-Type-SIX-ATLN-1136457.htm Zavesca (Miglustat) First Treatment Available in Canada for Rare Progressive Niemann-Pick Type C Disease] {{Webarchive. link. (17 May 2017)
(December 2020). "Association of Miglustat With Swallowing Outcomes in Niemann-Pick Disease, Type C1". JAMA Neurology.
(26 April 2023). "Opfolda: Pending EC decision".
(27 June 2023). "Opfolda Product information".
{{ClinicalTrialsGov. NCT00672022. Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses
{{ClinicalTrialsGov. NCT00537602. OGT 918 in the Treatment of Cystic Fibrosis
(May 2012). "A randomized placebo-controlled trial of miglustat in cystic fibrosis based on nasal potential difference". Journal of Cystic Fibrosis.
(March 1994). "Secretion of human hepatitis B virus is inhibited by the imino sugar N-butyldeoxynojirimycin". Proceedings of the National Academy of Sciences of the United States of America.
(October 1996). "N-butyldeoxynojirimycin-mediated inhibition of human immunodeficiency virus entry correlates with changes in antibody recognition of the V1/V2 region of gp120". Journal of Virology.
(October 2008). "N-Butyldeoxynojirimycin is a broadly effective anti-HIV therapy significantly enhanced by targeted liposome delivery". AIDS.

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