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Miglustat


Miglustat
Systematic (IUPAC) name
1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol
Identifiers
CAS number	72599-27-0
ATC code	A16AX06
PubChem	51634
DrugBank	APRD01118
Chemical data
Formula	C₁₀H₂₁N O₄
Mol. mass	219.28 g/mol
Pharmacokinetic data
Bioavailability	97%
Protein binding	Nil
Metabolism	Nil
Half life	6–7 hours
Excretion	Renal, unchanged
Therapeutic considerations
Licence data	US
Pregnancy cat.	X(US)
Legal status	℞-only(US)
Routes	Oral

Miglustat is a drug used to treat Gaucher disease.

It inhibits the enzyme glucosylceramide synthase,^[1] an essential enzyme for the synthesis of most glycosphingolipids.

It is only used for patients who cannot be treated with enzyme replacement therapy with imiglucerase. ^[2]

It is marketed under the trade name Zavesca.

It has also been investigated for use in treating Niemann-Pick disease.^[3]

References

^ van Giersbergen PL, Dingemanse J (2007). "Influence of Food Intake on the Pharmacokinetics of Miglustat, an Inhibitor of Glucosylceramide Synthase". doi:10.1177/0091270007305298. PMID 17720777.
^ Weinreb NJ, Barranger JA, Charrow J, Grabowski GA, Mankin HJ, Mistry P (2005). "Guidance on the use of miglustat for treating patients with type 1 Gaucher disease". Am. J. Hematol. 80 (3): 223–9. doi:10.1002/ajh.20504. PMID 16247743.
^ Patterson MC, Vecchio D, Prady H, Abel L, Wraith JE (2007). "Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study". Lancet neurology 6 (9): 765–72. doi:10.1016/S1474-4422(07)70194-1. PMID 17689147.

v • d • e Other alimentary tract and metabolism products (A16)
Amino acids and derivatives	Levocarnitine - Ademetionine - Levoglutamide - Mercaptamine - Carglumic acid - Betaine
Enzymes	Alglucerase - Imiglucerase - Agalsidase alfa - Agalsidase beta - Laronidase - Sacrosidase - Alglucosidase alfa - Galsulfase - Idursulfase
Various alimentary tract and metabolism products	Tioctic acid - Anethole trithione - Sodium phenylbutyrate - Nitisinone - Zinc acetate - Miglustat