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Miglustat

Generic Name
Miglustat
Brand Names
Opfolda, Zavesca, Miglustat Dipharma, Miglustat Gen.Orph, Yargesa
Drug Type
Small Molecule
Chemical Formula
C10H21NO4
CAS Number
72599-27-0
Unique Ingredient Identifier
ADN3S497AZ
Background

Miglustat, commonly marketed under the trade name Zavesca, is a drug used to treat Gaucher disease. It inhibits the enzyme glucosylceramide synthase, 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. Miglustat is now the first and only approved therapy for patients with Niemann-Pick disease type C (NP-C). It has recently been approved for treatment of progressive neurological symptoms in adult and pediatric patients in the European Union, Brazil, and South Korea. Miglustat was first developed as an anti-HIV agent in the 1990s. However, clinical experience with miglustat showed that therapeutic levels of the drug could not be achieved in patients without a high incidence of adverse effect.

Indication

For the treatment of adult patients with mild to moderate type 1 (nonneuropathic) Gaucher's disease for whom enzyme replacement therapy is not a therapeutic option (e.g. due to constraints such as allergy, hypersensitivity, or poor venous access). Now approved in some countries for the treatment of progressive neurological symptoms in adult and pediatric patients with Niemann-Pick disease type C (NP-C).

Associated Conditions
Mild, moderate Gaucher Disease, Type 1
Clinical Trials
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• The FDA has approved two new treatments for Niemann-Pick Type C, a rare and fatal genetic disorder, marking a significant milestone for patients and families. • Miplyffa (arimoclomol) by Zevra Therapeutics, in combination with miglustat, is approved for neurological symptoms in patients aged 2 and older. • Aqneursa (levacetylleucine) by IntraBio Inc. has also been approved as a stand-alone therapy for neurological manifestations of Niemann-Pick disease type C. • These approvals underscore the FDA's commitment to supporting the development of new treatments for rare diseases, providing hope for improved management of symptoms.
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