Miglustat

Enzyme Stabilizer — ALIMENTARY TRACT AND METABOLISM

Key facts

Miglustat is an ofp drug in the Enzyme Stabilizer class catalogued in DrugCentral. It was first approved by the EMA in 2002. Its mechanism of action is classified as Enzyme Stabilizers and Glucosylceramide Synthase Inhibitors. DrugCentral lists 15 molecular targets, including GAA, Gba2 and GBA2. Recorded indications include Glucosylceramide beta-glucosidase deficiency, Lateonset Pompe disease and Niemann-Pick disease, type C.

• Approval status: ofp (first approved 2002 by the EMA).
• Drug class: Enzyme Stabilizer; Glucosylceramide Synthase Inhibitor.
• Mechanism of action: Enzyme Stabilizers; Glucosylceramide Synthase Inhibitors.
• Molecular targets: GAA, Gba2, GBA2 and P0504H10.9 (15 listed in DrugCentral).
• Indications: Glucosylceramide beta-glucosidase deficiency, Lateonset Pompe disease and Niemann-Pick disease, type C.
• ATC code: A16AX06 (Various alimentary tract and metabolism products).
• Molecular formula: C10H21NO4; molecular weight 219.28 g/mol.

Indications & contraindications

Drug–disease relationships curated by DrugCentral (OMOP / SNOMED concepts).

Indications (3)

• Glucosylceramide beta-glucosidase deficiency
• Lateonset Pompe disease
• Niemann-Pick disease, type C

Contraindications (4)

• Breastfeeding (mother)
• Impaired renal function disorder
• Peripheral nerve disease
• Pregnancy, function

Mechanism & targets

• Mechanism of action (MoA): Enzyme Stabilizers; Glucosylceramide Synthase Inhibitors
• Established pharmacologic class: Enzyme Stabilizer; Glucosylceramide Synthase Inhibitor

Molecular targets (15)

Target	Gene	Action	Activity
Lysosomal alpha-glucosidase	GAA	unknown	IC50 7.00
Non-lysosomal glucosylceramidase	Gba2	unknown	IC50 6.85
Non-lysosomal glucosylceramidase	GBA2	unknown	IC50 6.64
Putative alpha-glucosidase	P0504H10.9	unknown	IC50 6.38
Sucrase-isomaltase, intestinal	SI	unknown	IC50 6.30
Sucrase-isomaltase, intestinal	Si	unknown	IC50 6.24
Sucrase-isomaltase	Sis	unknown	IC50 6.22
Maltase-glucoamylase, intestinal	MGAM	unknown	IC50 5.68
Lysosomal alpha-glucosidase	Gaa	unknown	IC50 5.68
Ceramide glucosyltransferase	UGCG	inhibitor	Ki 5.13
Lysosomal alpha-glucosidase	Gaa	unknown	IC50 5.05
Glycogen debranching enzyme	AGL	unknown	IC50 5.00
Trehalase	Treh	unknown	IC50 4.89
Ceramide glucosyltransferase	Ugcg	unknown	IC50 4.64
Uncharacterized protein	TREH	unknown	IC50 4.44

Regulatory approvals (2)

Date	Agency	Marketed as	Applicant	Orphan
2003-07-31	FDA	—	ACTELION PHARMS LTD	Yes
2002-11-20	EMA	—	—	Yes

ATC classification

ATC code	Therapeutic hierarchy
A16AX06	ALIMENTARY TRACT AND METABOLISM › OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS › OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS › Various alimentary tract and metabolism products

Reported adverse-event signals

Strongest FDA FAERS disproportionality signals (log-likelihood ratio). A signal is a statistical association in spontaneous reports, not proof that the drug causes the event.

MedDRA term	LLR
Seizure	285.4
Gastrostomy	142.1
Death	141.2
Diarrhoea	110.9
Pneumonia aspiration	106.7
Disease progression	105.3
Dysphagia	104.9
Concomitant disease aggravated	102.7
Tremor	93.4
Weight decreased	82.3
Epilepsy	79.5
Neurological decompensation	69.7

Chemical structure

• Molecular formula: C₁₀H₂₁NO₄
• Molecular weight: 219.28 g/mol
• CAS number: 72599-27-0
• InChIKey: UQRORFVVSGFNRO-UTINFBMNSA-N
• PubChem CID: 51634

CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO

Frequently asked questions

Answers are generated from the structured data on this page, not written by hand.

What is Miglustat used for?

DrugCentral lists the following indications for Miglustat: Glucosylceramide beta-glucosidase deficiency, Lateonset Pompe disease and Niemann-Pick disease, type C.

How does Miglustat work?

Miglustat is classified as Enzyme Stabilizers and Glucosylceramide Synthase Inhibitors; its molecular targets in DrugCentral include GAA, Gba2, GBA2 and P0504H10.9.

When was Miglustat first approved?

Miglustat was first approved by the EMA in 2002.

What drug class does Miglustat belong to?

Miglustat is in the Enzyme Stabilizer class.

What is the chemical formula of Miglustat?

Miglustat has the molecular formula C10H21NO4 and a molecular weight of 219.28 g/mol.

Sources

• DrugCentral drug card 1807 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL1029 — compound record and bioactivity
• PubChem CID 51634 — chemical identifiers
• DrugBank DB00419 — drug record
• FDA UNII ADN3S497AZ — substance identity
• DailyMed: FDA labels for Miglustat — current prescribing information (label search)
• UniProt P10253 (GAA) — target protein
• UniProt Q9HCG7 (GBA2) — target protein
• UniProt P14410 (SI) — target protein
• UniProt O43451 (MGAM) — target protein
• UniProt Q16739 (UGCG) — target protein
• UniProt P35573 (AGL) — target protein
• FDA Adverse Event Reporting System (openFDA FAERS) — adverse-event signals, as aggregated by DrugCentral

How this page is built: methodology · editorial policy & corrections.